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Thalidomide: Research advances in cancer and other conditions

http://www.mayoclinic.com/health/thalidomide/HQ01507

Despite its dubious history, thalidomide has proved effective in treating some diseases. Weigh
the benefits and risks of the drug to help you decide whether thalidomide is right for you.
By Mayo Clinic staff

For many, the word "thalidomide" conjures up images of babies born with shortened or missing
arms and legs. Between 1957 and the early 1960s, thalidomide was used by several thousand
pregnant women across the world to ease their morning sickness. But many who took
thalidomide in the early stages of pregnancy gave birth to babies with severe birth defects.

Now, decades later, thalidomide isn't used for morning sickness. But it has received approval
from the Food and Drug Administration (FDA) to treat one skin condition and a type of cancer,
and it's being investigated as a treatment for many other disorders.

Thalidomide's return isn't without controversy, though. Children born to mothers who took
thalidomide fear that allowing people to take the drug could mean more babies will be born with
severe disabilities.
Thalidomide proves useful for skin lesions and multiple myeloma

In the mid-1960s, scientists determined that thalidomide was an effective treatment for erythema
nodosum leprosum, skin lesions caused by leprosy. The FDA approved thalidomide (Thalomid)
for this use in 1998.

Since then, thalidomide has also demonstrated usefulness in treatment of multiple myeloma — a
blood and bone marrow cancer. In May 2006, the FDA approved thalidomide, in conjunction
with dexamethasone, for the treatment of newly diagnosed multiple myeloma. Thalidomide
appears to slow the growth of myeloma cells and prevent them from attaching to bone marrow
cells.

Areas of thalidomide research

Researchers continue to investigate thalidomide for use in treating a variety of diseases and
conditions. Though more study is needed to evaluate the risks and benefits of the drug,
thalidomide has shown promise in treating:

■Inflammatory diseases. Thalidomide reduces the production of tumor necrosis factor-alpha

(TNF-alpha) — a cell protein that can cause inflammation. People with inflammatory conditions
such as arthritis and Crohn's disease have high levels of TNF-alpha in their bodies.
■HIV-related mouth and throat ulcers. Although this use hasn't been approved by the FDA,
doctors can prescribe thalidomide for these HIV-related ulcers (off-label use). Research shows
thalidomide might also help treat Kaposi's sarcoma, a cancer of the blood vessel walls mostly
found in people with HIV, as well as help treat weight loss and body wasting associated with
HIV.
■Cancer. Thalidomide may interfere with the formation of new blood vessels (angiogenesis),
which tumors use to get nourishment to help them grow and spread. If thalidomide prevents the
formation of blood vessels to tumors, it could stop the growth and spread of some cancers.
Preliminary clinical studies have found that thalidomide, when combined with other drugs, may
show some promise in treating several types of cancers, including kidney (renal cell) cancer,
brain tumors, melanoma and myelofibrosis.

Thalidomide: Research advances in cancer and other conditions

Special procedures required to prevent pregnancy

If you and your doctor decide thalidomide is the right treatment for you, you will need to agree
to the terms of a restricted distribution program required by the FDA to prevent birth defects. As
part of this program, you will:

■Receive a packet of patient education materials

■Sign a consent form
■Use two forms of contraception and undergo frequent pregnancy testing if you're a woman
■Use a condom if you're a man

If you suspect you're pregnant, stop taking thalidomide and contact your doctor immediately.
Remember: No method of birth control is completely reliable except for avoiding sexual
intercourse.

Side effects other than birth defects

People taking thalidomide might also experience other side effects, such as:

■Peripheral neuropathy
■Blood clots
■Drowsiness
■Seizures
■Rash
■Dizziness

Take your medication exactly as prescribed by your doctor. Check with your doctor before
taking any other prescription or over-the-counter medications.
Creating a safer thalidomide

Drugs that work like thalidomide but have fewer side effects may one day be available.
Researchers are working on thalidomide analogs — drugs chemically similar to thalidomide.
Lenalidomide (Revlimid) is one such analog. This drug is approved for myelodysplastic
syndrome (with 5q- syndrome) and advanced multiple myeloma.

Talk to your doctor if you have concerns about thalidomide. Understanding thalidomide's
history, its risks and its potential benefits can help you decide if it's right for you.

© 1998-2009 Mayo Foundation for Medical Education and Research (MFMER). All rights
reserved. A single copy of these materials may be reprinted for noncommercial personal use
only. "Mayo," "Mayo Clinic," "MayoClinic.com," "EmbodyHealth," "Reliable tools for healthier
lives," "Enhance your life," and the triple-shield Mayo Clinic logo are trademarks of Mayo
Foundation for Medical Education and Research.

References
1. Rosenbach M, et al. Dermatologic therapeutics: thalidomide. A practical guide. Dermatologic Therapy.
2007;20:175.
2. Thalomid (prescribing information). Summit, N.J.: Celgene Corp.; 2007.
http://www.thalomid.com/pdf/Thalomid_Pl.pdf. Accessed Oct. 30, 2008.
3. Patient information sheet thalidomide (marketed as Thalomid). U.S. Food and Drug Administration Center
for Drug Evaluation and Research. http://www.fda.gov/cder/drug/InfoSheets/patient/thalidomidePIS.htm.
Accessed Oct. 30, 2008.
4. Woodcock J. Supervisory review of NDA 20-785. U.S. Food and Drug Administration Center for Drug
Evaluation and Research. http://www.fda.gov/cder/news/thalinfo/20785medr.htm. Accessed Oct. 30, 2008.
5. Label and approval history. Drugs@FDA.
http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?
fuseaction=Search.Label_ApprovalHistory#apphist. Accessed Oct. 30, 2008.
6. Melchert M, et al. The thalidomide saga. The International Journal of Biochemistry & Cell Biology.
2007;39:1489.
7. Revlimid (prescribing information). Summit, N.J.: Celgene Corp.; 2006.
http://www.fda.gov/cder/foi/label/2006/021880s001.pdf. Accessed Oct. 31, 2008.

© 1998-2009 Mayo Foundation for Medical Education and Research (MFMER). All rights
reserved. A single copy of these materials may be reprinted for noncommercial personal use
only. "Mayo," "Mayo Clinic," "MayoClinic.com," "EmbodyHealth," "Reliable tools for healthier
lives," "Enhance your life," and the triple-shield Mayo Clinic logo are trademarks of Mayo
Foundation for Medical Education and Research.
1. Circle the stereogenic center in each enantiomer.

2. Label each enantiomer as either S- or R-.