Two small Maryland companies reported encouraging results yesterday from early clinical trials using thalidomide, the sedative banned since the 1960s for causing birth defects, to treat cancers and an AIDs-related condition.
The results add further weight to a growing body of evidence that the drug may have potential in treating some of humans' most vexing diseases.
In one clinical study, co-sponsored by Andrulis Pharmaceuticals Inc. of Beltsville, researchers found that thalidomide dramatically reduced or eliminated AIDS-related mouth and throat ulcers.
Meanwhile, Rockville-based EntreMed Inc. reported promising early results from two small clinical trials in which patients with brain cancer or Kaposi's sarcoma were treated with thalidomide.
The results of the study on thalidomide's potential for treating mouth ulcers are being reported today in the New England Journal of Medicine.
"The results were dramatic, if not extraordinary," said Dr. Lawrence Fox, one of the authors of the article and a medical officer in the human immunodeficiency virus research branch at the National Institutes of Health in Bethesda.
Ulcers cleared up entirely in 55 percent of the people given it in the study, compared with just 7 percent in a group given a placebo. The study involved 67 HIV-infected patients.
Researchers found that just a month of treatment with thalidomide brought relief from the painful ulcers. The ulcers, usually treated with prednisone, make it difficult to eat, exacerbating the weight loss often seen in AIDS patients. In some cases, patients can't even swallow their own saliva, medical authorities say.
"This is a terrific potential therapy much better than anything now available," Fox said. "I'd have to say the chances are probably good the [Food and Drug Administration] will eventually approve this drug for this indication."
One unexpected downside the study found was that thalidomide elevated blood levels of the HIV virus.
Peter Andrulis, president and chief executive officer of the privately held Beltsville company which supplied the thalidomide, said, "The results were really striking but we were expecting that based on a number of anecdotal studies we've looked at."
He said his company plans to file a new drug application in the next 12 months to market thalidomide to treat canker sores in the mouths and throats of patients with HIV or AIDS.
As for EntreMed's trial results, the company said early results in brain cancer and Kaposi's sarcoma patients treated with thalidomide were encouraging.
A total of 32 patients with brain cancers were evaluated in the study. The study found that 50 percent had responses: 12 patients' disease stabilized; tumors in two were reduced by 50 percent; and tumors in two others were reduced by less than 50 percent.
In the study of Kaposi's sarcoma, a cancer that often strikes AIDS patients, researchers found that three of the five patients who received thalidomide achieved at least a 50 percent reduction in the number of skin lesions associated with the disease.
Dr. Howard A. Fine, a neuro-oncologist at the Dana-Farber Cancer Institute in Boston, said the results "clearly indicate the important role thalidomide could play" as a new cancer therapy.
Pub Date: 5/22/97