SATURDAY, July 3, 2004 (HealthDayNews) -- The rehabilitation of thalidomide continues with two preliminary studies showing it may have a place in the treatment of multiple myeloma and melanoma.
The drug first achieved notoriety in the 1960s when it was pulled from the market after it was found to cause birth defects in the babies of women who had been taking it for morning sickness.
Thalidomide was reintroduced to the market, with stringent birth-control restrictions, as a treatment for leprosy in 1998.
At the recent meeting of the American Society of Clinical Oncology (ASCO) in New Orleans, two sets of researchers showed it may have some benefit in patients with multiple myeloma, a type of blood cancer, and melanoma.
In the multiple myeloma trial, Italian researchers found thalidomide, combined with melphalan and prednisone in a Phase II clinical trial, elicited a better response than conventional chemotherapy. After being treated with the three drugs, 93 percent of participants had myeloma protein reductions of greater than 50 percent. Moreover, 26 percent had a complete remission, while near-complete remissions were seen in 19 percent. These response rates are similar to those observed in those who have had bone marrow transplants.
Though the response rate was promising, thalidomide therapy came with some unpleasant and dangerous side effects. In the Italian study, for instance, 26 percent of the 42 patients got infections, 19 percent were treated for deadly blood clots, 14 percent developed an unusually low level of infection-fighting white blood cells, and 28 percent were constipated. Two patients died during the study, and 36 percent discontinued thalidomide therapy because of the side effects.
Still, "it appears that thalidomide is useful when added to standard chemotherapy in newly diagnosed multiple myeloma patients," said Alan Kinniburgh, vice president of research at the Leukemia & Lymphoma Society. "The real take-home message is that this isn't a cure for myeloma."
Kinniburgh said the side effects aren't as serious as they are with some other cancer drugs such as Velcade, so it "is another weapon to treat myeloma that can help the patient live longer."
New treatments are desperately needed for multiple myeloma, the second most common blood cancer in the United States. "The five-year survival rate is under 30 percent," Kinniburgh said. "There is no cure. It is a clearly fatal disease."
There may be more hope in combining thalidomide with the drug Velcade, Kinniburgh added. Those studies are under way.
Thalidomide is under review by the U.S. Food and Drug Administration (FDA) for this indication. "It's another drug in the arsenal and, hopefully, with some of the clinical trials ongoing, physicians will find out how to use it with other new drugs," Kinniburgh said. "Velcade comes to mind, and it may perhaps be useful in the post-transplant setting as well. Time will tell."
The second trial looked at thalidomide in combination with another cancer drug, temozolomide, for metastatic melanoma in people who were currently free of the disease but were at a high risk for recurrence. Melanoma is a particularly virulent form of skin cancer.
The study, done at Memorial Sloan-Kettering Cancer Center in New York City, is still ongoing, but the researcher, Dr. Wen-Jen Hwu, a physician at the cancer center, said they were achieving "excellent results."
Both studies were sponsored by Celgene Corp., which makes thalidomide.
Thalidomide may also have benefit for patients with metastatic prostate cancer.
In a phase II randomized trial conducted by the National Cancer Institute, 75 patients were randomly assigned to receive either docetaxel (a chemotherapy drug) on its own or docetaxel plus thalidomide.
After a median follow-up of just over two years, those in the docetaxel-plus-thalidomide group had a median progression-free survival of 5.9 months vs. 3.7 months in the docetaxel group. At 18 months, overall survival in the docetaxel/thalidomide group was 68.2 percent, compared to 42.9 percent in the docetaxel group. The regimen was well tolerated after the patients received heparin, a blood thinner.
The results appeared in the July 1 issue of the Journal of Clinical Oncology.
Hwu is involved in yet another study looking at the promise of thalidomide in patients whose melanoma has spread to the brain. She said she has found "efficacy and increased survival time."
The U.S. Food and Drug Administration has more on thalidomide.