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Thalidomide: Research advances in cancer and other conditions

Despite its history, thalidomide has proved effective in treating some diseases. Consider the benefits and risks of thalidomide to help you decide whether this drug may be right for you.

By Mayo Clinic staff

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In the 1950s 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, such as shortened or missing arms or legs.

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 proves useful for skin lesions and multiple myeloma

Research into potential uses for thalidomide has determined that thalidomide is an effective treatment for erythema nodosum leprosum — skin lesions caused by leprosy. The FDA has approved thalidomide (Thalomid) for this use.

Thalidomide has also proved useful in the treatment of multiple myeloma — a blood and bone marrow cancer. The FDA has 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, thalidomide has shown promise in treating:

  • Inflammatory diseases. Thalidomide reduces the production of certain chemicals cells make that cause inflammation. Lowering the levels of these inflammatory chemicals may help people with inflammatory conditions, such as skin diseases, lupus and rheumatoid arthritis.
  • 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.
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References
  1. Cook KM, et al. Angiogenesis inhibitors: Current strategies and future prospects. CA: A Cancer Journal for Clinicians. 2010;60:222.
  2. Thalomid (prescribing information). Summit, N.J.: Celgene Corp.; 2006. http://www.accessdata.fda.gov/drugsatfda_docs/label/2006/021430s000,020785s031lbl.pdf. Accessed Nov. 1, 2010.
  3. Chen M, et al. Innovative uses of thalidomide. Dermatologic Clinics. 2010;28:577.
  4. Prommer EE. Palliative oncology: Thalidomide. American Journal of Hospice & Palliative Medicine. 2010;27:198.
  5. Szekanecz Z, et al. New insights in synovial angiogenesis. Joint Bone Spine. 2010;77:13.
  6. Revlimid (prescribing information). Summit, N.J.: Celgene Corp.; 2010. http://www.accessdata.fda.gov/drugsatfda_docs/label/2010/021880s018lbl.pdf. Accessed Nov. 1, 2010.
HQ01507 Dec. 18, 2010

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