Cancer Current Topics in Cancer

Once-Condemned Drug May Control Blood Cancer

A drug once denounced for causing birth defects seems to be a promising tool in the fight against a form of blood cancer,

In a late-stage study involving 270 patients, researchers looked at whether the controversial drug, thalidomide, would create a better treatment combination for multiple myeloma when taken with dexamethasone, a commonly used treatment for this cancer.

Thalidomide raised a storm of controversy after it caused serious birth defects in the babies of pregnant women taking the drug to relieve morning sickness. The drug was subsequently withdrawn from the market in the early 1960s, and only recently was it approved in the United States for use in treating a symptom of leprosy.

In this current study, thalidomide was so successful at slowing the cancer, the team stopped the study early and offered the drug to those who were only taking a placebo with dexamethasone.

In fact, the thalomide-dexamethasone combination held the cancer in check for more than 75 weeks on average, as compared to fewer than 30 weeks in patients treated with dexamethasone alone.

"We believe that this data demonstrated the significant benefit that thalidomide provides to patients with newly-diagnosed multiple myeloma," said Patrick J. Mahaffy, president of Pharmion Corporation, the company helping to develop the drug.

Currently affecting over 50,000 people in the United States, multiple myeloma is a blood cancer that causes the overproduction of plasma cells, a type of white blood cell. Left untreated, the disease can cause anemia, kidney damage, increased susceptibility to infection and osteoporosis. There is no cure, but with the right treatment it can be managed.

While thalidomide is often prescribed off-label for the management of multiple myeloma, the Food and Drug Administration is now reviewing the results of the study to consider approving this drug specifically for the treatment of this cancer. Thalidomide is already approved for this use in Australia, Israel, New Zealand and Turkey.

The most common side effects reported by study participants were insomnia, tremors and dizziness. However, ten percent of the patients taking thalidomide suffered from deep vein thrombosis, a disease that causes dangerous blood clots to form. In contrast, fewer than two percent of patients taking just dexamethasone had this problem.

And while thalidomide does still carry a serious risk of birth defects, both Celgene, the company that manufactures thalidomide, and the FDA have agreed on guidelines to ensure that patients do not become pregnant while taking the drug. This program requires all women taking the drug to use two forms of contraception, and men must use latex condoms if they have sex. All patients must undergo monthly reviews, including pregnancy tests for women, before they can receive a new prescription for the drug.

"It's a check and balance system," said Christy Celli, medical information specialist for Celgene.