Thalidomide Continues Its Comeback Against Multiple Myeloma
The drug thalidomide continues to show promise as a treatment for the resilient blood cancer called multiple myeloma, researchers said Tuesday at the 2000 American Society of Clinical Oncology annual meeting. Their results confirm a smaller study published last fall and lead the way to a large, definitive trial of the drug.
In the study conducted at the Arkansas Cancer Research Center, Little Rock, researchers gave thalidomide to 169 patients whose cancer had not responded to chemotherapy or bone marrow transplants. After 18 months, 55 percent of patients were still alive, a result that lead researcher Bart Barlogie, M.D., called "stunning." Patients in a low-risk category, who did not have a specific defect in chromosome 13, showed an even higher 18-month survival rate of 77 percent. [Editor's note: The final results from this trial were subsequently published in the July 15, 2001, issue of Blood; see the journal abstract.)
Multiple myeloma is characterized by a proliferation of white blood cells called plasma cells. This flood of errant cells crowds out healthy blood cells in the bone marrow, leading to fatigue, bone pain, anemia, kidney failure, and recurrent infections. Bone tumors are also common. More than 14,000 cases are diagnosed each year in the United States.
The Arkansas researchers tracked thalidomide's action by measuring the levels of a molecule called paraprotein, which abounds in people with multiple myeloma. Twelve percent of the patients had a complete reduction in paraprotein, another 20 percent had a three-quarters reduction in the protein, and nearly all patients showed some reduction.
Side effects included constipation, weakness, sleepiness, and numbness, but 90 percent of patients tolerated the 400 milligram per day dosage.
The study did not directly compare thalidomide to standard treatments, the gold standard for any new cancer therapy. Standard treatments yield a five-year survival rate of 29 percent, a figure that Barlogie is hopeful thalidomide can exceed.
"Of all the cancers examined to date with thalidomide, multiple myeloma shows the greatest reported action," said Barlogie. "The question now is: Is it better to use the drug alone or in combination therapy?" Barlogie's research team is studying that question in a clinical trial that has already enrolled 125 patients.
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