A Hated Drug Makes a Reappearance as a Potential Treatment for Mesothelioma

Saturday, September 24, 2005

The drug Thalidomide first appeared in Europe in the 1950's as a sleeping pill. It became a tragic example of how wrong things can go with a licensed drug. Thalidomide was also used for pregnant women with morning sickness. When the drug is taken in the 1st trimester of pregnancy the drug can cause very severe deformities. The drug can have an adverse effect on any part of the baby in development. Many babies were born with tiny paddle-like arms and legs or deaf, blind, without a lung, with heart and digestive tract deformities and with with cleft palates. The drug can kill a fetus and many women had stillborn babies.

In the late 1950's and early 1960's thousands of "Thalidomide babies" were born before the cause was identified and the drug was pulled from the market. Some estimates are as high as 10 to 12,000 babies were born with Thalidomide related birth defects. Today there are about 5000 survivors throughout the world.

In 1998 the FDA allowed the return of Thalidomide for the treatment of the disfiguring lesions of Hansen's Disease (Leprosy). Because of the devastating side effects, patients are closely monitored and receive extensive education on the risks of pregnancy.

This review discusses the history of Thalidomide as well as some of its off-label uses.

One of the mechanisms of action for Thalidomide is its anti-angiogenesis properties, that is, it inhibits the formation of new blood vessels. For a tumor to survive and grow it needs a blood supply.

Several studies have shown Thalidomide to inhibit tumor growth in Multiple Myeloma. Several trials are ongoing in the US and in Europe to investigate Thalidomide as a single agent and in combination with other drugs.

A article in the journal Lung Cancer in May 2005 describes a study done to evaluate toxicity and efficacy of Thalidomide with Malignant Mesothelioma. The study found that a daily 200 mg dose was well tolerated and routine monitoring for neurotoxicity could be avoided. In the study 27.5% (11 patients) showed disease stabilization for greater than 6 months. It may be genetic studies could further target patients who would respond to this treatment.

Despite Thalidomide's horrible track record, modern medicine has found some limited uses for it. The preliminary data on the treatment of Malignant Mesothelioma warrants further investigation.