Nov. 17, 1999 (Seattle) -- Thalidomide, a drug best known for causing horrific birth defects, is prolonging the lives of people with a rare form of cancer, according to a study in TheNew England Journal of Medicine Thursday.
The study, conducted by a team at the University of Arkansas for Medical Sciences in Little Rock, found that thalidomide helped a third of people with advanced multiple myeloma, an incurable cancer of the bone marrow. Most of the people in the Arkansas study had exhausted all other treatment options before they got the drug.
One of the researchers, Bart Barlogie, MD, PhD, tells WebMD that the results represent "a milestone in therapy for myeloma." Barlogie, an internationally known expert on the disease, says the prospect of a new weapon to fight it is exciting because doctors have been using the same two drugs for about 40 years.
Multiple myeloma strikes nearly 14,000 people a year in the U.S. More than 70% of them die within five years. Thalidomide is a drug that was widely used in Europe to prevent morning sickness in pregnant women. It was pulled off the market after reports that it led to severe birth defects, including children with flipper-like arms and legs. In recent years, though, thalidomide has made a comeback as a treatment for leprosy.
In the new study, researchers gave increasing doses of thalidomide to 84 people with multiple myeloma who were failing other treatments. Most were getting sicker despite having received the highest possible doses of chemotherapy -- usually the final treatment option.
But despite the advanced stage of their disease, about one-third of the patients got better with thalidomide. Tests that indicate the presence of cancerous cells in the bone marrow showed dramatic reductions for about 10% of the patients, including two who had a complete remission. Barlogie says it's not clear yet how long the benefits last.
In an editorial about the study, two cancer researchers from Harvard Medical School called the results "remarkable." One of the researchers, Kenneth Anderson, MD, tells WebMD that thalidomide is generating huge excitement in the medical community because multiple myeloma has proven so difficult to treat.
Anderson says the results of this study are particularly striking because the study included people who were no longer responding to any other treatment. "It's likely to be even more effective with people in an earlier stage of the disease," he says.
But scientists say that they still need to determine the ideal dose of thalidomide, which in large amounts can cause side effects including constipation, sleepiness, and fatigue. They also hope to learn how the drug works against myeloma.
"We are in the midst of examining several different mechanisms," says Barlogie. One involves the drug's ability to prevent the formation of new blood vessels, he says, which help cancers grow. Barlogie says it's also possible that thalidomide helps the body's immune system attack myeloma cells.
- Multiple myeloma is a rare, incurable cancer of the bone marrow, striking 14,000 Americans each year.
- In a study of people in advanced stages of the disease who were not responding to traditional therapy, the drug thalidomide improved the condition of one-third of the patients.
- Researchers suspect the drug will be even more effective for patients in early stages of the disease, but further research is necessary.