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Addressing the Barriers to Patient Access RIGHT PATIENT, RIGHT MEDICINE, RIGHT NOW
By JONATHAN WILCOX / Policy Director, Vital Options International
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By STACEY L. WORTHY, Esq. / Director of Public Policy, The Alliance for the Adoption of Innovations In Medicine t may seem obvious to proclaim that the cancer patient journey is one
T
of crossroads moments and good news/bad news scenarios. But per-
he definition of their insurance companies finally don’t have to travel to the doctor’s haps now more than ever, the oncology world is stirred by astonishing
“conundrum” is agree to pay for the drug prescribed office every week, resulting in time breakthroughs in lifesaving medicines simultaneous with ever-higher
“a confusing and by their doctors. away from work or family. Yet, be- and more complex barriers to access for patients who need them.
difficult problem One disturbing and wasteful insur- cause the co-pays can be hundreds When it comes to the science of cancer, these are definitely the best
or question,”—a ance practice is what is euphemisti- or thousands of dollars per month, of times. Exciting discoveries occurring in every area of cancer research
definition that clearly cally called “step therapy” or “fail studies find almost 10 percent of are producing treatments targeted to the unique molecular and genetic
applies to the growing number of first.” In this practice, although a insured patients choose not to fill characteristics of each person’s cancer. Add the excitement over new
cancer patients who are prescribed doctor may prescribe a medication their initial prescriptions for oral immunotherapies that train a patient’s immune system to destroy cancer
state-of-the art cancer medicines most suitable to the patient’s indi- anti-cancer medications. and it is clear that the transformative change of precision medicine is well
and yet, delay taking them or don’t vidual needs, the insurance company To save lives, the patient com- underway.
take them at all. usurps the doctor’s prescribing munity, cancer advocacy groups, For some cancer patients seeking to utilize these life-saving thera-
According to the latest projec- authority by requiring the patient to and public health organizations are CONTACT YOUR ELECTED pies, however, all the world’s medical innovation might as well never have
tions, nearly half of Americans first fail on several inferior medica-pressing Congress and state legis- OFFICIAL to Support Patient occurred. Even as medical advances create new, potentially life-saving
latures to cap co- Access to Medical Innovation medicines for patients – and more quickly than ever before – ready access
pays on specialty to these life-enhancing therapies is not assured.
medicines and American College of Rheumatology Why? The path is blocked in two critical ways.
“We have a health insurance failure for innovative medicines. ensure equality of LEGISLATIVE ACTION CENTER: The first is governmental. What we call “new” cancer treatments are
www.rheumatology.org/actioncenter
If you get cancer today, your copays can prevent you from access and insur- often 8-10 years old by the time they reach patients. Why isn’t this a mat-
ance coverage American Society of Hematology ter of months? The main reason is that FDA regulations were designed
accessing the medicines you desperately need. That’s a failure for all anti-cancer ORAL CANCER DRUG PARITY: to evaluate the modernism of the VCR and 8-track tapes. Appropriate for
of health insurance.” Tomas Philipson, Ph.D., Professor, University of Chicago regimens. To date, www.hematology.org/Advocacy/
Campaigns/667.aspx
their time, perhaps, but increasingly ill-equipped to co-exist with today’s
39 states and speedy scientific advances.
Washington D.C. Arthritis Foundation The second is access. Tens of thousands of cancer patients are custom-
have enacted oral SPECIALTY TIERS/CAP THE COPAY: arily confronted with rising copays, restricted coverage and all too often,
diagnosed with a chronic disease tions before it will pay for the drug chemotherapy access laws, while 15 www.arthritis.org/advocate/ insurance practices that deny access completely. This is the bitter reality
take their medicine as directed that the doctor originally prescribed. states and the District of Columbia our-policy-priorities/pass-the-patients of today’s access fights – with patients and their doctors denied medica-
-access-to-treatments-act.php
only 50 percent to 60 percent of Equally onerous is placing new can- have either introduced or passed tions every day by a complex coverage and reimbursement insurance
the time. The consequence of this cer medicines into the highest phar- bills to limit what patients pay for International Myeloma Foundation system that surely does not want to be seen as standing in the way of sick
poor medication adherence is more macy (“specialty”) tier and forcing specialty medicines. ORAL ANTICANCER TREATMENT people and their disease-altering treatments.
doctor visits, trips to the emergency patients to pay a large percentage of The Alliance for the Adoption of ACCESS LEGISLATION: In response, a patient revolution has ignited and touched down in 39
www.cqrcengage.com/myeloma/access
room, and hospitalizations, all of the drug’s price. According to a re- Innovations in Medicine supports states and Washington, D.C. to address the problem of inequitable cover-
which is estimated to cost the health cent Leukemia & Lymphoma Society these policies because adhering to Leukemia & Lymphoma Society age by requiring health plans to equalize the patients’ out-of-pocket costs
care system between $100 billion report, even the new state exchange cancer medicines is a necessity. It is LEGISLATIVE ACTION CENTER: between oral and intravenous therapies. Further state-by-state reform
and $300 billion annually. plans implemented under the Patient time to end the adherence conun- www.lls.org/advocate/legislative- efforts will surely follow.
action-center
However, for Americans fighting Protection and Affordable Care Act drum by reducing the cost-sharing As a nonprofit organization working to improve cancer care, Vital
a deadly cancer, the consequences for previously uninsured patients use barriers for cancer patients, so they National Organization for Options International (VOI) believes patients should not be forced to battle
can also be life-threatening and this cost containment strategy. This can fill their prescriptions and take Rare Diseases the system and their cancer at the same time. This is why VOI supports
CURRENT INITIATIVES AND ISSUES
thus, the conundrum: Why are can- means patients can pay up to 50 the medicines they desperately need. energetic and innovative movements underway to update the drug ap-
UPDATES:
cer patients skipping doses or tak- percent of the costs of new can- It’s the right thing to do for www.rarediseases.org/advocacy/ proval process using 21st century computing capabilities, genomic analysis
ing less than the prescribed amount cer therapies, which leads to poor patients, the healthcare system and initiatives-updates and a new understanding of cancer on a molecular level.
of their cancer medicines? The adherence. our economy. We also hope legislators and regulators will work with doctors,
Patient Services, Inc.
answer is simple: In the name of Then, there is the practice of LEGISLATION: patients, caregivers and payors to provide faster and easier access to the
cost-containment, insurance com- shifting as much as 25 percent of the www.patientservicesinc.org/ latest medications – and do so in a way that prioritizes patient safety and
panies increasingly restrict patients’ cost of oral anti-cancer medicines Advocacy/Legislation supports the integrity of new drug evaluations.
access to breakthrough cancer to patients in high co-pays—even To almost countless patients, there is only one obvious move left:
medicines through high copays and though these drugs are less invasive Towards a regulatory future that embraces access and accepts nothing less
practices that require patients to fail than intravenous infusions, they than the right patient, the right medicine and right now.
on medication after medication until carry fewer side effects, and patients
COVER ILLUSTRATION by Nicholas Sutton Bell
The visionaries who continue to reshape biotechnology How a nation’s policies attract and discourage biopharma investment Head, Data Analytics,
Scientific American Custom Media | YALI FRIEDMAN
BY MEIR PEREZ PUGATCH, DAVID TORSTENSSON, RACHEL CHU, AMIR DAYAN & NOA WEINSTEIN
—and the world Copy Chief & Content Consultant | GEORGINA KEENAN
Digital Production | ZOYA LYSAK
BENCH TO BUSINESS WORLDVIEW EVENTS
Research & Administrative | ALEXANDRA HARIRI
22 FROM EUREKA TO USEFUL 72 AN EVENING WITH TOP MEDICAL INNOVATORS BOARD OF ADVISERS
Developing an idea into a productive tool demands Today’s visionaries battle cancer with cutting-edge science and the stubbornness GEORGE BAEDER
experience, foresight and a persistent champion to make it work BY MIKE MAY Director, China Global Insight
JORGE BARRERO
BY MIKE MAY Deputy to the President,
Spanish Association of Biotechnology
COUNTRY SPOTLIGHTS
26 ROOM TO GROW MARY BOOTE
CEO, Truth About Trade and Technology
LabCentral gives start-ups the space and 76 DISPATCHES FROM: China, Germany, India, Japan, Norway, Poland, STEVEN CASPER
Henry E. Riggs Professor of Management,
resources to thrive BY JOHANNES FRUEHAUF Romania, Russia, Scotland, United States Keck Graduate Institute
ANANDA CHAKRABARTY
28 BIOTECHNOLOGY’S WORLDVIEWPOINT
Distinguished University Professor,
University of Illinois at Chicago
CRUCIAL QUESTION JOSEPH DAMOND
Senior Vice President, International Affairs, BIO
In this industry, what matters the most: technology 84 AGING 2.0 NINA DUDNIK
Founder and CEO, Seeding Labs
or people? BY STEPHEN M. SAMMUT Stem cells and digitized DNA may hold RICHARD GALLAGHER
the key to high-performance longevity President and Editor-in-Chief, Annual Reviews
BY ROBERT HARIRI ANITA GOEL
Chairman and CEO, Nanobiosym
BOB HARIRI
Chairman, Celgene Cellular Therapeutics
KAREN M. HAUDA
Senior Director, Regulatory Policy, Novo Nordisk
ROBERT KILPATRICK
Cofounder and Partner, Technology Vision Group
2015 GANESH KISHORE
CEO of Malaysian Life Sciences Capital Fund
TOMASZ MROCZKOWSKI
Professor, Department of International Business, Kogod
School of Business, American University
ANDREW POWELL
CEO, Asia Biobusiness
STEVE SAMMUT
Senior Fellow, Health Care Management,
Wharton School, University of Pennsylvania
RAM SASISEKHARAN
Alfred H. Caspary Professor of Biological Engineering
and Health Sciences and Technology, Massachusetts
Institute of Technology
I
typically fails when there being have turned single n the past two decades,
is multicausality for any illnesses, such as cancer, the pace of medical
SPECIAL REPORT
TRACKING INNOVATION’S
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n our article “Trans- fails. In addition, patients and other strategies, such forms of cancer.
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forming Medicine: A are unique genetically and as immune therapy. Yet, Despite progress, chal-
LETTER FROM THE EDITORS
Manifesto” (Scientific environmentally, and hence virtually every researcher, lenges remain in translating
he cornerstone of Scientific American which highlights some of the top innovators in medicine American Worldview, 2014), should not be averaged in company and regulatory basic science discover-
Worldview—our annual Scorecard, evalu- and two of the patients who reaped the benefits (page 72) we challenged the founda- large populations. Rather, agency is organized around ies into medical practice.
ating the life science innovation capacities of modern medical ingenuity. As in previous years, our tion of “evidence-based the personalized multi- the one drug–one disease Whereas the 20th century
of countries around the globe (page 36)— “Country Spotlights” section (page 76) will take readers medicine.” We noted that dimensional data clouds of business model. saw a focus on develop-
emerged from a desire to bring a much- on a tour of science-in-action, showcasing biotechnology methods used to generate each individual should be Consumers—via social ing better treatments for
needed critical eye and third-party trans- success stories around the planet. this evidence, the basis for analyzed independently, networks, multi-dimen- disease, there has been far
parency to the international landscape of Last but far from least, the worldVIEWpoint (page 84) less attention to preven-
biotechnology. We are proud to have done this for seven essay reveals a modern, data-infused approach to finding ... patients are unique genetically and environmentally, and hence should tion, early detection and
years now, adding an objective, data-driven voice to an a science-based “fountain of youth.” The author, a pioneer interception of disease.
arena with a lot of unchecked cheerleading. This year, we in stem cell therapeutics, describes his journey into this
not be averaged in large populations. The impact of condi-
add another data-led feature—albeit far less quantitative— field with an ironic story about turning what was once tions like Alzheimer’s
titled The Worldview 100 (page 8), celebrating the most in- considered medical waste into cutting-edge medical treat- medical practice and reim- and then patients with sional data clouds and disease, cancer, rheuma-
fluential people in biotechnology, as determined through ments. His success proves that life-changing advances can bursement—randomized similar features can be digital tools—are decipher- toid arthritis and diabetes
nominations and and do materialize from highly surprising sources. clinical trials (RCT) and aggregated into related ing biological complexity to continues to burden global
selections from We want to encourage readers But that’s just what we seek to uncover—the stories in comparative effectiveness groups of interest (e.g., match people to combina- healthcare systems. Patients
our international which something unexpected makes the difference. One research—are dangerously those responding effectively tions of treatment. They affected by these diseases
panel of experts.
to explore biotechnology from a of the guiding themes of Scientific American Worldview is broken. to a drug). We are throw- are bypassing large clinical often spend years of their
As with any list, broad and global perspective. that innovation and ingenuity can occur in the most un- There is a broad biparti- ing away unknown hoards trial networks to get better lives in a state of declining
this one will sure- likely places. We’ve found evidence of that in every edition san effort to move towards of relevant data about both answers more quickly with health. According to the
ly spawn differences of opinion about the people on it and of our Scorecard. As our ranking system shows year after personalized and precision the variability of response individualized approaches U.S. Centers for Disease
those missing. Ultimately, though, driving discussion lies year, small or resource-challenged countries can dominate medicine because getting and multiplicity of causes to their disease. We should Control and Prevention,
at the heart of this publication’s mission. We want to en- various aspects of biotechnology achievement, and even the right treatments to the at the genetic and pheno- enable this movement to in the United States alone,
courage readers to explore biotechnology from a broad the giants can tumble on some metrics. This dynamic and right patients at the right typic level when we average accelerate personalized about half of all adults, 117
and global perspective. developing industry continues to intrigue and amaze us. time can reduce the cost of patient populations. With medicine. million people, live with at
Two sections—one on turning an idea into a product And we are once again delighted to bring you the people, healthcare and save lives. the analysis of individual least one chronic condition.
(page 22) and another on the successes, failures and fu- places and perspectives that are moving it forward. But good policy has to be patients we can gain fun- COLIN HILL Earlier this year, Jans-
LEROY HOOD
ture directions in communication about biotechnology As always, we offer our profound thanks to our sponsors guided by good science and damental new insights SUI HUANG
sen Research & Develop-
(page 30)—trigger fresh ways of thinking about these top- and partners: Celgene Corporation, Cure Forward, Cred- a systems-driven medicine into disease through N=1 STUART KAUFFMAN ment, one of the Janssen
ics. We continue this focus on fostering new dialogue with ible Meds, and The Biotechnology Industry Organization. that is predictive, preven- experiments and appropri- —founders of Transforming Pharmaceutical Companies
articles about the impact of location on drug development tive, personalized and par- ate statistical power can be Medicine: The Elizabeth of Johnson & Johnson,
Kauffman Institute
(page 66) and which countries excel and flounder when it Sincerely, ticipatory (P4). Marginal gained through the aggre- announced the launch
comes to attracting biotechnology investment (page 68). Jeremy Abbate, Publishing Director changes to evidence-based gation of related patients. of three new research
We did not, however, set out to spark debate from every Mike May, Editorial Director medicine (e.g., comput- Further, the one drug– platforms focused on
element of the 2015 Scientific American Worldview. And Yali Friedman, Head, Data Analytics erizing data collection) one disease model of inno- disease prevention, disease
few are likely to contest one “list” in this year’s edition, will themselves have only vation (based on Pasteur’s interception and the mi-
P
make enormous gains
and development—an era sporadically and usually in resident Obama’s ics International (CDI) pecially critical to treating
Our healthcare system is strapped with the marked by the promise of remote areas, there simply Precision Medicine that ultimately may lead
in understanding how them. Advancing research
burgeoning costs of chronic care and will new firsts, including a day is no commercial market. Initiative recently to a treatment for macular to approach medical through genetic testing
ultimately seek more cost-effective approaches. when the term “patient” is a Who would buy it? Out- pledged US$215 million to degeneration with an au- treatment. and affecting change in the
historical artifact. breaks usually involve only fund research to use per- tologous (the patient’s own) regulatory process should
the human genome, we therapeutic or combination a couple of hundred cases sonalized genetic informa- iPSC-derived retinal cell remain key areas of the
WILLIAM N. HAIT
are able to understand an approaches for T1D inter- and typically occur every tion as the starting place for transplant. This is expected initiative’s multi-pronged
Global Head
individual’s susceptibility ception by understanding Janssen Research & few years in poor rural understanding individual to lead to the first clinical HEMATOLOGY approach. However, clinical
to disease, calculate one’s
risk and intervene before
the role of potential triggers
and initiating steps on the
Development, LLC
Raritan, New Jersey
communities in Africa. So,
manufacturers would be
response to disease and
treatment. While the initia-
trial in the United States
using iPSCs.
TRIAL NUANCES trial management can’t be
forgotten.
L
illness occurs. pathway to T1D. unlikely to see a return on tive is necessary, it fails to The convenient ouis J. DeGennaro’s Enhancing and refin-
Today, early disease in- The Janssen Human that investment. address the questions that criticism of personalized “Embracing The ing our understanding of
terception and prevention Microbiome Institute THE EBOLA- This means that even DNA and genetics alone medicine is the perceived Process” (Scientific these cancer pathologies
strategies are still largely (JHMI) was designed to VACCINE if one of these candidate cannot answer, questions cost. But the price should American Worldview, 2014) can increase the number of
unexplored for many of
the illnesses that plague
explore the relationship of
the organisms that inhabit
FUNDING GAP Ebola vaccines receives
clinical approval, we’ll still
that must be posed at the
cellular level, where the
decline, just as the cost of
sequencing the human ge-
draws much needed atten-
tion to the lack of targeted
targeted therapies to enable
truly personalized treat-
W
our societies. The Janssen our body in maintaining ith the number be left with a significant answers may dramatically nome rapidly declined. The treatment options available ments. Moreover, ensuring
Prevention Center (JPC) health and causing disease. of new cases of funding gap. Gavi, the Vac- improve patient healthcare. time is right for the United for acute myeloid leukemia careful trial design and
will focus on the preven- Ultimately, this unique Ebola appear- cine Alliance, is committed Induced pluripotent States to fully embrace (AML). I applaud the steps precise trial execution will
tion of chronic, non-com- Janssen initiative will de- ing to level off, and with to purchasing necessary stem cells (iPSCs), with this rapidly developing the Leukemia & Lymphoma yield data that can inform,
municable diseases such as velop new ways to maintain clinical trials for Ebola vaccines for this outbreak the potential to be manu- field with research dollars. Society is taking to address and perhaps significantly
Alzheimer’s, heart disease, health and treat disease. vaccines now underway, and creating a stockpile for factured from virtually Personalized medicine has this unmet medical need impact, the greater oncol-
cancer and autoimmune External collaborations for there is an almost palpable future outbreaks as well as any patient’s blood cells, arrived, and the United with the Beat AML initiative. ogy community.
diseases, which increasingly this organization will be sense that the crisis is over. incentivizing next-genera- set the stage for research States needs to lead this Beat AML needs to also
RAY REILLY
impact aging populations fostered through anchor The reality, however, is that tion vaccine development, to make enormous gains race to the finish line. consider the impact clinical
Director of Project
and burden healthcare sys- research centers located in even if a safe and effective but as a public-privately in understanding how to trial management has on Management
tems globally. The JPC will Cambridge, Massachusetts, vaccine emerges and the funded global health orga- approach medical treat- BOB PALAY commercial success and the Hematology Division
CEO and Chairman Novella Clinical
leverage the world-class and Beerse, Belgium. epidemic is brought under nization we still have not ment. These personalized Cellular Dynamics
nuances surrounding prop-
Morrisville, North Carolina
vaccine discovery expertise During the last 120 control, we are still in many yet determined who will stem cells provide a more International (CDI) er hematological oncol-
of the former Crucell Vac- years, Johnson & Johnson ways no better prepared for provide these funds. comprehensive system for Madison, Wisconsin ogy study design. Because
cine Institute (The Nether- has pushed forward many future outbreaks than we We need to stop waiting understanding the behavior hematologic oncology trials Contact us at:
lands) in collaboration with “firsts”—evolving from were a year ago. for evidence of a disease of particular diseases than are fundamentally different saworldview@sciam.com
We developed and finalized this list in several steps through a process carried out from December 2014 through March
2015. First, we invited dozens of leaders in biotechnology and biosciences to nominate their choices for the most influential
people in the field. We encouraged the nominators to select living experts currently working in the sector from a range of
contributing areas, including industry, academia, public policy, finance, law and beyond. That process generated a list of
almost 400 nominees. We then recruited more experts—many of them from the original group of nominators—to suggest
anyone on the list of nominees worthy of selection to a more refined coterie of the 100 most influential figures in today’s
world of biotechnology. For the final step, we tallied the votes, and the 100 people most selected formed The Worldview
100. Here, we highlight the 10 individuals who received the highest number of votes. We present the other 90 honorees in
alphabetical order.
GEORGE CHURCH
Church started his career with sequencing, especially through the development Hood played a role in the development of five instruments that drive today’s bio-
of advanced devices, and he continues to help scientists collect and analyze data logical sciences: automated DNA sequencers, DNA synthesizers, protein sequenc-
LEE HOOD
about the most basic life traits. As the director of PersonalGenomes.org, he pro- ers, peptide synthesizers and an ink-jet printer for constructing DNA arrays. Today,
vides open access to data that explore the foundation of human traits around the he works on integrating biology, computation and technology to build so-called P4
world. His work on next-generation sequencing and cellular and tissue engineer- medicine, which is predictive, personalized, preventative and participatory. In the 2012
ing spawned a dozen companies based on medical genomics or synthetic biology. Scientific American Worldview, Hood posited that the traits of a successful entrepre-
Kirkus Reviews called his book Regenesis, coauthored with science writer Ed Regis, neur are “having a clear picture of the future that is very different from what other
“a valuable glimpse of science at the edge.” people have, and an ability to drive towards that future.”
director | U.S. National Institutes of Health | Bethesda, Maryland, U.S. professor of biology | MIT | Cambridge, Massachusetts, U.S.
FRANCIS COLLINS
ERIC LANDER
“I think my greatest contribution to biotechnology arose from the charge I was After publishing a 2005 article in Nature on the chimpanzee genome, Lander said,
given to lead the international Human Genome Project,” Collins told Scientific “The goal is not just butterfly collecting or mammal collecting to simply describe
American Worldview. “Through the dedicated work of 2,400 scientists in six countries, mammals. All of that comparative work across mammals is about informing the
we successfully sequenced and made immediately available the 3 billion base pairs human genome for medicine. Until we actually understand all the working parts
in the human genetic blueprint—ahead of schedule and under budget.” To make within our genome, we won’t really be able to practice the most informed medi-
biotechnology even more effective, he said, “It is especially important to support cine.” As a core member of the Broad Institute, Lander continues to explore what
the creative minds who are pursuing high-risk projects that, if successful, may genomics can tell us about human physiology and diseases—especially how to
yield high rewards for expanding biological knowledge or fighting human disease.” treat them.
BOB LANGER
BILL GATES
Best known as the cofounder of Microsoft, Gates turned his wealth into philan- to create materials that enable the controlled release of macromolecules.” Such
thropic giving through his and his wife’s foundation, which, its website states, devices can deliver drugs—even genetically engineered proteins—for long periods,
works to “bring about the kinds of changes that will help people live healthier and and Langer is even working on versions that can be controlled through magnetic,
more productive lives.” Clearly, innovation has always played a fundamental role ultrasonic and enzymatic methods. The best way to increase the effectiveness of
in his career. He once noted, “I believe in innovation and that the way you get biotech today, he said, is to provide “more funding for basic research.” Langer’s
innovation is you fund research and you learn the basic facts.” That thinking took output—including more than 1,000 patents, which have been licensed to over 300
him a long way. companies—attests to his indefatigable drive.
co-chair and trustee | Bill & Melinda Gates Foundation | Seattle, Washington, U.S. founder | J. Craig Venter Institute | La Jolla, California, U.S.
MELINDA GATES
CRAIG VENTER
In her 1982 high school valedictorian address at Ursuline Academy in Dallas, In 2013, Venter told Bloomberg, “Genome design is going to be a key part of the
Texas, Melinda Gates offered the following wisdom: “If you are successful, it is future. That’s why we need fast, cheap, accurate DNA synthesis so you can make
because somewhere, sometime, someone gave you a life or an idea that started a lot of iterations of something and test them.” By then, he and his colleagues had
you in the right direction. Remember also that you are indebted to life until you already created a bacterial genome from scratch. Now, as the CEO and cofounder
help some less fortunate person, just as you were helped.” At that time, no one of Human Longevity, Inc., Venter hopes to combine information about various
could even imagine the help that she would bring to the world. According to her biological features—including the genome, proteome, biome and more—with
foundation’s website, it has given grants totaling US$32.9 billion. advanced algorithms and computing to create new therapies to extend our years of
high-quality life.
former commissioner | U.S. Food & Drug Administration | Silver Spring, Maryland, U.S.
PEGGY HAMBURG
JANET WOODCOCK
director | Center for Drug Evaluation & Research | U.S. Food & Drug Administration
managerial capacity. Before working for the FDA, she served as commissioner Silver Spring, Maryland, U.S.
of the New York City Department of Health and Mental Hygiene, where she Woodcock helped to develop a regulatory framework to accommodate future
significantly slowed the spread of tuberculosis. When President Obama named advances in biotechnology. Nonetheless, the discipline’s effectiveness could be en-
Hamburg the FDA commissioner in 2009, Georges Benjamin, then executive hanced even more, she said, through “greater attention to translational science.” So
director of the American Public Health Association, said, “She’s all about integrity far, the biggest impact biotech has had on our daily lives is “the food revolution,”
and science…. She can be tough when she needs to be, and she’s going to need to she said, adding that “forms of gene therapy may be ‘coming of age.’” And the most
be real tough in that job.” How right he was. embarrassing moments of her career? “Too numerous to count!” she answered.
©KEITH NEGLEY
Larry Page once said: “Invention Ntseoane’s story illustrates a fun- ing.” And when assessing the way for- stumbled through five of them while
is not enough. Tesla invented damental truth in the field of bio- ward—from the discovery in the lab to its business faltered. But when Jobs
the electric power we use, but technology: getting the most out of a working toward commercialization— took over again, it rose to become the
he struggled to get it out to technical innovation requires vision, he warns against a common mistake: world’s most valuable company. The
people. You have to combine
both things: invention and inno- FROM EUREKA courage and commitment—plus the
ability to take advantage of available
underestimating the task. “The major
thing people do is underestimate how
lesson, Langer stresses, is that even
the most astoundingly innovative
TO USEFUL
vation focus, plus the company resources. long it takes, how expensive it is and ideas need the leadership of a cham-
that can commercialize things how many difficulties you’ll run into.” pion to succeed.
and get them to people.” ACCELERATING In order to combat these issues, he
Delivering cutting-edge Developing an idea into a productive tool demands experience, INNOVATION’S APPLICATION encourages biotech entrepreneurs to MAKE THE RIGHT MEASURES
F
biotechnology to people around “Innovation is the creative lifeblood of be prepared. “Surround yourself with The excitement of a eureka moment
foresight and a persistent champion BY MIKE MAY
the world requires a host of every country,” wrote The Honorable great people,” he says, “and have more in the lab, however, can get some sci-
capabilities apart from simply or decades, a combination of politics and peer pressure pushed Birch Bayh in the forward to Michael capital rather than less.” entists moving too fast. “If the ambi-
having an innovative concept Eve Ntseoane away from farming in her native South Africa. A. Gollin’s Driving Innovation. Many Also, Langer firmly believes that tion is to commercialize an idea,” says
with great potential. This sec- Although her parents both grew up on farms, they couldn’t stay would argue that the same could be any innovation with a good chance of Anders Nordström, a senior advisor at
tion delves into that journey there as adults. “Black people were moved to townships, because said for the complex and promising successful commercialization requires Sweden’s Uppsala Innovation Centre,
“from bench to business” by of remnants of the Land Native Act of 1913,” she explains. During field of biotechnology.
meeting the researchers, men- school vacations, though, Ntseoane’s mother would take her and The question is: How can scien- In South Africa, Eve Ntseoane
tors, CEOs, nonprofit leaders, her brother to a farm where her uncle worked. “As a child I hated tists turn “eureka moments” into more than doubled her yield
venture capitalists and end us- every first few days of the visit, but with chickens and puppies around, I would useful products and services more with genetically modified corn.
ers whose lives and livelihoods start enjoying it,” she remembers. “One morning my cousin and I climbed on quickly, more effectively? In Think
have been forever changed by it the wagon carrying farmworkers to the corn fields. It was fascinating to help Like a Freak, Steven D. Levitt and Ste-
(see “From Eureka to Useful,” out, though we played most of the time.” phen J. Dubner write: “The modern
this page). Then, a look inside Ntseoane studied agriculture during her first three years of high school, but world demands that we all think a bit
LabCentral’s “launchpad” for she says, “It came with a stigma that farming was for the not-so-clever.” Eventu- more productively, more creatively,
bioentrepreneurs (page 26) and ally, she succumbed to the peer pressure and her parents’ wishes, and became a more rationally; that we think from
an essay by industry expert teacher. She only taught for three years before moving to the corporate world, a different angle, with a different set
Stephen Sammut (page 28) where she worked in communications. of muscles, with a different set of ex-
attest to the countless ways Nonetheless, her past and changing social circumstances eventually lured pectations; that we think with neither
people in our field get together Ntseoane back to the corn fields. Through the South African government’s fear nor favor, with neither blind op-
to nurture the evolution of ideas Land Reform Strategy, she obtained a 539-hectare farm south of Johannesburg. timism or sour skepticism.”
into commercial products. As The plot is situated “near a little town called Vanderbijlpark, which is popular In addition to that new kind of
these stories illustrate, this for the Vaal Dam that supplies water to most South African provinces,” she says. thinking, would-be innovators must
process often takes a number She began farming corn, working with traditional varieties. But in 2011, also learn from experience. As Lev-
of complicated, yet fascinating, she says, “AfricaBio, an organization in South Africa, introduced me to Bt itt and Dubner point out, “The key
turns along the way. maize.” This corn gains insect resistance through a bacterial gene derived from to learning is feedback. It is nearly
ROOM TO GROW
work here every day. The LabCentral environment provides tremendous
Upon moving in, the typical company consists of four flexibility that spurs innovation. As Michael Schrader,
people or less, most of them from academic institutions or cofounder and CEO of Vaxess Technologies, explains:
LabCentral gives start-ups the space and teaching hospitals. To move forward with their technol- “LabCentral has allowed us not to expend a ton of money
I
for equipment or
resources to thrive BY JOHANNES FRUEHAUF
long-term leases. As
n 2006, when Peter Parker and I started Cequent we look to expand,
Pharmaceuticals, we needed a place to work, to do we have grown our
our science. There were loads of space options for The result is an open floor plan with a very deliberate network of contacts
tech companies that only needed a good computer traffic flow pattern and transparent work, lab and tenfold through the
and a high-speed Internet connection to launch a play spaces that inspire collaboration and sharing. collection of compa-
blockbuster, but few options for biotechnology com- nies at LabCentral,
panies like ours. We needed labs with sophisticat- compared to operat-
ed—meaning expensive—equipment, and the permitting, ing on our own. We
infrastructure and people to support it. Getting the right love the facility, the
lab space setup took us six months and consumed precious ogy, they have already secured the rights to the underlying open floor plan and comingling with other teams who are
resources, both human and financial, that we should have intellectual property through an option or a license agree- not directly competing with our science, but rather com-
been spending on science. There had to be a better way. ment. Many are repeat founders or CEOs with experience plementing it— facing the same challenges in fundraising,
In 2010, we started to believe that sharing could be the launching and growing life science ventures. Others are science and business development, from whom we can
answer. We had just sold Cequent, and Peter and I both FILLING A VOID first-time entrepreneurs. learn and with whom we can share to the benefit of all.”
started new ventures—Bio-Innovation for Peter and Cam- Biotech start-ups need lab space and resources to test out, Having started working at LabCentral, companies may Schrader’s neighbors agree. “LabCentral makes it as
bridge Biolabs for me—at the Cambridge Innovation Cen- challenge and nurture early ideas. To do that, these bud- grow, adding headcount quickly. Because they have access easy to start a biotech company as it is
to start a purely
ter (CIC). At that facility, we thrived in the creative atmo- ding companies usually need affordable, move-in-ready to millions of dollars worth of the best lab equipment pos- IT company,” says P. Shannon Pendergrast, cofounder and
sphere of the shared space, and decided to try to adapt the laboratory facilities. That’s just what LabCentral provides, sible, they can perform experiments that they could never chief science officer of Ymir Genomics. “It provides the
co-working model into a shared laboratory environment and more. It gives emerging life science companies the have dreamed of if they had to do it alone. What’s more, critical infrastructure, including sophisticated scientific
for biotechnology. bench space, equipment, infrastructure and services that ©KEITH NEGLEY / PHOTOS: ©PAUL AVIS they can spend their capital and time on advancing their equipment bio-innovators need to excel.” Most important,
Cambridge Biolabs proved the concept that a biotech they need for the early-stage research that is necessary to science rather than buying equipment or building infra- he adds, it allows Pendergrast and his fellow bioentrepre-
can get started on a small scale in a shared lab space. So transition into commercial-stage enterprises. structure. This gives them the flexibility to morph their neurs the freedom to focus on the work at hand: “All I have
Peter and I joined forces with Tim Rowe, CIC’s founder and Nonetheless, it’s the “more” that makes up LabCen- original vision into one with greater promise, to reach to do is science.”
CEO, to found a nonprofit called LabCentral. A US$5 mil- tral’s “secret sauce.” As the tech world figured out long their milestones more quickly, and makes it more likely
lion grant from the Massachusetts Life Sciences Center got ago, ideas expand when they can bump, mix and mingle for them to succeed overall. Johannes Fruehauf is the president and executive director of
us started, and we opened in late 2013 on Kendall Square with others; but this rarely occurs in life sciences start- Although some might call LabCentral an incubator, LabCentral. For more information, visit www.labcentral.org.
in Cambridge. Over time, we leveraged that funding to ups, which typically set up in traditional lab settings with we eschew that term. It suggests that biotech start-ups are
bring in nearly $20 million in additional commitments of distinct and separate accommodations. Mingling hap- fragile, needing life support to survive. The opposite is true
cash, state-of-the-art equipment and in-kind services. pens every day at LabCentral, and deliberately. In fact, for the companies we select to take residency here. So, we
T
BY STEPHEN M. SAMMUT the starting point of new venture cre- into people with entrepreneurial lean- of strategies, including licensing and its people.
ation in this industry and all others. ings? It means there must be 15,000 partnering.
he reflexive answer to dustry is entirely people driven and Entrepreneurs possess a combination entrepreneurial founders. If each com- Capital formation, replenishment Stephen M. Sammut is a senior fellow,
this question is most enabled. The very definition and re- of native skills, education, experience pany needs, say, six entrepreneurial and stabilization: define capital re- health care management at The Wharton
School of the University of Pennsylvania
likely: the technology. alization of a product needs definers and personality traits that put them enablers, that would compute to a to- quirements and sources over long in Philadelphia, and developer of the
Without a new meth- and builders who know how to find in a category of their own. Some are tal of 90,000 of them worldwide. So, time periods, and accumulate the fi- Biotechnology Entrepreneurship Boot Camp.
od, cure, device or and marshal the necessary intellec- aware early in their careers that en- overall, the industry requires 105,000 nancial resources necessary to meet
something that makes tual, managerial and financial capital trepreneurship is their career destiny. bio-entrepreneurs. the development and commercial ob-
a difference in the to make things happen. Others discover their inner-entrepre- Beyond the numbers, these lead- jectives of the company.
lives of people or communities, there The human resources biotechnology neurship tendencies after years of ers have to possess many capabilities: Communication management: en-
has been no progress. As true as that demands fall into four broad categories: commercial responsibilities that build capsulate the mission and methods
©KEITH NEGLEY
rings, there is at the very least a code- their confidence and hone their skills. Leadership: formulate human re- of the company into a succinct and
pendence of the underlying technol- Scientific and technical. source needs and manage the hiring persuasive message in order to attract
ogy and the people who turn it into This includes the Ph.D.s and other Entrepreneurial enablers. and development of teams. human resources, build internal con-
reality. Some might argue—myself graduates in the life and medical The entrepreneurial academic litera- Strategic thinking: make choices sensus, drive strategy and operations
included—that the biotechnology in- sciences, whether they be recently ture implies, but seldom studies, this based on alternatives and options. in an organized fashion, assure stake-
thorities make the procedure avail- Schwitzer and a team of reviewers nies have even co-opted the media to responsibility—from the media con-
able in Canada. The media coverage looked at about 1,900 health-related convince people that they are sick and sumers hungry for miraculous cures
snowballed, prompting an unprec- stories about drugs, medical devices in need of medical treatment. to the companies seeking profits, and
edented amount of political involve- or other interventions published by Such was the case with restless everyone in between. As Sumner says,
ment in the allocation of research U.S. news organizations between 2006 legs syndrome. Lisa Schwartz and “All of us are not watchful enough.”
COMMUNICATION
broader interpretations of risk, such tobacco in 1950s and ’60s, such as cre- exercise in understanding respect the communication process will need
as psychological and social factors. ating fake online grassroots groups (based on shared values and inter- to be assimilated into the process.
D
eman and his late colleague Amos Experts, including regulators, view such as propaganda and third-party not aimed at acceptance, but aimed
espite decades of global- The evolution of how people deal Tversky showed that decision making risk as measurable hazard, the product at putting people in the best possible
ly expanding use of ge- with risk is a popular social concept— about risk is neither purely rational of probability and magnitude. People position to make a decision consistent
netically modified (GM) dubbed the “risk society” by German nor purely “irrational.” Still, many facing the risks, however, tend to focus with their values and beliefs.
crops, broad public ac- sociologist Ulrich Beck—that has biotechnology organizations employ more on what concerns them, rather Science communicators should
ceptance still eludes this resulted in an environment today a strategic and tactical mandate pred- than the “technical” hazard itself. continually monitor social-science re-
technology. The history where new technologies are scruti- icated on only “rational” thinking. Peter Sandman, one of risk communi- search to gain further insights into the
of the dialogue behind these crops, nized more, and GM crops have been Some experts know that informa- cation’s pioneering practitioners, calls groups with which we are communi-
however, reveals what we can learn embroiled in controversy from their tion cannot be the sole source of com- this concern “outrage,” and it’s a pow- cating. As a result, we will learn to un-
from communications science. introduction. Such controversial sub- munication about GM crops to re- erful force that can escalate if not ad- derstand existing and new concerns—
Over the past 20 to 30 years, the ject matter requires specific handling ally get across the point. Recently, Paul dressed. Moreover, outrage might even not just about GMOs, but also about
world has changed considerably, with from a communications standpoint. Teng, chairman of the International increase in the face of sound technical all areas of innovation. When there are
populations becoming aware of the The ongoing controversy shows how Service for the Acquisition of Agri- data, because the higher concerns are perceptions of risk, we should use the
risks in their everyday lives. Conse- this has largely failed. biotech Applications (ISAAA), said: not addressed and these facts may con- science of communication in the com-
quently, people in general have grown “Communication is key for GM prod- flict with deeply held cultural views munication of science. If we do, some
more averse to risk. With health scares Following Failed Philosophies uct acceptance, but just sharing infor- and commitments. of the glaring problems with GMO
in food and agriculture, like mad-cow In many cases, efforts to communi- mation alone is not sufficient. Infusing Industry often shuns this higher communication might be addressed.
©ALEX NABAUM
disease in the United Kingdom and cate about GM crops have followed a values into communication messages level of the risk debate. Instead, indus-
melamine in infant formula in China, linear, educational, one-way delivery, will contribute greatly to making GM try tends to reframe the issues in terms Andrew D. Roberts and Andrew D. Powell are,
people around the world have started focused mainly on technical elements products part of everyday life.” of technical expertise and experience, respectively, COO and CEO of Asia BioBusi-
ness Pte Ltd. in Singapore and cofounders of
losing trust in the people and organi- of the science. Likewise, these efforts Still, some groups try to get across where it is undoubtedly most comfort- the Centre for Risk Communication Asia.
zations that regulate the risks. failed to discuss, or even acknowledge, those messages with techniques that able. Simply put, facts about the risk
First Came Access THE MANY AUDIENCES AND VALUES OF CURE FORWARD:
T
able for patients. The proprietary process in Cure Forward software, But it can take 2–3 years to fill a
he irony of our new therapy or clinical trial that platform will enable patients to: patients will be able to connect with targeted trial, and more than half
modern era, where might offer hope could be unseen, » Learn about diagnostic testing and their HCPs and cooperate on trial of trial sites fail to accrue a single
precision medicine, unavailable or simply unknowable is clinical trials. This information will matching. HCPs will also be able to patient! 85% of patients don’t know
molecular diagnostics, a source of frustration and some- help them in dialogues with their view the Cure Forward test directory that clinical trials are an option to
targeted therapies and times hopelessness. doctor about ordering tumor-profil- to find tests that might be useful for consider, yet 75% say they would
other major medical We all know too well, whether ing and other tests that can provide their patients. seriously consider a trial if it were
innovations on the horizon are help- personally or through family better outcomes. available. Patients in the community
ing to individualize and personalize members, the anguish of receiv- » Electronically retrieve their test For Diagnostic Test Providers – setting are simply not visible to trial
treatments while revolutionizing en- ing a difficult diagnosis of a deadly results from diagnostic labs into Because patients, their families, sponsors until they apply for entry,
tire categories of disease, is that all disease. Even in an era of boundless their Cure Forward account. If their and their physicians will use Cure and too often those applications
of these tools and solution providers health information, digital connec- lab doesn’t make results available Forward to find tests that may be never come. Cure Forward offers a
are not working as effectively and tivity and modern diagnostic tools, on the network, users will be able to relevant to their care, Cure Forward mechanism for patients to instan-
efficiently as they could. The stake- patients and their loved ones can and offer a sum much greater than enter results manually or send their is a potent marketing channel for di- taneously make themselves visible
holder least able to take advantage feel powerless against the forces of its parts, combating disease, creating report to Cure Forward for manual agnostic laboratory partners (DLPs). to all recruiting trials that might suit
of the progress of precision medicine their condition. Questions inevitably efficiencies and opportunities, and transcription. Cure Forward DLPs will have the them. Trial sponsors receive notifica-
is the patient. While patients may arise: “Am I doing EVERYTHING that reducing that ominous feeling in the » Read Gene Stories, magazine distinction of being patient-friendly tions when relevant patients post,
be informed, even empowered with can be done to combat my illness? pit of patients’ or their family mem- articles written by journalists to tell in their stance about patient access and can then invite those patients to
medical information, they do not Do I truly understand the molecular bers’ stomachs. It is truly the first the story of each gene and describe and use of their data. apply through the platform. Patients
have any way to act upon the data, diagnosis and am I able to have a Patient Activation Company. its importance in cancer care. The Patients inevitably have questions choose from their available invita-
leveraging every possible avenue thorough discussion with my doctor? In the same way multi-sided stories are designed to make ge- about their genomic test results. tions, and Cure Forward makes the
technology platforms proactively nomic findings understandable and These are complex tests, often with introductions.
Cure Forward puts patients in the driver’s seat and gives connect relevant stakeholders to memorable to ordinary people. many findings that are not read- By applying a marketplace solu-
them the ability to take advantage of every option of care produce value for all parties, much » Post their information to a clinical ily understood. Cure Forward can tion to a scientific problem, Cure
available to them, activating ALL parts of the health ecosystem. like Airbnb or Kickstarter, Cure trial exchange, a marketplace where answer many of the basic questions Forward aims to increase patient
Forward will enable patients, with trial sponsors can see them for the that otherwise might be handled by participation in clinical research, to
toward the best care options pos- Are there clinical trials available as complete anonymity and privacy, duration of their post and then invite the testing laboratory. Consumer- help individuals gain access to new
sible for their very specific diagnosis an option for me to explore? How do to access their molecular data (a them to apply to their trials. This is level customer support can be time therapies and to help those new
and minimizing the chance of any I even do that?” This questioning and tumor profile), understand it through the opposite of blind searches on the consuming, and partnering with innovations get to market sooner for
potential treatment slipping through trying to find relevant information educational tools, connect with pa- Internet. Cure Forward will help a laboratory everyone else.
the cracks. quickly can, in short order, become tients across the world with similar » Interact with other patients on the focus its time and resources toward No money will ever change hands
The ultimate goal of our medical daunting, time consuming and ulti- conditions, and give the patients the platform. Patients will be matched its main objectives of providing high unless a trial recruiter has already
ecosystem is to identify the causes mately frustrating. ability to seek out clinical trials in a to “tribes” who resemble them on quality testing and interpretation. experienced success recruiting
of disease and proceed with the simple way without having to have molecular, clinical, personal, and A recent U.S. Health and Human through Cure Forward. That means
most effective treatment as effi- ENTER CURE FORWARD, an advanced degree to understand demographic factors, or set up their Services rule stipulates that patients fast, broad, precise, low-cost recruit-
ciently and timely as possible. While A PATIENT ACTIVATION the complexities of the trial require- own tribes. Within tribes, people will can access their diagnostic test re- ing, and pay-for-performance eco-
the last two decades have seen the COMPANY. Cure Forward is lead- ments. A patient does not have to be able to ask and answer questions sults directly from the test provider. nomics. This is a new way to accrue
rise in more available sources of ing the way towards innovative health trawl endless trial descriptions or or share relevant information. This rule, while progressive and clinical studies.
information through web hubs, new solutions that put patients in the fear they won’t connect to all of potentially beneficial to the patient,
apps and other platforms, the task of driver’s seat and gives them the abil- the information available. Innova- For Health Care Providers – can result in extra work for labora-
assessing large amounts of informa- ity to take advantage of every option tive new therapeutics can quickly As personalized and “precision” tories, who may not have systems in Learn More -
tion and acting intelligently on it can of care available to them, activating find their beneficiaries through an medicine approaches become in- place for constructive delivery of test To learn what a Patient Activation
be daunting for patients, as well as ALL parts of the health ecosystem. activated patient network. And com- creasingly available and relevant in results. Cure Forward can be a con- Company is all about, and to see
other stakeholders in the system. Leveraging the power of sci- munities, created around specific managing genetic-related diseases structive channel for results delivery, how innovation can better serve
The fear that one is wasting time ence and the power of community, disease states, molecular profiles like cancer, health care providers without added cost or time commit- every part of the medical ecosystem,
while the disease progresses brings Cure Forward seeks to provide and other criteria, will be valuable (HCPs) are faced with the chal- ment from the DLP. Cure Forward please visit:
that gnawing feeling in the pit of the a 360-degree approach for the sources of support, information and lenge of effectively describing test does not charge its DLPs for this
stomach. The worry that a brand entirety of the medical ecosystem, timely input. results to patients. This is especially marketing presence and constructive www.cureforward.com
difficult in a short office visit. The results delivery. Beta release begins in June 2015
SCIENTIFIC AMERICAN
WORLDVIEW SCORECARD
Our Scorecard analyzes and interprets data on
the innovation potential in biotechnology for 54
countries. Based on seven Scorecard Categories
POWER
—created from 27 components—this analysis
combines information from biotechnology inputs
and outputs through government protection and
policies and far beyond. The results reveal the
POINTS
dramatic range of capabilities in this dynamic
field, and which countries are moving ahead and
which ones are falling behind.
To get the most from this analysis, work your
way through the introduction and Scorecard
Categories on your way to the Overall Scores Which places around the globe
ahead. Beyond that, finer-grain analyses add even
more depth to our understanding of the innovation
are powering the success of
potential in biotechnology around the world. biotech today?
Start exploring!
2015
grow and thrive. ponents. (For detailed methods, see page 46.)
.74
The 2015 Scorecard also examines the drivers of global Our analysis of gross and relative metrics provides a .58
investment. After so many years of reporting slow declines rich assessment of the biotechnology sector worldwide. .50
in the financial position of biotech firms around the world, The results reveal a number of surprising shifts in the in- .31 .30 .30 .27 .21
.14 .13 .10 .10
data from our last Scorecard presented a clear indication of dustry, as well as several unexpected players exceling in .07 .06 .06 .05 .04 .02 .02 .02 .02
economic recovery. This year, we see even more evidence certain areas. As the biotech field continues to evolve, so
of bounce back, especially in one particular country. do our methods for tracking those places around the globe
UNITED STATES
AUSTRALIA
UNITED KINGDOM
FRANCE
CANADA
GERMANY
DENMARK
SWEDEN
SWITZERLAND
ISRAEL
BELGIUM
NETHERLANDS
IRELAND
NORWAY
CHINA
ITALY
JAPAN
HONG KONG
INDONESIA
AUSTRIA
FINLAND
POLAND
TAIWAN, CHINA
As in the past, we built the Scorecard using a diverse where today’s innovators have the best chance of bringing
collection of metrics—from education and political tomorrow’s innovations to the fore. —THE EDITORS
BRAZIL 2.87 IRELAND 2.85 SWEDEN 2.62 BELGIUM 2.44 UNITED KINGDOM 2.38 MEXICO 2.27 CANADA 2.21
RUSSIA 2.14 FRANCE 2.04 NORWAY 1.92 NETHERLANDS 1.91 S. KOREA 1.70 HONG KONG 1.67 S. AFRICA 1.30
POLAND 1.14 GERMANY 1.07 AUSTRIA 1.05 INDIA 1.01 JAPAN 0.91 CHINA 0.90 ITALY 0.78
SINGAPORE 0.69 FINLAND 0.64 TAIWAN, CHINA 0.59 INDONESIA 0.16 ARGENTINA 0.13 TURKEY 0.13 THAILAND 0.08
CHILE 0.07 UAE 0.07 GREECE 0.06 MALAYSIA 0.06 PHILIPPINES 0.06 PORTUGAL 0.06 SAUDI ARABIA 0.05
CZECH REP. 0.04 UKRAINE 0.03 HUNGARY 0.02 KUWAIT 0.01 QATAR 0.01
9.53
9.47
9.26
9.21
8.87
8.87
8.84
8.82
8.74
8.74
8.50
8.49
8.34
8.34
8.31
8.20
8.15
8.09
7.84
7.78
7.61
7.48
7.40
7.06
6.76
6.57
6.24
6.20
5.98
5.85
5.73
5.66
5.62
5.53
5.47
5.34
5.33
5.30
5.20
5.13
5.07
4.86
4.75
4.21
4.19
4.19
4.01
3.27
3.08
2.97
2.89
2.69
2.48
0.92
FINLAND
QATAR
JAPAN
PUERTO RICO
CANADA
NETHERLANDS
UNITED KINGDOM
UNITED STATES
FRANCE
IRELAND
SWITZERLAND
SINGAPORE
BELGIUM
DENMARK
SWEDEN
LUXEMBOURG
UAE
GERMANY
AUSTRIA
NEW ZEALAND
NORWAY
AUSTRALIA
SOUTH AFRICA
HONG KONG
PORTUGAL
ESTONIA
ITALY
ISRAEL
TAIWAN, CHINA
HUNGARY
CZECH REPUBLIC
GREECE
CHILE
MALAYSIA
SOUTH KOREA
SPAIN
SLOVAK REPUBLIC
CHINA
POLAND
PHILIPPINES
ICELAND
LITHUANIA
TURKEY
LATVIA
INDIA
MEXICO
SAUDI ARABIA
BRAZIL
RUSSIA
ARGENTINA
KUWAIT
UKRAINE
INDONESIA
THAILAND
PUBLIC COMPANIES / MILLION POPULATION
PUBLIC COMPANY EMPLOYEES / CAPITA
SCORECARD CATEGORY #3: PUBLIC COMPANY REVENUES / US$B GDP
INTENSITY BIOTECH PATENTS /TOTAL PATENTS FILED WITH PCT
Top marks for Denmark in VALUE ADDED OF KNOWLEDGE- AND TECHNOLOGY-INTENSIVE INDUSTRIES
driving innovation
medicines for domestic needs. For of protection, enforcement mecha- We measure a country’s overall tion (Lawrence, S. & Lähteenmäki, U.S. National Science Foundation’s
SCORECARD CATEGORY#2: R. Nat. Biotechnol. 32, 626–632
example, a company may be inclined nisms and restrictions (e.g., compul- efforts to boost biotech innovation Science and Engineering Indicators
IP PROTECTION to abandon a drug lead if it cannot sory licensing). Recognizing that per- by combining five characteristics (2014)). For instance, dividing each was the source for “Value added of
Objective and perceived measure- ceptions also influence investment into a measurement designated country’s employee counts by its knowledge- and technology-inten-
find profit-enabling markets for it.
ments can diverge considerably decisions, and that the perception of as Intensity. These characteristics population, as sourced from the sive industries.”
In prior issues of Scientific American
“Intellectual property is the invisible Worldview, we have also shown that a country’s IP protection might not are normalized for population size U.S. Census Bureau International Last year, Denmark held its
infrastructure of innovation,” writes the strength of a country’s IP protec- match objective measures, we ac- and overall economy. As a result, Database, produces the “public long lead in this category, followed
Michael A. Gollin in Driving Innovation: tion correlates with its concentra- count for subjectivity with Schwab’s Intensity helps identify smaller biotechnology company employees by the United States, Australia
Intellectual Property Strategies for a tion of clinical trials. Accordingly, IP “perceived IP protection” metric nations that exhibit outsized activity per capita” metric. Likewise, the and Singapore. This year the same
Dynamic World (Cambridge University protection can impact whether a na- (Schwab, K. The Global Competi- in the sector. Whereas Productivity measurement of “public biotech- three countries took the top three
Press, 2008). Nowhere is that state- tion’s scientists and physicians even tiveness Report, 2014–2015. World measures outputs, like commercial nology company revenues per spots. Spain advanced to fourth
ment more accurate than in the field play a role in global drug develop- Economic Forum (2014)). This index products, Intensity measures GDP” is generated by dividing a place, largely due to a surge in the
of biosciences. The capital and time ment, and if drugs will be developed was created using feedback from inputs—the elements that propel nation’s Productivity score by its proportion of biotech patent appli-
invested in a new drug can only be for locally pervasive conditions. a global group of business leaders companies to success. Those look- GDP, as sourced from the IMF cations filed there.
recouped through strong IP Protec- We measure IP protection both when asked about their perceptions ing for research partners or a place World Economic Outlook Database.
tion. Without that assurance, it is objectively and subjectively. “IP of domestic IP protection. to develop biotechnology should Data for both “biotech patents per
difficult to attract funding or convince strength” is drawn from a study Although the United States consider countries with high Inten- total patents” (filed with the Patent
ambitious scientists and business (Park, W.G. Research Policy 37, comes in first in the objective mea- sity scores. Cooperation Treaty) and “business
founders to take on the risks of pio- 761–766 (2008)) that calculates the surement, “IP strength,” Finland, The data used in the components expenditures on biotechnology
neering research and development. unweighted sum of five measures: Qatar and Japan take the lead when of Intensity are gathered from vari- R&D” came from the Organisa-
In addition, IP protection can patentable inventions, membership the “perceived IP protection” metric ous sources, including company tion for Economic Co-operation
encourage foreign development of in international treaties, duration is added to the calculation. disclosures and published informa- and Development (OECD). And the
SWITZERLAND 3.40
SOUTH KOREA 3.71
SAUDI ARABIA 4.33
LUXEMBOURG 5.29
SINGAPORE 4.13
AUSTRALIA 4.97
PORTUGAL 3.20
THAILAND 3.90
DENMARK 3.79
GERMANY 4.20
FINLAND 3.71
IRELAND 4.22
ICELAND 4.14
SWEDEN 3.10
AUSTRIA 3.36
CANADA 4.34
POLAND 2.85
FRANCE 4.25
ISRAEL 4.28
JAPAN 3.46
2.82 ESTONIA
2.78 SPAIN
2.71BELGIUM
2.65 ITALY
2.65 NORWAY
2.61 RUSSIA
2.48 NETHERLANDS
2.10 LITHUANIA
2.06 ARGENTINA
2.02 MALAYSIA
1.94 GREECE
1.85 CHILE
1.85 TURKEY
1.82 UKRAINE
1.81 INDONESIA
1.70 HUNGARY
1.61 BRAZIL
1.53 LATVIA
1.39 MEXICO
1.22 PHILIPPINES
1.06 CHINA
0.72 UAE
0.23 INDIA
0.11 KUWAIT
0.04 QATAR
EDUCATION/WORKFORCE
POST-SECONDARY SCIENCE GRADUATES / CAPITA
PHD GRADUATES IN LIFE SCIENCES PER MILLION POPULATION
R&D PERSONNEL PER THOUSAND EMPLOYMENT
TALENT RETENTION (reciprocal of brain drain)
BRAIN GAIN (share of global students studying outside their country)
ENTERPRISE SUPPORT
BUSINESS-FRIENDLY ENVIRONMENT (higher = better)
BIOTECH VC, 2007 (US$MM)
VC AVAILABILITY
CAPITAL AVAILABILITY
NETHERLANDS 5.06
SWITZERLAND 5.33
LUXEMBOURG 3.99
PUERTO RICO 4.22
PHILIPPINES 3.09
HONG KONG 8.93
SINGAPORE 9.20
AUSTRALIA 5.43
ARGENTINA 0.40
INDONESIA 3.46
LITHUANIA 4.77
PORTUGAL 3.92
THAILAND 5.44
DENMARK 4.89
MALAYSIA 7.97
GERMANY 5.08
HUNGARY 2.48
BELGIUM 4.26
FINLAND 6.12
IRELAND 4.83
ICELAND 5.50
UKRAINE 1.29
AUSTRIA 3.97
SWEDEN 6.18
NORWAY 6.06
ESTONIA 6.52
CANADA 6.07
POLAND 2.55
FRANCE 4.72
GREECE 2.06
TURKEY 3.14
KUWAIT 3.71
MEXICO 3.77
RUSSIA 3.12
BRAZIL 2.30
ISRAEL 6.49
LATVIA 4.63
JAPAN 4.53
QATAR 7.33
CHINA 4.83
SPAIN 3.50
CHILE 5.27
INDIA 3.24
ITALY 2.46
UAE 7.34
age of a category’s component of a nation’s rough performance The table’s color-coding helps
(A) PUBLIC COMPANY
REVENUES (US$MM)
(K) BUSINESS-FRIENDLY
ENVIRONMENT
(T) BUSINESS
EXPENDITURES
2015 SCIENTIFIC scores (e.g., “IP strength” and in each category and the oppor- to reveal the weaknesses of some
(B) PUBLIC COMPANIES (HIGHER = BETTER) ON R&D (% of GDP) AMERICAN WORLDVIEW “perceived IP protection”) provides tunity to compare the numbers high finishers and the strengths of
(C) PATENT STRENGTH (L) BIOTECH VC, 2007 (U) GOVERNMENT OVERALL SCORES the category score. A simple sum of when desired. Because the score is certain countries with lower overall
(D) PERCEIVED IP
(US$MM) SUPPORT OF R&D A growing lead for the United the category scores generates the composed of averages of available scores. For example, fifth-place
(M) VC AVAILABILITY (% of GDP)
PROTECTION
(V) INFRASTRUCTURE
States, with fierce competition overall innovation score. We use data, any gaps in the data, indi- Singapore performs poorly in the
(N) CAPITAL AVAILABILITY
(E) PUBLIC COMPANIES/ QUALITY (roads, ports, among the followers normalization techniques that give cated as blanks in the table, do not Intensity category. Conversely,
MILLION POPULATION (O) POST-SECONDARY electricity, etc.)
(F) PUBLIC COMPANY SCIENCE GRADUATES/
(W) ENTREPENEURSHIP
each component and each category affect the overall scores. 40th-place Saudi Arabia ranks rela-
EMPLOYEES/ CAPITA CAPITA
& OPPORTUNITY Our 2015 Scorecard ranks the bio- equal weight. The United States remains at tively well in Education/Workplace.
(G) PUBLIC COMPANY (P) PHD GRADUATES
IN LIFE SCIENCES PER (X) POLITICAL STABILITY & technology innovation potential of This table lists all 54 countries the top, extending its lead from last As the data make clear, the
REVENUES/ $USB GDP ABSENCE OF VIOLENCE/
(H) BIOTECH PATENTS/
MILLION POPULATION 54 countries. A nation’s aggregate in top-to-bottom order based on year over the second-place country. top finishers overall tend to score
TERRORISM
TOTAL PATENTS FILED (Q) R&D PERSONNEL performance in each of the seven their overall innovation scores. It The next tier of finishers, however, respectably in each category. That
PER THOUSAND (Y) GOVERNMENT
WITH PCT EFFECTIVENESS categories—Productivity, IP Protec- also illustrates a nation’s rank in is rather tightly packed. Second- stands to reason, as a healthy bio-
EMPLOYMENT
(I) VALUE ADDED OF each category, showing the sub- place Denmark, for example, tech sector is dependent on so many
KNOWLEDGE- AND (R) TALENT RETENTION (Z) REGULATORY QUALITY tion, Enterprise Support, Intensity,
TECHNOLOGY-INTENSIVE (RECIPROCAL OF BRAIN (AA) RULE OF LAW Education/Workforce, Foundations, score for each category color-cod- scored 13% higher than 10th-place factors—from a diverse workforce
INDUSTRIES DRAIN) ed on a coarse scale in comparison Canada. And there was only a 22% skilled in both bench science and
and Policy & Stability
(J) BUSINESS EXPENDI- (S) BRAIN GAIN (share of
global students studying —yields the final score. (For the to other countries. As a result, difference in the overall scores business to support systems includ-
TURES ON BIOTECH-
NOLOGY R&D outside their country) complete Methods, see page 46.) In the table offers a quick overview between the second and 20th-place ing everything from the availability
brief, for each country the aver- of the final results, an indication holder, Luxembourg. of capital to the access to ports.
number of public companies Lawrence, S. & Lähteenmäki, R. Nat. Biotechnol. 32, 626–632 (2014), and company disclosures
IP PROTECTION
IP strength Park, W.G. Research Policy 37, 761–766 (2008)
perceived IP protection Schwab, K. The Global Competitiveness Report, 2014–2015. World Economic Forum (2014)
INTENSITY
public companies per million population Lawrence, S. & Lähteenmäki, R. Nat. Biotechnol. 32, 626–632 (2014),
company disclosures and U.S. Census Bureau International Database
public biotechnology company employees Lawrence, S. & Lähteenmäki, R. Nat. Biotechnol. 32, 626–632 (2014),
SCIENTIFIC AMERICAN per capita company disclosures and U.S. Census Bureau International Database
WORLDVIEW SCORECARD METHODOLOGY public biotechnology company revenues Lawrence, S. & Lähteenmäki, R. Nat. Biotechnol. 32, 626–632 (2014),
This Scorecard assesses 54 countries on their innovation potential in bio- per GDP company disclosures and IMF World Economic Outlook Database
technology through a meta-analysis. The analysis includes 27 components biotech patents per total patents Organisation for Economic Co-operation and Development
arranged in seven categories: Productivity, Intellectual Property (IP) Pro- business expenditures on biotechnology R&D Organisation for Economic Co-operation and Development
tection, Intensity, Enterprise Support, Education/Workforce, Foundations,
and Policy & Stability. The table shows the components of each category value added of knowledge- and technology- U.S. National Science Foundation’s Science and Engineering Indicator
intensive industries
and the sources of data.
For each component, countries are ranked on a scale from 0 to 10, with
the lowest-ranked country scored as 0 and the highest-ranked one scored ENTERPRISE SUPPORT
as 10. A nation’s score in a category is derived from the average of the
business-friendly environment Doing Business 2014 (World Bank and the International Finance Corporation)
available component scores—any gaps in the individual components were
ignored in calculating the averages for each category. biotechnology venture capital Organisation for Economic Co-operation and Development
The overall innovation score is a sum of the category averages, indexed to venture capital availability Schwab, K. The Global Competitiveness Report, 2014–2015. World Economic Forum (2014)
a score from 0 to 50. The normalization involved in calculating the category capital availability Milken Institute Capital Access Index
and overall scores considers each component and each category on equal
weighting. In short, the Scorecard gives equal importance to all components.
EDUCATION/WORKFORCE
Post-secondary science graduates per capita UNESCO and U.S. Census Bureau International Database
Ph.D. graduates in the life sciences Organisation for Economic Co-operation and Development
per capita
R&D personnel per thousand employment Organisation for Economic Co-operation and Development
FOUNDATIONS
R&D business expenditures per GDP Organisation for Economic Co-operation and Development
infrastructure quality Schwab, K. The Global Competitiveness Report, 2014–2015. World Economic Forum (2014)
political stability & absence of violence/ World Bank’s 2014 World Governance Indicators
terrorism
government effectiveness World Bank’s 2014 World Governance Indicators
AUSTRALIA
CANADA
DENMARK
FINLAND
HONG KONG
NEW ZEALAND
SINGAPORE
SWEDEN
SWITZERLAND
UNITED KINGDOM
36 UNITED STATES
32
10
28 8
1
2009 2015 2009 2015
LESS ROCKING IN card—placing second four out of 7th in 2009, then dropped to 18th
MOST LEADERS LACK in first place, its overall score 10 finishers have jumped up and THE RANKING seven years. Sweden also earned for two years, before getting back
does fluctuate, even though it was down and back over the years. For relatively steady ranks over the in the Top 10 in 2012 and staying
CONSISTENT SCORES The order of the Top 10 countries
years, always in the Top 10 and in there—moving all the way to 3rd in
Ongoing ups and downs plague nearly the same this year and last. example, Denmark’s scores for varies less than the scores
Compared to the others in the Top 2009–2015 were 31.6, 27.3, 31.9, the Top Five six of our seven years. 2015. Hong Kong’s ranking leaps
many countries at the top
10, the United States earned fairly 37.2, 35.7, 29.7 and 29.8. New Zea- Likewise, Singapore has secured around as well: 17th in 2011, 13th
steady scores for 2009–2015: 37.1, land’s scores move all over as well In comparison to the overall a Top 10 ranking throughout the in 2012, then 20th, 12th and 11th in
Some of the fiercest competition 36.6, 39.0, 38.0, 37.6, 39.6 and 39.6. in that time: 30.0, 23.3, 24.9, 32.7, scores, the ranking at the top looks Scorecard’s history, never dropping 2013, 2014 and 2015, respectively.
on the Scorecard is seen among That’s a seven-year average of 38.2, 28.6, 27.5 and 28.1. relatively stable. It starts with the lower than 8th place, and otherwise Like any industry, biotechnol-
the Top 10 countries. This graphic with the annual scores remaining So far, our growing database United States earning a steady always reaching the Top Five. ogy demands ongoing efforts to
shows the volatility of their fin- fairly close to that number. does not show any conclusive first place across all years. Next, Not every top-placing country, maintain a nation’s standing on the
ishes by overall scores. Although Going down the list, however, trends in scores at the top for any Denmark grabs the second-highest however, was so constant. New world stage.
the United States is consistently we see that all of the other Top country except the United States. ranking over the life of the Score- Zealand, for example, started in
UNITED KINGDOM
grabs two
golds 3rd PLACE
FINLAND
AUSTRALIA
2nd PLACE
1st PLACE
SINGAPORE
HONG KONG
NEW ZEALAND
SOUTH KOREA
SWEDEN
ISRAEL
NETHERLANDS
SAUDI ARABIA
LUXEMBOURG
FRANCE
CANADA
ARGENTINA
THAILAND
GERMANY
UKRAINE
BELGIUM
NORWAY
BRAZIL
some categories, such as relative
JAPAN
QATAR
CHINA
CHILE
INDIA
LARGE AND SMALL ones, size doesn’t even matter. For
WINNERS, AND LOSERS instance, Luxembourg—smaller
Countries Countries of all sizes can be giants than Rhode Island, and with a
around the in their category population of only about half a mil-
world excel lion—won “most R&D personnel
Freeman Dyson, now retired from per total employment,” designat- GREATEST PUBLIC GREATEST PERCEIVED BEST TALENT RETENTION STRONGEST PERCEIVED
his work as a physicist at the Insti- ing it as a highly educated, albeit COMPANY REVENUES: PATENT STRENGTH: (most U.S.-trained doctorate ENTREPRENEURSHIP
undersized, nation. United States, Australia, Finland/Singapore graduates intending to return CLIMATE: Sweden,
tute for Advanced Study in Prince-
Ranking first, though, is not al- United Kingdom home): Saudi Arabia, Denmark, Switzerland
ton, New Jersey, once said, “I see a GREATEST PERCENTAGE Thailand, Chile
bright future for the biotechnology ways desirable. India, China and the MOST PUBLIC OF PATENTS IN BEST POLITICAL
industry when it follows the path Ukraine, for example, would surely COMPANIES: United BIOTECHNOLOGY: WORST BRAIN DRAIN STABILITY: New Zealand,
of the computer industry, the path prefer not to win the “worst brain States, Australia, Canada Denmark, Singapore, (most U.S.-trained doctor- Switzerland, Finland
that von Neumann failed to foresee, drain” category, which means that Belgium ate graduates intending to
becoming small and domesticated they lead the world in the propor- GREATEST PUBLIC stay in the United States): GREATEST GOVERNMENT
rather than big and centralized.” tion of their students who would COMPANY MARKET GREATEST ENTERPRISE India, China, Ukraine EFFECTIVENESS: Finland,
rather not return home after their CAPITALIZATION: SUPPORT: Singapore, Singapore, Denmark
Maybe it’s a little early in the evolu-
Ph.D. studies abroad. United States, Australia, United States, Hong Kong BEST BRAIN GAIN
tion of biotechnology to call it small United Kingdom (share of global graduate GREATEST REGULATORY
and domesticated. Thus far, many This map illustrates the state GREATEST VENTURE QUALITY: Singapore,
students): United States,
parts of the industry remain big and of biotechnology today, both in the MOST PUBLIC COMPANY CAPITAL AVAILABILITY: United Kingdom, France Hong Kong, Sweden
centralized. Nonetheless, the lead- tools that it requires and the plac- EMPLOYEES: United Qatar, United States/United
ers in the wide range of categories es where it excels. Perhaps as time States, Australia, France Arab Emirates GREATEST BUSINESS BEST RULE OF LAW:
shown here hail from all around the goes on, a map like this will look EXPENDITURES ON Norway, Sweden, Finland
globe, and they vary dramatically more and more like Dyson’s vision GREATEST REVENUE MOST PH.D. GRADUATES R&D (% of GDP): South
of “small and domesticated.” PER PUBLIC COMPANY: IN LIFE SCIENCES PER Korea, Israel, Japan LARGEST PUBLIC
in size. MARKETS FOR BIOTECH-
Netherlands, United CAPITA: New Zealand,
Small countries can be big States, Denmark Canada/United Kingdom GOVERNMENT SUPPORT NOLOGY: United States,
players within a certain category. OF R&D (% of GDP): South Australia, United Kingdom
Take Qatar, for example. Not quite GREATEST REVENUE MOST PH.D. GRADUATES Korea, Israel, Finland
the size of the state of Connecticut, PER PUBLIC COMPANY IN THE LIFE SCIENCES: BEST GROWTH IN
it is home to just a little over 2 mil- EMPLOYEE: Finland, United States, United BEST INFRASTRUCTURE BIOTECHNOLOGY PUBLIC
lion people, and its GDP is be- United Kingdom, Swit- Kingdom, Germany QUALITY: Switzerland, MARKETS: United States,
tween the Gross State Products of zerland/United States Hong Kong, United Arab Australia
MOST R&D PERSONNEL Emirates/Finland
Hawaii and New Mexico. Even so, it TOP BIOTECHNOLOGY
STRONGEST MEASURED PER TOTAL EMPLOY-
takes the gold medal in “greatest PATENT PROTECTION: MENT: Luxembourg, CROP PLANTINGS: United
venture capital availability.” And in United States Israel, Finland States, Brazil, Argentina
UNITED STATES
The U.S. sees an increase in The United States takes its The U.S. industry’s value
public biotechs biggest lead yet in public- is higher than ever
800,000
company market capitalization
Information from public compa- 35,000
nies—those whose shares are One of the most telling metrics
traded on public stock exchanges— we use to chart the global biotech 600,000
provides a strong objective mea- landscape is public-company mar- 250
surement of a country’s economic ket capitalization, or the market
performance. Because they are value of a business’s outstanding 400,000
publicly traded, public companies shares. When considered in com-
have greater transparency than bination with the number of public 30,000
private firms, and offer a clearer companies in each country, this UNITED STATES
picture of a nation’s business land- EX-US 200,000
information provides a broad inter-
scape. We collect this information national comparison of the industry.
from a published study (Lawrence, Our data on market capitalization
S. & Lähteenmäki, R. Nat. Biotechnol. was gathered from a published
32, 626–632 (2014)) and company 200
study (Lawrence, S., Lähteenmäki,
disclosures. R. Nat. Biotechnol. 32, 626–632 PUBLIC COMPANY MARKET CAPITALIZATION
For most of Scientific American (2014)) and company disclosures. (US$ MM) 25,000
Worldview’s history, the number of The United States has histori-
AUSTRALIA
public companies has been falling cally led the world in biotech market AUSTRIA
around the world. As we’ve noted capitalization, and continues to do BELGIUM
in the past, a decrease in public CANADA
so this year. But what’s striking is CHINA
businesses should be considered how much that lead has increased. A DENMARK
in the context of other factors, such year ago, the U.S. market capitaliza- FINLAND
FRANCE
as employee counts, revenues or tion was close to four times that of GERMANY 20,000
150
market capitalization. Increases the non-U.S. global total—and this HONG KONG
in these other factors as company year it ballooned to nearly six times ICELAND
INDIA
counts drop could indicate that the that total. Australia had the second- IRELAND
industry is consolidating, which can largest growth, adding nearly US$ ISRAEL
be a positive sign. ITALY
5 billion in market capitalization, or NUMBER OF PUBLIC JAPAN
This year, the tally of U.S. public roughly the sum of all of Sweden’s COMPANIES NETHERLANDS
biotechnology companies increased public biotechnology companies. (Worldview 2009–2015) NORWAY
POLAND 15,000
for the first time in the history While most of the Top 10 countries RUSSIA
of Scientific American Worldview. saw progress in this area, Ireland SWEDEN
Most other countries have seen a was the lone standout, continuing its SWITZERLAND
100 TAIWAN, CHINA
decrease in the number of public two-year drop. 2009 UK
firms, although France has demon- The strong U.S. gains in both the 2010
2011
strated a positive trend in this area number of public companies and 2012
since we started measuring in 2009. public-company market capitaliza- 2013
Understanding the complete story, 2014
tion indicate the likelihood of further 2015 10,000
however, also requires an analysis growth ahead. In other nations,
of the market capitalization. decreases in the number of firms,
paired with increases in market
UNITED KINGDOM
AUSTRALIA
GERMANY
SWITZERLAND
SWEDEN
NETHERLANDS
DENMARK
ISRAEL
TAIWAN, CHINA
BELGIUM
HONG KONG
NORWAY
IRELAND
AUSTRIA
FINLAND
ICELAND
POLAND
JAPAN
CHINA
ITALY
RUSSIA
INDIA
2009 2015
DENMARK
WHERE R&D IS DONE
A clear win for the U.S., and then 15
it’s a tighter race
R&D US$ MM
As we’ve noted in the past, countries
promote domestic biotechnology
SWITZERLAND industries for, among other things,
the financial benefits of bringing
in high-wage jobs associated with
research and development. Accord-
ingly, we take a look at where that Russia lags
R&D predominantly occurs. 10 the field in
FRANCE Using data from a published relative biotech
study (Lawrence, S. & Lähteenmäki, investing
R. Nat. Biotechnol. 32, 626–632
(2014)) and company disclosures,
we present the simple sum of R&D
expenditures in 23 nations. Here,
AUSTRALIA the United States is the world
leader—spending nearly 22 times
as much as the United Kingdom,
in second place. Then there is a 5
near-tie between Australia, France,
Switzerland and Denmark. Not
surprisingly, these counties also
rank high on market capitalization.
This indicates that for many compa-
SLOVAK REPUBLIC
UNITED KINGDOM
CZECH REPUBLIC
nies the bulk of their research and
UNITED STATES
NETHERLANDS
SOUTH AFRICA
SWITZERLAND
development is located in the same
AUSTRALIA
PORTUGAL
DENMARK
GERMANY
SLOVENIA
BELGIUM
FINLAND
IRELAND
SWEDEN
country as their headquarters.
AUSTRIA
ESTONIA
NORWAY
CANADA
POLAND
FRANCE
MEXICO
RUSSIA
ISRAEL
KOREA
JAPAN
SPAIN
ITALY
US
BELGIUM
CANADA
CZECH REPUBLIC
DENMARK
ESTONIA
FINLAND
FRANCE
GERMANY
IRELAND
ISRAEL
ITALY
KOREA
MEXICO
NETHERLANDS
NORWAY
POLAND
PORTUGAL
SLOVAK REPUBLIC
SLOVENIA
SOUTH AFRICA
SPAIN
SWEDEN
SWITZERLAND
UNITED STATES
AUSTRALIA
AUSTRIA
BELGIUM
CANADA
ESTONIA
GERMANY
ISRAEL
ITALY
KOREA
MEXICO
NEW ZEALAND
POLAND
PORTUGAL
SLOVENIA
SOUTH AFRICA
SPAIN
UNITED KINGDOM
PERCENTAGE OF DEDICATED
80
BIOTECHNOLOGY FIRMS
BY APPLICATION,
PERCENTAGE OF BIOTECHNOLOGY (latest available year)
R&D EXPENDITURE PERFORMED
BY SMALL BIOTECHNOLOGY R&D FIRMS
40
20
THE SPENDING These data consist of public Of all of the countries on this THE SPENDING categories: health, agriculture, food rate—only about 13%—went to
SPREAD BY SIZE and private companies, mak- list, Israel was the only one in SPREAD BY and beverages, natural resources, industrial processing.
ing this analysis more inclusive which small companies invested environment, industrial processing, In some nations, biotechnol-
Big businesses outspend small
than the public company figures more in biotechnology R&D than
APPLICATION and bioinformatics. ogy spending is spread a bit more
ones in biotech R&D While biotechnology encom-
presented earlier. For the majority medium and large ones. There, In most countries there is equally.For instance, New Zealand
passes many sectors, the most
Is Dyson’s vision about the future of of the countries considered here, small companies supplied 67.1% of significantly more R&D spending dedicated about 31% of its R&D
investment is in healthcare
biotechnology—that it may be head- large businesses accounted for the spending. on health applications than on expenditures to health applications,
ing toward an industry composed of most of the spending on biotech Clearly, the numbers suggest In addition to knowing the size of the any other category. The second and roughly 20% each to food and
small companies—taking shape? R&D. That was especially true in that it will take some time before companies engaged in biotech R&D highest amounts spent were on beverages, agriculture and indus-
Our data may hold the answer. Us- Denmark, the Netherlands and the biotech industry reaches Dy- spending, understanding the indus- industrial processing and agri- trial processing.
ing the OECD’s “Key Biotechnology Switzerland, where large compa- son’s predicted point. try also requires an assessment of culture. In the majority of cases, Somewhat surprisingly, bioin-
Indicators” for October 2014, we nies provided 94.6%, 92.1% and where the money goes. To find out, however, these areas received far formatics receives very little of the
collected information for 25 coun- 90.9% of biotech R&D expendi- we turned once more to the OECD’s less funding than healthcare. For funding. In a field that is already
tries on the percentage of biotech tures, respectively. The United “Key Biotechnology Indicators” for example, in the United Kingdom very data-focused, one might
R&D spent by small firms (meaning States and South Korea were not October 2014, where we gathered 84% of R&D investment was on expect more support of this area.
they have less than 50 employees) far behind, with large firms mak- data for 18 nations. Spending on bio- health applications. The United It will be interesting to see if that
versus medium and large ones. ing up 88.4% and 87.7%. technology R&D is divided into seven Kingdom’s next highest spending trend changes over time.
SLOVAK REPUBLIC
UNITED KINGDOM
CZECH REPUBLIC
3
UNITED STATES
TAIWAN, CHINA
NETHERLANDS
NEW ZEALAND
SOUTH AFRICA
SWITZERLAND
SOUTH KOREA
SAUDI ARABIA
LUXEMBOURG
PUERTO RICO
PHILIPPINES
GUATEMALA
HONG KONG
ARGENTINA
COSTA RICA
SINGAPORE
BOTSWANA
AUSTRALIA
INDONESIA
BARBADOS
LITHUANIA
PORTUGAL
2
THAILAND
DENMARK
BULGARIA
MOROCCO
MALAYSIA
GERMANY
HUNGARY
URUGUAY
ROMANIA
LESOTHO
BAHRAIN
BELGIUM
UKRAINE
FINLAND
IRELAND
ICELAND
JAMAICA
SWEDEN
NAMIBIA
AUSTRIA
ESTONIA
NORWAY
PANAMA
CROATIA
UGANDA
CANADA
POLAND
NIGERIA
FRANCE
GREECE
TURKEY
KUWAIT
ZAMBIA
MEXICO
SERBIA
RUSSIA
BRAZIL
ISRAEL
GABON
LATVIA
JAPAN
CHINA
SPAIN
CHILE
1
INDIA
PERU
ITALY
UAE
7
BUSINESS IMPACT OF RULES ON FDI
6
SLOVAK REPUBLIC
UNITED KINGDOM
CZECH REPUBLIC
UNITED STATES
TAIWAN, CHINA
NETHERLANDS
NEW ZEALAND
SOUTH AFRICA
SWITZERLAND
SOUTH KOREA
SAUDI ARABIA
LUXEMBOURG
MADAGASCAR
MOZAMBIQUE
TIMOR-LESTE
PUERTO RICO
SEYCHELLES
PHILIPPINES
GUATEMALA
HONG KONG
NICARAGUA
ARGENTINA
SINGAPORE
CAMEROON
TAJIKISTAN
AUSTRALIA
MAURITIUS
2
INDONESIA
BARBADOS
ZIMBABWE
LITHUANIA
PORTUGAL
MONGOLIA
COLOMBIA
THAILAND
DENMARK
MOROCCO
MALAYSIA
GERMANY
URUGUAY
ROMANIA
SENEGAL
ETHIOPIA
BAHRAIN
BELGIUM
UKRAINE
FINLAND
VIETNAM
IRELAND
RWANDA
ALBANIA
GEORGIA
ICELAND
JAMAICA
SWEDEN
NAMIBIA
AUSTRIA
ESTONIA
NORWAY
PANAMA
CROATIA
UGANDA
BHUTAN
CANADA
POLAND
ANGOLA
FRANCE
GUYANA
MALAWI
GREECE
CYPRUS
TURKEY
KUWAIT
BOLIVIA
ZAMBIA
MEXICO
RUSSIA
BRAZIL
ISRAEL
GHANA
GABON
YEMEN
LATVIA
KENYA
MALTA
JAPAN
QATAR
EGYPT
CHINA
SPAIN
OMAN
INDIA
PERU
ITALY
UAE
7
NEW TECHNOLOGY BROUGHT IN BY FOREIGN INVESTMENT
6
SLOVAK REPUBLIC
UNITED KINGDOM
CZECH REPUBLIC
UNITED STATES
TAIWAN, CHINA
NETHERLANDS
NEW ZEALAND
SOUTH AFRICA
SWITZERLAND
SOUTH KOREA
SAUDI ARABIA
LUXEMBOURG
PUERTO RICO
PHILIPPINES
GUATEMALA
HONG KONG
ARGENTINA
SINGAPORE
HONDURAS
AUSTRALIA
INDONESIA
BARBADOS
LITHUANIA
2
PORTUGAL
THAILAND
DENMARK
MOROCCO
MALAYSIA
GERMANY
HUNGARY
URUGUAY
ROMANIA
BELGIUM
UKRAINE
FINLAND
IRELAND
RWANDA
ICELAND
SWEDEN
AUSTRIA
ESTONIA
NORWAY
PANAMA
CANADA
POLAND
FRANCE
JORDAN
GREECE
TURKEY
KUWAIT
MEXICO
RUSSIA
BRAZIL
ISRAEL
LATVIA
MALTA
JAPAN
QATAR
CHINA
SPAIN
CHILE
1
INDIA
PERU
ITALY
UAE
expanding its domestic biotech nation’s business environment—its add to Ireland’s appeal. It leads in surprisingly, Ireland secured the
industry. To examine the impact of rules and regulations—encourages “business expenditures on biotech- highest score, 6.4. Most of the other
FDI on various nations, we used a or discourages FDI. This metric nology R&D,” ties for second in FDI countries also saw a significant
FOREIGN EXCHANGE published study (Schwab, K. The is scaled from strongly discour- and leads business impact of rules amount of technology introduced
How host nations prosper from Global Competitiveness Report, ages (1) to strongly encourages (7). on FDI. That combination cements through FDI. The lowest score, 3.1,
foreign direct investment 2014–2015. World Economic Forum According to our data, the rules Ireland’s position as a desirable went to Argentina.
Irelands attracts
(2014)) and analyzed three metrics: and regulations in most countries location for foreign businesses to Countries with strong technolo-
foreign investment
One of the many ways a country can prevalence of foreign ownership, encourage FDI. Ireland was ranked explore. gy flows gain a second benefit from
and technology
improve its biotechnology business business impact of rules on FDI, most encouraging, with a score of To measure how FDI can devel- FDI. In addition to the increased
environment is through foreign and FDI and technology transfer. 6.6, which explains part of the rea- op a nation’s biotech industry and employment in domestic enter-
direct investment (FDI), or when a For the prevalence of foreign son it enjoys so much FDI. And even increase its capabilities, we looked prises, this transfer of capabilities
person or organization controlling ownership metric—which is scored though Zimbabwe was saddled with at the levels of new technology often seeds a new generation of
a company in one nation is from an- from extremely rare (1) to highly the most discouraging score, 1.8, it brought in by foreign investment. homegrown tech-based companies.
other. So if someone from France, prevalent (7)—most of the coun- still received a reasonable grade for Using the same information source,
for example, owns a business in tries received a 4 or higher. The top the prevalence of FDI, suggesting we assigned scores from 1 (mean-
Qatar, that would be considered score, 6.3, went to Luxembourg, and that rules and regulations are not ing FDI had no impact on bringing
FDI for Qatar. In this way, foreign the lowest, 3.0, to Kuwait. the only factors influencing foreign in new technology) to 7 (signifying
capital and even expertise can flow The business impact of rules investment. it brought in new technology to a
to the host country, enhancing and on FDI assesses to what degree a Without a doubt, these metrics great extent) to each country. Not
NUMBER OF PATENTS
MOTHERLAND OF
INVENTION
Mapping the source of patents to
track innovation around the world
W
developed for Western conditions, and more drugs being patent with two Swiss inventors and one German inventor, vation Economy (http://web.mit.edu/pie/) research group
here are drugs currently invented, developed for previously neglected conditions. Switzerland would receive two-thirds credit and Germany observed that innovative companies in the United States
and where will future drugs come one-third credit. tend to emerge from
from? A key question facing drug de- WHERE ARE DRUGS INVENTED? In the global continental distribution of drug patent new entities, but in
velopers and policymakers is whether This study extends prior investigations (Friedman, Y. Nat. inventors, North America—largely the United States—has The continued Germany they are
drug development will shift locations, Rev. Drug Discov. 9, 835–836 (2010) and Friedman, Y. maintained a roughly 50% representation, Europe about often built on lega-
in much the same way that manufac- Nat. Biotechnol. 32, 523–524 (2014)) and goes further to 30% and Asia approximately 10% (Figure 1). For all of
dominance of cies. So, using new
turing jobs and business administrative jobs have moved examine patents covering marketed pharmaceuticals by the drug manufacturing occurring in Asia, there is little established company formation
from industrialized to emerging economies. leveraging objective drug-patent linkages. The logic is as measurable innovation. The continued dominance of pharmaceutical (i.e., the U.S. model)
Although the location of innovation might initially follows: 1) The United States is the world’s largest phar- established pharmaceutical innovation hubs suggests that as a metric for
seem to affect primarily the economics of healthcare— maceutical market, so most—if not all—globally valuable pharmaceutical innovation is very difficult to relocate. Just
innovation hubs innovation would
reducing drug prices or increasing throughput of new drugs should have U.S. patents. (The primary purpose of as Hollywood has maintained its position as the global suggests that create an unfair bias
drugs—it can also have qualitative implications. For this study is to compare innovation outside the United hub of the television and movie industries, so too have the pharmaceutical against Germany.
knowledge-based activities, such as drug development, States, so any potential bias emerging from focusing United States and Western European countries maintained The MIT group also
the scope of the research projects constrains the outputs. on U.S. patents must be weighed against the benefits of their leadership in pharmaceuticals (data not shown).
innovation is very noted that, contrary
For example, strong research funding in Western coun- leveraging rigid U.S. patent inventor listing rules and the When asked which nations in Asia are responsible difficult to relocate. to popular opinion,
tries directed at locally endemic diseases results in the objective drug-patent linkages provided by the U.S. Food for its drug inventorship, many propose that India or Chinese firms excel
development of drugs directed at those diseases. Likewise, and Drug Administration (FDA).); 2) For U.S. patents, all China might be the regional leader. A closer look at Asian in mass manufac-
if tropical countries, for instance, participated more in of the individuals who had “intellectual domination” of the countries demonstrates otherwise (Figure 2). Japan is the turing “not because of low-cost labor, but because of their
biopharmaceutical research, then more new drugs directed research must be listed as inventors, along with their loca- source of more than 90% of Asia’s drug patent inventors. ability to move complex advanced product designs into
at tropical diseases would be expected. tions, and listing too many or too few inventors can yield a Japan’s dominance in Asia further proves the permanence production and commercialization.”
While the precise outputs of innovative research proj- patent that is either unenforceable or invalid; 3) The FDA of pharmaceutical innovation hubs. Innovation has not Accordingly, if Chinese innovation is based on process
ects cannot be predicted (many successful drugs emerge as requires pharmaceutical manufacturers to submit a strictly moved in decades. improvement, rather than low-cost labor or develop-
ment of novel drugs, then one must ask: Why are China’s
FIGURE 1. FIGURE 2. policies directed at Western objectives? Through directed
INVENTORSHIP BY REGION incentives, China has established itself as a world leader
in scientific publishing and in patent filings (as shown in
previous issues of Scientific American Worldview). Low
citation rates of scientific papers and low patent-grant rates
JAPAN 779 demonstrate that these policies are not producing effective
NORTH AMERICA SOUTH KOREA 28 inputs for innovation.
EUROPE CHINA 12
INDIA 9
In other words, China’s current strategy promotes
ASIA
OCEANIA Western-style outputs to anachronistically build founda-
SOUTH AMERICA tions, rather than supporting its substantial capabilities
MIDDLE EAST
already in place. A better development path would be to
ASIAN DRUG PATENT INVENTORS leverage China’s unique strengths in advanced manufac-
(2000–2014) turing and design to improve on existing drugs and to
develop global leadership in research in areas that other
countries cannot address.
2001
2002
2003
2004
2005
2006
2007
2008
2009
2010
2011
2012
2013
2014
G
ing whether to allocate further re- innovation policies in place—such as increasing locally produced medicines sured by the U.S. Chamber of Com- investment. In addition, low costs,
iven the inherent sources in the nation. This is precisely the United States, United Kingdom and growing export markets. Yet, these merce’s GIPC International IP Index demand and market potential are not
challenges involved what the Biopharmaceutical Com- and Switzerland—score at the top of policies came prior to adequately ad- (GIPC Index), tend to host three to sufficient prerequisites for invest-
in the policymak- petiveness and Investment Survey the sample (Figure 1). In contrast, dressing areas like quality of manufac- five times fewer clinical trials than ment; rather, the policy environment
ing process, not (BCI)—a global survey-based index even considering their high levels of turing and scientific capabilities and the countries scoring in the upper half is an equally, if not more, important
least when it comes of countries’ biomedical-investment demand and future market potential, soundness of its regulatory and legal (Figure 3). In fact, regression analysis determinant. Finally, the outputs
to healthcare and attractiveness—aims to understand. those markets that do not yet provide frameworks. Not surprisingly, Russia of the data suggests that the strength themselves, or the lack thereof, actu-
biomedical innovation, the question is First presented in the 2012 Scien- wide-ranging support for biomedical scores below 60% of the total possible of IP protection can explain over 40% ally shed light on which policy inputs
often asked whether policies actually tific American Worldview (“Does your innovation—such as Brazil, Russia, BCI score, and is categorized as “strug- of clinical trial intensity, which is sig- are necessary to promote investment.
matter. At the end of the day, does the country deserve investment from India and China, the BRICs—still tend gling to compete.” Moreover, Russia nificant given that a number of other Ultimately, looking at the policies
state of the policy environment really biopharma?”), the BCI ranks nations’ to place at the bottom of the group. is ranked lowest in the exact areas in factors—such as adequate capabilities of countries that secure biopharma
translate into more or less investment biopharma investment environments Take, for example, countries which it seeks to be competitive, such and infrastructure—are also typically investment might provide a roadmap
and innovative activities in the bio- based on the perspectives of local using protectionist-type policies as in manufacturing, where it scores considered important for attracting for all nations seeking the same success.
medical sector? The answer is yes. executives. The BCI is composed of or requirements to stimulate local well below the rest of the group at just clinical trials.
A growing body of data shows 50 questions that capture the entire biomedical manufacturing or R&D. 54% (Figure 2). China makes an interesting exam- Meir Perez Pugatch is managing director
that there is a positive link between biopharmaceutical ecosystem, from The BCI results suggest that these and ple. By some measurements China’s of Pugatch Consilium and IPKM profes-
sor at the University of Maastricht. David
a country’s policy inputs—all of the scientific capabilities to market condi- similar policies that seek to coerce IP laws and their enforcement are Torstensson and Rachel Chu are partners,
laws, regulations and initiatives in tions. Using statistical analysis, re- localization do not constitute part of gradually making strides, but overall Amir Dayan is CTO, and Noa Weinstein is a
REASONABLY COMPETITIVE
STRUGGLING TO COMPETE
and its outputs, such as biomedical RESULTS BY
STRONGLY COMPETITIVE
BCI CATEGORY
R&D and the production of new OVERALL
OVERALL MARKET
MANUFACTURING
SCORES &
OVERALL SCORE
ENVIRONMENT
CONCLUSIONS
RANKING
CAPABILITIES
CONDITIONS
EFFECTIVE
UNITED KINGDOM
ARGENTINA 8.29 9.36 8.50 8.14 9.29 7.50 7.86 1.36 60.29 ARGENTINA
competitiveness of the local sector,
UNITED STATES
SOUTH AFRICA
INDIA 6.94 9.17 10.00 8.56 6.61 8.00 9.11 1.56 59.94 INDIA
but they also shed light on which
ARGENTINA
SINGAPORE
AUSTRALIA
TURKEY
INDONESIA
TURKEY 6.11 10.11 10.18 8.14 7.14 8.46 8.43 1.36 59.93
COLOMBIA
THAILAND
MALAYSIA
UKRAINE
areas nations might want to focus on
VIETNAM
NIGERIA
FRANCE
RUSSIA
TURKEY
RUSSIA 6.58 9.67 7.58 7.63 7.38 9.42 8.96 1.42 58.63
MEXICO
RUSSIA
BRAZIL
JAPAN
CHINA
CHINA
in order to enhance their chances of
INDIA
CHINA 7.73 7.92 8.62 6.69 7.42 8.19 9.38 1.65 57.60 CHINA
UAE
securing investment. BRAZIL 4.93 7.86 8.79 7.93 7.57 9.00 9.00 1.50 56.57 BRAZIL
©BALFOUR WALKER
tion created by CredibleMeds into medication, or recommends moni- level data from multiple clinical trials
has revolutionized patient
the systems that support healthcare toring or safe alternative(s) shared by several major pharmaceu-
care by harnessing the
decision-making. This work is also tical companies in order to better
power of collaboration. PHYSICIAN:
supported by grants from the Bert understand the course of Alzheimer’s
W. Martin Foundation and Oracle Prescription for » Directs therapy and provides disease. This database is now being
P
Health Sciences and made possible Azithromycin learning on how information re- used to design and simulate new
rescription drug use in CredibleMeds began as part of a by a new partnership between Credi- ceived can be made more useful clinical trials with greater likelihood
America continues to national network of Centers for Edu- bleMeds, Banner Health Systems or improved of success in testing new treatments
rise, and while properly cation and Research on Therapeutics and the University of Arizona Col- for this and other diseases.
prescribed medications (CERTs) launched by the federal lege of Medicine – Phoenix. yes no “Today, digital and cloud tech-
can have a markedly Agency for Healthcare Research and “We have developed a system to nologies allow us the ability to ana-
Accepted Medical
positive impact on pa- Quality in 1999 following a ten-year analyze drug safety evidence that can Indication lyze and communicate vast amounts
tients’ well-being, incorrect or inap- campaign by Dr. Woosley to obtain fuel a ‘behind-the-scenes’ software of information in an instant,” Dr.
no yes
propriate use of medicines can also Congressional legislation and fund- program that we call the ‘Auto-Pilot,’” Woosley concludes from his office in
have devastating consequences for ing for such programs. Dr. Woosley explains. “When a medi- TdP Risk Score Positive Oro Valley, Arizona, where he works
patients. Assessing and communi- “There was a critical need for cine is prescribed, all of the medical closely with scientists in the local
cating those benefits and risks has research that drug companies would evidence about the safety of that no yes Sanofi and Roche-Ventana facilities.
been the life’s work of Dr. Raymond medicine is com- “I feel like the tools and infrastruc-
ECG
Woosley. Over the past thirty years, “AZCERT’s concept for ‘Auto-Pilots’ is a bined with in- Screen
Concomitant
TdP Risk Drug
ture now exist to enable research
Dr. Woosley has matched his keen novel integration of innovative technologies formation found and healthcare delivery systems to
scientific acumen, vision and orga- in the patient’s fully utilize the enormous amount of
nizational skills, carving out new that furthers the FDA’s mission to reduce electronic medi- data and knowledge our scientists
areas of consensus science capable preventable harm from medicines.“ cal record. From QT yes are generating. But, partnerships
of tackling the complexities in both —Ali Mohamadi, M.D. that analysis, the such as those created by Credi-
the efficient development and safe system provides bleMeds and C-Path will continue
use of life-saving medications. He normally not perform,” he explains, valuable guidance to the patient’s to be essential for patients to reap
has made the greatest impact on “and that the National Institutes of healthcare team.” (See sidebar.) no maximum benefit from this invest-
medical progress as the founder of Health just doesn’t fund.” Of particu- Parallel to the development ment in science. Future advances
the Critical Path Institute and Credi- lar concern to Dr. Woosley was the of CredibleMeds is the continued will require collaborations among
bleMeds—both of which are guided potential for certain medications to growth of the Clinical Path Insti- translational scientists, those who
by the concepts of collaboration induce sudden cardiac death (SCD). tute (C-Path), which Dr. Woosley Dispense Dispense Azithromycin Suggest Safe deliver healthcare and those who
and data sharing. “My brother died of SCD in 1996 founded in 2005 in response to the Azithromycin with QT monitoring Alternative train healthcare practitioners.”
SUPER SIMULATIONS For decades healthcare ex- NATURAL HEALING As Abbate noted earlier, the
perts predicted that computers would transform the field, “tweaking” of natural processes can lead to innovative dis-
but until Colin Hill came along we did not know precisely coveries, which was the case for Bob Hariri. “We realized
Today’s visionaries battle cancer with cutting-edge Indeed, when a researcher’s discovery yields a revolution- how. Hill is combining computational physics, systems bi- probably 20 years ago that for cellular medicine to have a
science and the stubbornness to make it work ary new medicine, that tweaking can change lives. “Innova- ology and personalized medicine to completely rewrite the meaningful impact on patients’ lives we were going to have to
BY MIKE MAY | Illustrations by MEEN CHOI tion is inventiveness put to good use,” Bob Goldberg, one of way that clinical trials are done. Instead of running them identify a reliable, renewable source that could be turned into
S
the cofounders of CMPI, told the audience. “Inventiveness in people, he plans to simulate them in computers. a product and put into the hands of physicians—sort of the
urviving cancer requires teamwork. On the springs from the imagination of our honorees this evening.” That night in California, I asked Wuest: “Why test a same way we provide them with pharmaceuticals,” he said.
evening of Monday December 8, 2014, a small As I took the podium, I started to call up four of the drug in a computer?” While working as a neurosurgeon, he realized that
army of some of the best “soldiers” in the battle innovators who joined us that night: Patricia Ernst of the “You can gain a lot of insights,” she answered, “and stem cells would be particularly useful in treating head
against blood cancer mingled with patients and University of Colorado Denver–Anschutz Medical Cam- there are more opportunities to study disease on a ho- and spinal cord injuries, but he needed a source of them.
members of the press in “The Swamp” at the pus; Bob Hariri of Celgene Cellular Therapeutics; Carl listic level in ways that are cheaper and in ways that are So he turned to what he called “the leftovers of full-term
California Academy of Sciences in San Francisco. Just feet June of the University of Pennsylvania’s Perelman School faster because we can take in data that’s already being healthy pregnancies, namely, the placenta.” He added,
away, Claude—an albino alligator—laid still, but deadly, of Medicine; and Diane Wuest, representing Colin Hill of collected in the healthcare system.” That information can “This organ is nature’s stem-cell factory.”
much like an undiagnosed cancer waiting to unleash itself GNS Healthcare. I felt awed to be there as I invited Carl then be used to find just the right treatment for each in- At Celgene Cellular Therapeutics, Hariri and his col-
on an unsuspecting victim. The crowd gathered there that June to the panel. dividual patient. leagues convert stem cells into new therapies for diseases
night to learn how sophisticated research from dedicated CHANGING THE ODDS June’s groundbreaking ap- MINING NEW TARGETS Genetic solutions to cancer including cancer.
and driven scientists promises to—and in some cases al- proach to combating leukemia begins with the immune-sys- have been anticipated since the sequencing of the human NEVER GIVE UP! When I asked the panel how we can
ready does—cure cancer. tem cells from a particular patient, genetically re-engineers genome more than a decade ago, and some of today’s treat- be sure to keep innovation moving forward, June replied
This Top Medical Innovators forum was organized those cells to kill that person’s specific cancer and then injects ments do target changes in genes. But Patricia Ernst be- that scientists must be stubborn. “Basically, I tell people
through a partnership between Scientific American World- them back into that individual. He first used this protocol lieves that examining other cellular alterations will unveil when they start in my lab that you have to expect 90% of
view and The Center for Medicine in the Public Interest in 2010 to treat an adult with the disease—which typically even more targets. She looks at so-called epigenetics. the time—at least—you are going to fail, so if you’re play-
(CMPI). The event recognized eight experts for their work kills 80–90% of its victims—and the injected cells destroyed “Most people are familiar with the concept that when ing baseball, that means you bat 100,” he said. “You have to
in the fight against blood cancer, from diagnosis to treat- seven pounds of tumor. The man is alive and well today. you get cancer one of your genes has suffered a mutation get used to that, but when you do get something that works
ment (see “Innovator Alley”). Gaining ground in this dif- In addition, June had recently completed a study in and that’s why the cells grow uncontrollably,” Ernst ex- it’s an amazing thing.”
ficult battle demands crusaders like these to continue the which 39 children with leukemia—for whom several tra- plained, “but there are a lot of other things that can happen The things that work change lives (see “Marathon
mission of developing innovative new treatments. ditional treatments had failed—received their own re- to a cell.” For example, the molecules that control genes— Man” and “Saving Dolphins Despite Disease”). As Hariri
As Jeremy A. Abbate—publishing director of Scientific engineered immune cells. Of these children, 35 went into known as regulators—may also cause or affect the growth said, “I am moved and compelled by each story where the
American Worldview and global vice president for global complete remission. That translates into a response rate of of cancer. This is an example of epigenetics, and such investment and the time of creating a new drug, a new
media alliances at Scientific American—said in his intro- 92%! As June said, “It’s a heartwarming story, because you changes can impact the onset and severity of leukemia. therapeutic, has transformed those individuals’ lives.”
duction, “One of the most defining characteristics of the see kids who have gone through all the kinds of things that “There are many new drugs that are being developed As I thought over what the panel had said that night
enterprise of innovating is taking nature and making it a chemotherapy can do, or radiation, and most of them al- that not only influence heritable changes in gene expres- and looked over the crowd, I felt moved, too, and optimis-
little bit better, using what nature gives us and tweaking it ready relapsed after a bone marrow transplant—but after sion,” she continued, “but also directly target epigenetic tic about a more powerful today and an even more innova-
just a little bit.” immunotherapy, these kids have returned to a normal life.” regulators that are mutated in particular leukemias.” tive tomorrow.
I
n February, several weeks before he planned to run his
72-year-old Don Wright—a multiple myeloma DESPITE DISEASE
86th marathon, Don Wright made time to talk with survivor—takes aim at his 100th marathon Hardy Jones fights to protect marine
Scientific American Worldview. A few years earlier, in life even as he battles cancer
I
December 2012, he had achieved his goal of finishing one
marathon in each of the 50 states. Now, he wants to raise n 2000, life couldn’t have been better for life-long diver and filmmaker
his marathon count to 100, a feat he hopes to reach by Hardy Jones. Teaming up with actor Ted Danson, Jones founded BlueVoice.
the end of 2016. But without a doubt, his greatest accom- org to protect whales and dolphins. But in 2003, he was diagnosed with
plishment is staying healthy despite being diagnosed with multiple myeloma. His treatment started with an oral cancer medication and
multiple myeloma just after his first marathon, in 2003. a steroid, which stops the growth of cancer cells in the bone marrow. “My
Then, instead of giving in to the cancer, he decided to myeloma burden was reduced by 97%,” Jones says. “It was very fast.”
take on the Boston Marathon, followed by one race after He started on high doses of the medications, but his oncologist brought them
another. His wife and daughter often come along and down over time. Eventually, Jones got 16 months off without any treatment
run the half marathon at the events he attends. Wright at all. “In that 16 months,” he says, “I was quite stable, but then the myeloma
can keep running in spite of his myeloma because of a began to creep up.” So he tried another drug and experienced a similar cycle—
once-a-day pill. He has been on the therapy for seven treatment, quick cancer-killing response, eventual treatment vacation, followed
years—starting with a clinical trial five years before it was by cancer recurrence, which led to a new treatment.
approved—without any major side effects. Well, except for At 71 years old, Jones continues to receive treatment and pursue his work.
one, he laughs: “The big side effect is that it makes me want When he spoke with Scientific American Worldview, he was deep in a project to
to go run marathons!” save dolphins being killed in Peru for use as shark bait. “We’re making a film on
Jokes aside, Wright deeply appreciates his treatment it now,” he says. “If we don’t stop this dreadful practice, they will wipe out the
as well as his health. And of his current cancer-fighting dolphins and sharks.”
medication, he says, “It’s helped me live long enough to While Jones saves the world’s marine life, advanced drugs—with ongoing
meet my grandchildren, and that’s extremely important in innovations keeping his options coming—save him. There is no better team-
my life.” work than that.
THE MODI
sector interests,” says Qaim. “But the
GREAT STALL process. And in addition to these enormous increase in the registration SEEDS OF results don’t support that argument.”
OF CHINA
global firms, says Cho, “The domestic
companies who are focusing on their
of innovative medicines. “Now they
have their own R&D pipeline from CONTROVERSY Another widely cited concern is that
journals would only publish studies
OPERANDI
Regulatory paperwork further own R&D products are facing the China and around the world,” Cho German research provides clarity with significant benefits. “We didn’t Tackling India’s social
same challenges.” says. “They need to be able to cope find any evidence of such publication problems with IT
slows drug approval in the ongoing crop debate
RDPAC is working hard for with the advancement of the pharma bias,” he notes.
BY REBECCA KANTHOR change, but has had limited success R&D here. That is a very huge chal- BY KEREN SOOKNE In addition, some GM-crop BY ZACH GOLDBERG
P D B
so far. The real challenge is getting the lenge.” skeptics fear that the economic and
atience is a necessity for ear of the right high-level decision Last year, the CFDA announced espite numerous scientific agronomic benefits they provide are y 2001, the Western
pharmaceutical companies makers. “Policy making in China is so the recruitment of 20 new Center for studies demonstrating only short-term. “This is often men- Indian state of Gujarat—
seeking to bring drugs to fragmented, so we are facing different Drug Evaluation employees, adding the benefits and risks of tioned in connection with resistance known locally as the
market in China. Last year, ministries and to its existing staff of 80 overworked genetically modified (GM) development in insect pests or weeds,” “Jewel of the West”—was
the six-to-eight-year drug sometimes they reviewers, and earlier this year they crops, they continue to be Qaim says. But resistance develop- deep in the doldrums.
lag these firms have had to contend Last year, come up with announced the addition of 53 review- the subject of vehement debate. Low ment is not an issue that is specific Years of political instability, misman-
with was lengthened another two the six-to- different ideas ers. In May, CFDA deputy head Yin levels of public trust regarding GM to GM crops. “This can also occur agement and corruption had frittered
years. “A lot of products were asked eight-year about how things Li announced plans to outsource crop safety present a major problem with conventional and biological pest away precious resources. The public
to queue in the line again to get the drug lag should be done,” some of the reviewing to third-party for experts and governments, as the control technologies, and it hap- cried out for a savior. Instead, they
approval for clinical trial stage,” says these firms says Cho. At organizations. Even so, its capabilities demand for technological advances pens faster when good agricultural got an earthquake that killed 20,000
Joseph Cho, who heads RDPAC, an have had RDPAC’s March don’t compare to the FDAs in other focused on food security has never practices aren’t followed,” he says. The residents, destroyed nearly 400,000
association of 40 multinational R&D- to contend 2014 meeting countries like the United States. been more pressing. Seeing a need for bottom line: farmers cannot substitute homes and caused over US$5.5 bil-
based pharmaceutical companies with was with China’s As the agency struggles to get an objective breakdown of the facts, GM seeds for recommended agrono- lion in damages. From the fallout,
working in China. The extension was lengthened State Food and through the backlog of approvals and Matin Qaim, an agricultural econo- my techniques, such as crop rotation. Narendra Modi, a former tea peddler
unexpected. “We say it is a shock, not another Drug Adminis- pick up the pace, Cho says foreign mist at the University of Goettingen, As Qaim puts it, GM seeds “are not and science enthusiast, was catapulted
a surprise,” he relates. two years. tration (CFDA), pharma is increasingly frustrated. “I Germany, and his colleague Wilhelm magic bullets.” into power.
Since being recognized in 2005 as officials revealed think the sudden change of prac- Klümper conducted a meta-analysis As European consumer polls call Gujarat’s unpopular government
a nonprofit by the Chinese govern- the government’s tice is making the lives of our R&D on the effects of GM crops on pesti- for more public information, Qaim intended for him to be the deputy
ment, RDPAC has developed into a rationale for the people and regulatory people on the cide use, crop yields and farmer prof- continues to participate in the GM chief minister. But Modi refused: “I’m
lobbying voice for improving China’s added two years. ground and at headquarters frustrat- its. Their report, based on 147 studies, debate by addressing public con- either going to be fully responsible
pharmaceutical R&D environment. “They think ing. A lot of investments are being was published on November 3, 2014. cerns through his research. He notes for Gujarat, or not at all.” Some called
According to its website, the orga- they have been making mistakes in affected by these uncertainties here in “Many people in Europe believe that even in the face of clear scien- him arrogant, if not authoritarian. In
nization is “committed to securing the past, and they just want to do the China.” GM crops do more harm than good,” tific data, it may take some time to truth, Modi simply understood sci-
patients timely access to innovative right things,” he explains. explains Qaim. “We felt that a publicly overcome public distrust, as has been ence and technology’s ability to solve
drugs”—a formidable task, particu- Indeed, the CFDA is playing funded analysis of studies carried out the case with countless other scien- the seemingly unsolvable.
larly in China. As of early January catch-up with the pharmaceutical worldwide would be a useful contri- tific advances in history. Still, Qaim His model is simple: Lay the infra-
2015, the group reports that at least R&D industry. Whereas in the past it bution to the public debate.” The re- remains hopeful: “I’m optimistic that structure—electricity, roads, schools,
34 applications from multinational reviewed mainly generics, since China sults indicate that farmers employing more evidence about the benefits of public transportation, broadband and
drug companies have been delayed joined the World Trade Organiza- insect-resistant or herbicide-tolerant GM crops will contribute to wider so on—and harness information tech-
by a new step in the drug-approval tion in 2001 the agency has seen an GM seeds earned 69% higher profits public acceptance in the future.” nology (IT) to streamline governance
A
projects and sci- cals, Japan’s biggest pharma company. biofuels. The renewable-energy com-
entific research lthough Japan ranks third Five pharmaceutical firms, including pany Statkraft and the Swedish forest
hubs in the pipeline, Modi has faith worldwide in develop- Takeda and Eisai, helped jumpstart Norwegian wood may power company Södra are exploring biofuel
in the force-multiplying effects of sci- ing new pharmaceutical the fund by persuading Japan’s foreign tomorrow’s air travel production at a former cellulose fac-
ence and technology. products, its contribu- and health ministries to finance half tory in Hurum, while Avinor has
Consider Modi’s “Digital India” tion to global health lags of it. The remainder is currently BY NANCY BAZILCHUK pledged roughly US$12 million over
W
campaign: by expanding broadband far behind. That imbalance may be backed by six Japanese pharmas, the the next decade for biofuel projects. A
and mobile Internet coverage across shifting, however, with the launch of Gates Foundation and the United Na- ith more than 2013 study commissioned by Avinor
the country, virtual medical technolo- the Global Health Innovative Technol- tions Development Programme. 2,500 kilometers of and the Norwegian airline industry
gies can then be used to ameliorate a ogy (GHIT) Fund, the world’s first Although the GHIT doesn’t seek coastline rumpled concluded that Norway could sustain-
health crisis compounded by the 700 public-private partnership dedicated financial returns, awarded developers by deep fjords and ably produce up to 230 million liters
million Indians who live isolated from to supporting global health R&D. must show that their potential products rugged mountains, of biofuel from wood products at
the nearest hospitals. Since its establishment in 2013, the are innovative, feasible, cost-effective Norway seems tailor-made for the competitive prices by 2025.
On this front Anita Goel, CEO Tokyo-based, US$100 million initiative and accessible to the poorest of the airline industry. In fact, industry ex- “There is a need and space for
of Nanobiosym and developer of has funded 30 partnerships between poor. The fund’s investment covers perts are known to joke that “When aviation in the future world,” Larsen
the Gene-RADAR technology—a Japanese and non-Japanese companies drug discovery to clinical testing, and God created Norway, he was think- says. “We just have to make sure it is
wireless iPad-sized diagnostic tool and public institutes aiming to develop it opens the door to access Japan’s vast ing about aviation.” as sustainable as possible.”
M
He adds that very few pharmaceutical clear that the Romanian scientist was
companies have bothered to re-engi- not sufficiently recognized for his any experts wonder
neer their cell lines to achieve higher contributions to the field. And today, what will emerge from
J
to register four or five biosimilar for patients and healthcare providers centers in Bucharest, Cluj-Napoca, laborates with international advisors, Africa, whose growing momentum
ust as generic versions of drugs with the EMA in the next few worldwide. Brasov and other cities. But the first including the U.S.-based nonprofit can’t be ignored? And how will
small-molecule drugs have years, according to Klaus Martin, dedicated biotechnology cluster in Center for Integration of Medicine Russia’s past, with its complicated at-
helped to reduce health- the company’s head of biologics. Romania—and southeastern Europe for and Innovative Technology. titudes toward business and capital-
care costs, biosimilars offer “Like generics, that matter—is bioROne in Iaşi, located Eventually, scientists at bioROne ization, affect the country’s present
an alternative to the class biosimilars are in the northeastern part of the country. hope to commercialize their laborato- ability to cultivate aspiring entrepre-
of medications known as biologics. Over the very important Researchers at bioROne focus on ry discoveries. In order to develop its neurs from within?
Unlike small-molecule drugs, which next decade, to rein in health- biomedicine, including bioinformat- own pharmaceutical products, how- In Lonely Ideas: Can Russia Com-
are derived from chemicals, biolog- biosimilar care spending ics and biopharmaceuticals, genetic ever, the cluster will need far more pete? (The MIT Press, 2013), Loren
ics are produced from living cells. drugs may and allow wider testing and gene therapy. In the past funding. As it stands its entire annual Graham chronicles the brilliance of
Generic-like versions of biologics well provide access to drugs,” five years, scientists there have been budget would barely pay for a small Russian inventiveness, as well as the
are called biosimilars because they substantial he says. Cur- granted 100 patents, and the center’s fraction of the cost of developing a
work like their patented counterparts, price and rently, the firm cumulative budget during that period single drug. Alternatively, bioROne And how will Russia’s
but—unlike generics—can have small market has a proprietary was about US$56 million. is looking to attract clinical trials or past, with its complicated
structural differences. Over the next competition portfolio, but Truly a collaborative venture, start-ups to the region. To do this, attitudes toward business
decade, biosimilar drugs may well for biologics. Martin says at the cluster was launched by a group the Romanian government is taking and capitalization, affect
provide substantial price and market least some are representing the country’s leading steps to improve its infrastructure, the country’s present
competition for biologics, which monoclonal antibody drugs that will universities, research centers, industry for example, by joining forces with ability to cultivate aspiring
made up a US$170 billion industry in soon be coming off patent. “I’m quite members and other key organizations. the European Union and the Grigore entrepreneurs from within?
2013 and ranked among the world’s confident that Polpharma can deliver Among the 12 founding partners T. Popa University of Medicine and
most profitable drugs.
Many biologic drugs will soon
quite a cost-competitive set-up,” he
says. “If you look at originator drugs,
REDEFINING are the Grigore T. Popa University
of Medicine and Pharmacy Iaşi, the
Pharmacy Iaşi to raise about US$11
million for a research facility in nation’s continued failure to sustain
lose patent protection in Europe many of these use technologies that ROMANIA Institute of Macromolecular Chem- northeastern Romania to be used for and build upon its scientific achieve-
and the United States, opening the were available 10, 15, 20 years ago. istry “Petru Poni” Iaşi, the Institutul preclinical and clinical studies. ments. A leading U.S. scholar on
Generating the infrastructure
door to manufacturers poised to The cell lines they used years ago Naţional de Cercetare-Dezvoltare Despite Romania’s underappreci- Russian science and technology and a
bring biosimilar versions to market. were nowhere near as productive as
and financing to establish a pentru Fizică Tehnică Iaşi, the clinical ated status in the biotech field, some foreign member of the Russian Acad-
Europe has been at the forefront of modern systems.” vital biotech presence hospitals Spitalul Clinic Judetean de metrics do attest to its impact. For emy of Sciences, Graham goes on to
the biosimilar industry, adopting a The first biologics manufacturing BY BACHIR ABI SALLOUM Urgente Sf. Spiridon Iaşi, Spitalul example, a search of PubMed revealed question whether Russia can reverse
I
regulatory framework in 2005. The facilities relied on large, dedicated Clinic de Recuperare Iaşi and Insti- more than 500 scientific publications in its centuries-old pattern of stifling
European Medicines Agency (EMA) fermenters and fixed stainless steel n 1916, Romanian physiologist tutul Regional de Oncologie Iaşi, SC 2014 based on work carried out in Iaşi. domestic technological progress.
has approved 20 biosimilars, and the pipes that required time-consuming Nicolae Constantin Paulescu Antibiotice SA, which is the largest To move from research to commercial “[Russians] have made the
U.S. Food and Drug Administration and expensive work stoppages to developed a pancreatic extract Romanian-owned pharmaceutical results, however, Romania must ensure mistake again and again and again
approved its first biosimilar product, a clean and maintain. In contrast, that was an early version of the company, and the public agencies that the world sees its discoveries—and of thinking that modernization is
version of the white blood cell–boost- modern cell-line technology uses life-saving insulin used to treat Institutul de Medicina Legala Iaşi continue to build an industry that will the same thing as obtaining technol-
ing filgrastim, in March 2015. smaller-batch, flexible systems with diabetes. Although he was not among and Directia de Sanatate Publica generate more of them. ogy,” says Graham, who is a research
S M
making it possible for individuals and sea urchins, sea cucumbers.” Endotoxins or “pyrogens”—from resources official, crab suppliers and
small groups to seek funding outside ynonymous with golf and Researchers at Scottish companies ore spider than crus- the Greek for “fire” because they cause handlers to discuss best practices for
of state control and direction. In ad- whiskey, Scotland brims have been investigating seaweed and tacean, the horseshoe fever—reside in cell wall membranes minimizing loss as crabs are shuttled
dition, foreign technology companies with something else that its microscopic cousin, microalgae, crab resembles a ta- of E. coli and other so-called gram- between the ocean and lab and
have made inroads and forged col- might one day help define for a variety of uses, Fowler explains, rantula wearing an old negative bacteria that can induce back. In the early 1990s, alarmed by
laborations with Russian scientists and this nation: seaweed. At least including nutritional supplements, army helmet. It’s a liv- lethal bloodstream infections. These dwindling crab populations, Charles
nascent entrepreneurs. that’s the hope of biotechnology boost- anti-aging creams and other “cos- ing fossil, its basic design unchanged “dead bug parts,” as Dubczak calls River found itself in an unusual
Yet foreigners with available cash ers Donald Fowler and Jim Brown. meceuticals,” or pharmaceutical- in 440 million years. And the Atlan- them, in a concentration of 70 parts position for industry—lobbying for
don’t feel legally secure investing in Fowler, based in Argyll, is senior cosmetics hybrids. In addition, tic horseshoe crab, Limulus poly- per billion, “will make a person sick.” more regulation. South Carolina now
Russian science, according to Graham. development manager for life sciences Scottish biotechnologists see other phemus, can survive out of water for Limulus blood clots when it encoun- limits times and places where crabs
Reforming Russian laws to provide busi- with the Highlands and Islands (HIE), natural-product targets that fit today’s weeks, on beaches from Maine to the ters an endotoxin concentration of can be collected and bans their use as
nesses with greater intellectual property a government economic and commu- going-green philosophy. For example, Yucatan. Moreover, royal bluebloods just 1 part per trillion. bait. Consequently, crab populations
protections, he says, would go a long nity development agency for the north the Scottish firm CelluComp converts have nothing on the horseshoe crab. “You’ve seen an IV bag?” Dubczak rebounded.
way toward improving the country’s and west of Scotland. Brown directs the vegetable processing waste from car- Its blood really is blue, enlisting asks. “The solution in that bag has In recent years, Charles River
economic prospects. Otherwise, Russia annual Natural Product Biotechnology rots and beetroot and other naturally copper, rather than iron, to transport to be tested for the absence of these invented a cartridge that requires
will likely maintain the status quo, or conference in Inverness. derived waste products into pigments, oxygen. That blue blood—says John pyrogens using our LAL reagent.” So 20 times less blood than its original
as Graham describes it, its “consistent “There’s a huge resource of kelp specialty celluloses, coatings and Dubczak, general manager of the do the tubes and needles connecting assay. That’s good for business and
record, both brilliant and dismal.” forest off the west coast,” Brown says. rheology products. endotoxin and microbial detection the bag to the patient. horseshoe crabs.
AGING 2.0
thinking about these tools years ago. in our youth. Over the years, though, regenerative engine, and we do that
this regenerative “engine” runs lean by replenishing the reservoir of stem
FROM TRASH TO TREATMENT on fuel, the stem cells. As that hap- cells that provide synthetic versatility.
Early in my medical career, I special- pens, the defects of age start to accu- With this knowledge, we can iden-
Stem cells and digitized DNA ized in the treatment of head and mulate. In addition, stem cells orches- tify defective products—cells or tissues
may hold the key to high- spinal cord injuries. With one patient, trate our response to injury, making or organs—and then use stem cell–
performance longevity a defect in the tissue that surrounds them perhaps our best defense against driven synthesis to restore the function
A
the brain, the dura mater, needed to disease. The susceptibility to disease of those parts. Doing this, though,
lthough we can’t live for- be repaired after a serious head injury. thus increases as we age. depends on a deep understanding of
ever, we can aspire to live I realized, from my ob/gyn rotation, how the biological software, a stem
much longer and health- that the amnion—this amazing, clear REPOSITORY OF REPAIR cell’s DNA, drives repair in its youth
ier lives. In fact, living plastic-like tissue that surrounds an How can stem cells fix things? It’s in and loses that ability with age. So by
well into our 90s lies just embryo—looked a lot like the tissue their DNA, which forms a repository better understanding aging and the
ahead, if we keep fine-tuning the right around the brain that I needed to of synthetic repair. Every stem cell molecular changes that drive it, we can
tools. We start by understanding that replace. That moment spawned other contains information, in its DNA, that learn to slow it down or work around it
aging arises from an accumulation of ideas about biological tissues being codes for the production of molecules in places. We can find ways to use stem
defects in our biology, and this causes used in new ways. In particular, I that guide the signaling and synthe- cells—maybe our own, those from
joint decay, decreased muscle mass, started thinking that the placenta— sis behind all of the steps that make someone else or from a placenta—to
Alzheimer’s and so forth. With digital the leftovers of birth—could be used tissues and organs. In this process, control certain diseases or to restore
health—basically, using high-perfor- as a source of stem cells. Instead of a primordial stem cell undergoes a functionality as we age.
mance computational tools to study just throwing away the placenta after series of cellular divisions that make The future of stem cells and the
complex biological processes—we can future of cellular medicine will benefit
interrogate the cellular and molecu- from this analogy with computers and
lar events that occur during human Living well into our 90s lies just ahead, digital processing. Your software, your
aging, and identify those that can
be controlled or modified to slow or
if we keep fine-tuning the right tools. biological software, that is, resides in
the nucleus. It’s not that different than
arrest those that degrade or degener- having binary code that resides in the
ate our bodies over time. To make the birth, we could make use of it. My it more specialized at every step. We memory access of a computer. This
most of digital biology, I joined forces personal “eureka moment” led me can watch such a change under a thinking leads us to the concept of re-
with my friends and colleagues— to form Anthrogenesis, which later microscope, as this primordial cell programming the biological software
Craig Venter and Peter Diamandis— became Celgene Cellular Therapeu- turns into a heart cell or a neuron—all of stem cells, which is already hap-
to form Human Longevity, Inc. (HLI). tics, where we mined the placenta as a depending on its surrounding envi- pening in activities to create induced
At HLI, we combine knowledge source of stem cells that can be turned ronmental cues that drive the DNA to pluripotent stem cells. These tools
from many areas of biology—the into treatments. create different things. provide a platform for controlling fate
genome, proteome, biome and A stem cell carries the remark- This DNA makes up a sort of and function, and they have broad
more—with advanced approaches able capacity to participate in biological software. Like lines of biomedical applications. The most
to computing and informatics, all to renovation or repair at any place in digital code, the genes in the DNA exciting one to me is prolonging and
create sophisticated cellular therapies. the body. Moreover, we can get these can be processed to drive an action, extending the quality of life.
To build these treatments, we are col- cells from many places beyond the like generating a protein. As a stem
lecting data on genomes and health placenta, including bone marrow cell develops into a specialist like a Robert Hariri is the founder and chairman of
outcomes from people around the and even fat tissue. Stem cells all neuron, though, it loses the ability Celgene Cellular Therapeutics, where he turns
stem cells into therapies for many diseases,
world. All of that information will be “think” they are still in a fetus, and to be anything else. That neuron, for cofounder and vice chairman of Human
combined to build powerful cellular that is perhaps the most regenerative example, can’t turn on the genes that Longevity, Inc., and founder and chairman
therapies—actually developed from environment of all. In fetal surgery, make a heart cell. And this is what of Myos Corporation, a company developing
enhanced human cells—that will for instance, you can open the uterus happens as our cells age—they lose products that improve the health of muscle. He
trained as a neurosurgeon, is an avid jet and
©ALEX NABAUM
battle cancer, diabetes, obesity, heart early in a pregnancy, perform sur- their versatility. At some point, we
high-performance aviator and has produced
disease, dementia and more. Further, gery on the fetus, close up and let the lose the ability to rejuvenate. several feature films and documentaries.
these treatments will keep our bodies baby come to term—and you won’t As we gather data on human
and minds performing as if they were see a scar. You won’t see any evidence genes and the outcomes that they cre- Opinions expressed in worldVIEWpoint do
younger and for a longer time. of the surgery at all. So a fetus can ate, healthcare experts can turn that not necessarily reflect those of the editors.