May 21, 2000 (New Orleans) -- Thalidomide, a drug notorious for producing birth defects in babies born to women who took it for morning sickness in the 1960s, is proving to be effective for people with advanced multiple myeloma, a cancer of the bone marrow. However, thalidomide is not currently approved for this use by the FDA.
"I am very encouraged by these results," Bert Barlogie, MD, tells WebMD. "In patients with advanced disease who have failed all other types of therapies, thalidomide is able to cause their disease to go into remission in about one-third. These results are stunning as well as highly unusual." Barlogie is a professor of medicine and pathology at the University of Arkansas at Little Rock.
More than 14,000 new cases of multiple myeloma are diagnosed each year. The disease occurs when a particular type of cancerous immune system cell overtakes the bone marrow. Symptoms include bone pain, anemia, and multiple infections due to the lack of functioning immune system cells. The cancer cells produce an abundance of a protein that is actually the cause behind some of the complications of the cancer, such as kidney failure.
In April, FDA sent out a warning letter to Celgene, the manufacturer of Thalomid capsules brand of thalidomide, advising the company not to market the drug as an anticancer agent. In response to that, Barlogie says, "Our studies have been conducted as part of a National Cancer Institute project grant and have nothing to do with using thalidomide willy-nilly. I think we have found that thalidomide works in advanced stages of myeloma." Thalidomide was not available in the U.S. at the time it was found to be associated with severe birth defects and was only approved for use in the U.S. in 1998 when the FDA allowed Celgene to market the drug for patients with a serious skin disorder.
Barlogie and colleagues reported the results of a study using thalidomide in more than 150 people with advanced multiple myeloma at the annual meeting of the American Society of Clinical Oncology. The drug was well tolerated and significantly decreased the amount of the protein produced by the cancerous cells. "We are so encouraged by our results that we have started several other clinical trials using thalidomide," says Barlogie. "The question now has become when is it most appropriate to start using thalidomide in the treatment of multiple myeloma?"
"Thalidomide is an exciting new treatment for multiple myeloma," says independent reviewer and oncologist Steven Paulson, MD. "My personal experience with my patients is consistent with that of Dr. Barlogie's, with about one-third responding to treatment with disease remission. While we know that thalidomide is risky for some patients, it's highly unlikely that any of our patients would become pregnant." Paulson, who was not involved in the study, is associate professor of medicine at the University of Texas Southwestern Medical School, in Dallas.
In response to FDA's letter to Celgene, Paulson says, "I think most oncologists are guilty of wanting to do the best thing for their patients, even if that means using a potentially off-label medicine that might be helpful. Furthermore, most of these patients are desperate for treatment, and if we can offer a therapy that might work, especially when the risks seem minimal, then I think we will do that."