Thalidomide, the drug infamous for causing ghastly birth defects during the 1960s, appears to be effective against a lethal form of bone cancer, even in patients in advanced stages of the disease.
A study of 84 patients with multiple myeloma found that all signs of the disease disappeared in 10 percent of those getting thalidomide, and most of the others improved somewhat."These were tough patients for whom there is no standard treatment approach available," said study co-author Dr. Bart Barlogie of the University of Arkansas Cancer Research Center. "Thalidomide is the first new drug that has shown activity in multiple myeloma in the past 30-plus years."
While never approved in the United States, thalidomide was given to pregnant women in other countries for morning sickness. It was banned worldwide after causing 12,000 babies to be born with serious birth defects, including flipper-like arms. The drug became available in the United States for the first time last year as a treatment for leprosy.
Thalidomide is undergoing several studies as a possible cancer medicine. The new study in New England Journal of Medicine, offers some of the strongest evidence yet of its effectiveness.
Barlogie said researchers are not sure precisely what makes thalidomide work, but it apparently cuts off the blood supply to tumors.
Multiple myeloma, a bone marrow cancer that kills about 11,000 people in the United States each year, is considered incurable with conventional chemotherapy. The disease usually strikes the elderly, and with traditional treatment, they generally survive 2 1/2 to 3 years. When combined with a bone marrow transplant, chemotherapy can prolong life somewhat.
The new study showed that almost one-third of the patients studied had a positive response to thalidomide, and 10 percent experienced a complete or near-complete remission.
A positive response was defined as a reduction in the number of abnormal protein cells in the blood or urine. While complete remission means no evidence of the cancer remains, it does not necessarily mean patients are cured.
Before the study, 76 of participants had suffered a relapse after chemotherapy, and all were in later stages of the disease.
The patients received oral doses of thalidomide for 80 days. They started with 200 milligrams, and the dosage was increased to 800 milligrams per day.
Thirty-two percent of the patients responded to the treatment, with decreases in abnormal protein cells ranging from 25 percent to 90 percent in some people. After a year, 48 patients were still alive.
At least one-third of the patients suffered side effects such as constipation, weakness, fatigue, drowsiness and neurological complications. However, in most cases, a reduction in the dosage eased the problems.
Celgene Corp., the only company that sells thalidomide in the United States, hopes to gain federal approval to use it for cancer treatment.
Dr. Michael Gruber, director of neuro-oncology at New York University Medical Center, said the study adds to a growing body of evidence supporting use of the drug for some specific purposes.
"I think it's extremely exciting. These were essentially terminal patients who had reached the end of all other forms of treatment," he said.
The drug's proponents say it has shown promise in experimental treatments for other forms of cancer, wasting disease and ulcers in AIDS patients, rheumatoid arthritis, lupus, tuberculosis and other diseases.
In an accompanying editorial, Dr. Noopur Raje and Kenneth Anderson of the Dana-Farber Cancer Institute in Boston, said thalidomide opens "possibilities for novel treatments."
"Given that the patients in the study had relapsed after chemotherapy, which was usually given in massive doses, this effect of thalidomide is indeed remarkable," they wrote.