Thalidomide: Research advances in cancer and other conditions

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

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 also has 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 weight loss and body wasting.
• Cancer. Thalidomide may interfere with the formation of new blood vessels (angiogenesis). Tumors need nourishment from blood vessels to 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.

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 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. The most serious is peripheral neuropathy, a type of nerve damage. In addition, life-threatening blood clots can occur when thalidomide is used in combination with other medicines, such as steroids.

Less serious side effects include:

• Drowsiness
• Rash
• Dizziness
• Constipation
• Headache
• Nausea
• Dry mouth

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.