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

In the 1950s and the early 1960s, thalidomide was used to treat morning sickness during pregnancy. But it was found to cause severe birth defects.

Now, decades later, thalidomide is being used to treat a skin condition and cancer. 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 may be an effective treatment for several conditions. The Food and Drug Administration (FDA) has approved thalidomide (Thalomid) for treating:

  • Skin lesions caused by leprosy (erythema nodosum leprosum)
  • Multiple myeloma

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 that affect the skin, such as cutaneous lupus and Behcet's disease
  • HIV-related mouth and throat ulcers, as well as HIV-related weight loss and body wasting
  • Cancer, including blood and bone marrow cancers, such as leukemia and myelofibrosis, as well as cancers found elsewhere in the body

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.

To prevent pregnancy while you're taking thalidomide, you'll:

  • 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
  • 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 drugs chemically similar to thalidomide (thalidomide analogs).

Thalidomide analogs include:

  • Lenalidomide (Revlimid), which is approved for treating myelodysplastic syndrome (with 5q- syndrome), multiple myeloma and mantle cell lymphoma
  • Pomalidomide (Pomalyst), which is approved for treating 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.

May 25, 2019 See more In-depth

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