Clinical Research Finds New Uses for Thalidomide: Banned Drug Can Help AIDS Patients
Price, Joyce, The Washington Times (Washington, DC)
Thalidomide, the drug banned worldwide in 1961 for causing severe birth defects, is making a dramatic medical comeback - despite continuing concern about its side effects.
A new study that shows thalidomide heals aphthous ulcers - painful, open sores in the mouths and throats of AIDS patients - is just the latest evidence that this highly toxic medication may be an effective remedy for many serious disorders.
"If data from clinical trials are convincing, at some point in time thalidomide would be approved for serious life-threatening diseases," said Dr. Debra Birnkrant, a senior medical officer at the Food and Drug Administration and chairman of the agency's Thalidomide Working Group.
The drug's terrible reputation stems from its role in causing gross congenital deformities in more than 10,000 babies born between 1959 and 1962 - most of them in Canada, England and Germany.
The deformed babies - many with shortened limbs that resembled seal flippers - were born to women who had taken thalidomide as a sleeping pill or for morning sickness early in pregnancy.
Today, the same medication shows promise as a therapy for more than a dozen serious and potentially deadly diseases including breast cancer, brain cancer, prostate cancer, multiple sclerosis, tuberculosis, lupus, rheumatoid arthritis, and various AIDS-related illnesses.
"There is a lot of positive initial data with thalidomide" that involve a wide variety of diseases, said Sol Barer, president of Celgene Corp. of Warren, N.J., which manufactures thalidomide. The drug has never been approved for marketing in the United States.
Celgene has applied for FDA approval of thalidomide as a treatment for erythema nodosum leprosum, an inflammatory condition in leprosy patients. Later this year, the company plans to seek FDA approval to market thalidomide as a therapy to prevent weight loss in AIDS patients.
Another firm, Andrulis Pharmaceuticals of Beltsville, said it will seek FDA approval this year to market thalidomide for treatment of aphthous ulcers in AIDS patients.
Peter Andrulis, the president of Andrulis Pharmaceuticals, which also manufactures thalidomide and is sponsoring some drug trials, acknowledged some research findings are "very preliminary."
"But we are seeing some effects in clinical or chemical response in Crohn's disease [a chronic inflammation of the intestine]; in rheumatoid arthritis; and in Kaposi's sarcoma [a skin cancer] and prurigo nodularis, a rare dermatological disorder, in AIDS patients," he said.
"Here's a treatment that could be used for a variety of diseases for which there is currently no treatment," said Dr. Barer.
He said thalidomide has been shown to suppress overproduction of tumor necrosis factor-alpha, a potent hormone that's part of the body's defense system. Many of the diseases where thalidomide is showing promise are "linked to excess TNF-alpha" production.
Although the drug is not approved for marketing in this country, the FDA's Dr. Birnkrant noted that approximately 1,000 Americans are already using it, either on a "compassionate-use" basis authorized by the FDA or in clinical trials.
Most of those people, she said, are battling AIDS.
Dr. Lawrence Fox of the HIV research branch at the National Institute of Allergy and Infectious Disease, warned that thalidomide should only be used "when administered by a physician who is vigilant" because of the "possible serious side effects, including irreversible painful peripheral nerve damage, rash and birth defects."