Thalidomide: Research advances in cancer and other conditions

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Thalidomide: Research advances in cancer and other conditions

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.

Special procedures required to prevent pregnancy

If you and your doctor decide thalidomide is appropriate 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.

Last Updated: 2010-12-18
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