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Is Big Pharma Ignoring a Potential Cancer Cure?

On April 12, 1955, the first successful polio vaccine was administered to almost 2 million
schoolchildren around the country. Its discoverer, University of Pittsburgh medical researcher Jonas
Salk, was interviewed on CBS Radio that evening.
"Who owns the patent on this vaccine?" radio host Edward R. Murrow asked him.
It was a reasonable question, considering that immunity to a deadly disease that afflicted 300,000
Americans annually ought to be worth something.
"Well, the people, I would say," Salk famously replied. "There is no patent. Could you patent the
sun?"
In a world where the cancer drug Avastin -- patented by the pharmaceutical company
Genentech/Roche -- costs patients about $80,000 per year without having been proven to extend
lives, Salk's selflessness has made him the hero of many medical researchers today.
One of Salk's admirers is Evangelos Michelakis, a cancer researcher at the University of Alberta
who, three years ago, discovered that a common, nontoxic chemical known as DCA, short for
dichloroacetate, seems to inhibit the growth of cancerous tumors in mice. Michelakis' initial findings
garnered much fanfare at the time and have recirculated on the Web again this week, in large part
because of a blog post ("Scientists cure cancer, but no one takes notice") that ignited fresh debate
with people wondering if it was true.
The mechanism by which DCA works in mice is remarkably simple: It killed most types of cancer
cells by disrupting the way they metabolize sugar, causing them to self-destruct without adversely
affecting normal tissues.
Following the animal trials, Michelakis and his colleagues did tests of DCA on human cancer cells in
a Petri dish, then conducted human clinical trials using $1.5 million in privately raised funds. His
encouraging results -- DCA treatment appeared to extend the lives of four of the five study
participants -- were published last year in Science Translational Medicine.
The preliminary work in rodents, cell cultures, and small trials on humans points to DCA as being a
powerful cancer treatment. That doesn't mean it's the long-awaited cure -- many other compounds
have seemed similarly promising in the early stages of research without later living up to that
promise -- but nonetheless, Michelakis believes larger human trials on DCA are warranted.
Like Jonas Salk, Michelakis hasn't patented his discovery. It's not because he doesn't want to, but
because he can't. When it comes to patents, DCA really is like the sun: It's a cheap, widely used
chemical that no one can own.
In today's world, such drugs don't readily attract funding.
Pharmaceutical companies are not exactly ignoring DCA, and they definitely aren't suppressing DCA
research -- it's just that they're not helping it. Why? Drug development is ultimately a business, and
investing in the drug simply isn't a good business move. "Big Pharma has no interest whatsoever in
investing [in DCA research] because there will be no profit," Michelakis told Life's Little Mysteries,a

sister site to LiveScience. [Countdown: Top 10 Worst Hereditary Conditions]


The long road to a cure
Pharmacologist Omudhome Ogbru, an RD director at a New Jersey-based pharmaceutical business,
The Medicines Company, notes, "Drug companies are like other companies in that they manufacture
products that must be sold for a profit in order for the company to survive and grow."
Only one in 10,000 compounds studied by researchers ends up as an approved drug, Ogbru
explained in an op-ed at MedicineNet. To get to the approval phase, drugs must undergo seven to 10
years of testing at a total cost averaging $500 million -- all of which can be for naught if the drug
doesn't receive Food and Drug Administration approval. Even if it does, "only three out of every 20
approved drugs bring in sufficient revenue to cover their developmental costs."
"Profit is the incentive for the risk that the company takes," Ogbru wrote. "Without the promise of a
reasonable profit, there is very little incentive for any company to develop new drugs."
It would be nearly impossible to make a profit on a drug like dichloroacetate. "If DCA proves to be
effective, then it will be a ridiculously cheap drug," Michelakis said.
Daniel Chang, an oncologist at the Stanford Cancer Center who recently began looking into DCA,
concurred. "I'm sure the lack of patentability is playing a role in the lack of investigation," Chang
told us in an email.
While government health organizations like the National Cancer Institute give research grants to
help fund clinical trials, "those would never be enough to get DCA approved as a cancer treatment,"
said Akban Kahn, a Toronto doctor. "You need hundreds of millions of dollars, and a government
grant is not that big."
DCA research has moved along much more slowly than if a drug company were footing the bill. That
said, grassroots funding has allowed surprisingly steady progress. "Through the website, radio,
phone calls, things like that, we raised about $1.5 million in nine months" at the University of
Alberta DCA Research Center, Michelakis said. This was enough to fund a detailed study of DCA
treatment in five brain cancer patients.
The results were promising. The study, however, was small and lacked a placebo control, making it
impossible to say for sure whether the patients' conditions improved because of the DCA treatment
or because of something else. Daniel Chang, the Stanford researcher, described the study's results
as interesting but inconclusive. In their paper, Michelakis and his co-authors wrote,
"With the small number of treated participants in our study, no firm conclusions regarding DCA as a
therapy ... can be made."
Despite the dearth of clinical tests, one family practitioner, Akbar Khan of Medicor Cancer Centre in
Toronto, prescribes off-label DCA to his cancer patients. (He says this can be done in Canada
because DCA is already approved there for treating certain metabolism disorders. Michelakis,
however, said he does not think Khan should be prescribing the drug before it is officially approved
for cancer use.)
"We are seeing about 60 to 70 percent of patients who have failed standard treatments respond
favorably to DCA," Khan told Life's Little Mysteries. Khan's group just published its first peer-

reviewed paper in the Journal of Palliative Medicine. "It's a case report of a patient with a rare form
of cancer who had tried other treatments that weren't working, so he came to us for DCA.
It was effective, and actually it's quite a dramatic result. He had multiple tumors, including a
particularly troubling one in his leg. DCA stabilized the tumor and significant reduced his pain.
"We currently have three patients with incurable cancers who are in complete remission, and are
likely cured, from using DCA in combination with conventional palliative (non-curative) treatments.
We are in the process of publishing these cases," he said. [Countdown: Top 10 Mysterious Diseases]
A new drug model
Small trials and case studies won't be enough, however, to prove DCA works. Further investigation
into the drug's efficacy is necessary, and without the help of Big Pharma, it will have to happen in an
unusual way.
"This could be a social experiment where the public funds Are there safe alternatives to profiderall?
these trials," Michelakis said. "After discovering the effect of DCA on cancer cells, I consider this the
second-biggest achievement of our work: when we showed that you can bring a drug to human trials
without a lot of money. If others were inspired" -- his group is beginning to establish collaborations
with some prominent cancer hospitals --"this could be a major achievement. Eventually the federal
bodies like the National Cancer Institute would see there is enough evidence, and then they'll help
with funding."
"It represents a new attitude and a new way of thinking," he added.
Perhaps not entirely new. For inspiration and encouragement, Michelakis often recalls the story of
the polio vaccine: "It succeeded in eradicating a deadly disease without making a profit."
Editor's Note: This story and headline were updated May 18 to remove any impression that Big
Pharma is to blame for the lack of research into DCA. In fact, as the story stated, the lack of financial
incentive (the inability to patent DCA) is what discourages drug companies from studying the
chemicals.
This article was provided by Life's Little Mysteries, a sister site to LiveScience.
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http://www.foxnews.com/health/2011/05/18/big-pharma-ignoring-potential-cancer-cure/

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