The Bay Area Reporter - August 8, 1995
Matthew Sharp, ACT UP/Golden Gate Writers Pool

Through wider access, more people are now using thalidomide, and improvements are being seen: it is not a cure, but it evidently can promote quality of life in people with AIDS. Gilla Kaplan, a leading researcher at Rockefeller University in New York, is doing fascinating work on using thalidomide to lower the level of TNF (tumor necrosis actor, a normal body chemical that is often excessive in HIV disease, and can then cause wasting, and also make HIV grow faster). Her recent study, finally published in Molecular Medicine, shows that the drug reduces TNF alpha levels in patients with TB; half of those people were also infected with HIV.

The birth defect tragedy of 40 years ago changed the approval process for drugs at the Food and Drug Administration, the U.S. regulatory agency that oversees safety and efficacy for all new drugs being marketed. The implications of the effect drugs may have on unborn fetuses have influenced the way trials are run, and have unfairly caused exclusion of pregnant and non-pregnant women in most studies, regardless of precautions and screening procedures. Consequently, this issue has delayed important therapies like thalidomide for all people with AIDS, especially women.

In the absence of FDA approval for thalidomide, over 200 people are now using it underground: local physicians who are prescribing the drug are seeing definite weight gain in almost all those patients. Granted, this is anecdotal, but anecdotal among doctors.

Research - there is tons of literature on thalidomide - shows that the drug is relatively safe, although toxicity in people with AIDS is less clear. Additional AIDS studies underway will further add to our knowledge of its safety. Unfortunately, enormous media coverage and widespread panic has slowed progress in development of the drug against AIDS and other diseases today.

"As long as the drug is illegal it jeopardizes all of our positions for development," said Kaplan. "Until we enroll the clinical trials and finish the studies, compassionate use will never come about."

Clinical trials open

Clinical trials of thalidomide for aphthous ulcers, weight gain, and lowering tumor necrosis factor are having difficulty enrolling in the Bay Area and across the country. Some of the trials have limiting exclusion criteria and are placebo-controlled, but if you get drug, it is free. (For more information, call 1-800 TRIALS-A.)

These studies are an important venue for access and should concurrently provide researchers with important data on thalidomide, which the FDA is demanding before approval. Thalidomide is available for people with aphthous ulcers through an Emergency Release Program through the FDA - but somewhat ineffectively. Access had been problematic until buyers' clubs established an Underground Thalidomide Compassionate Use Program that provides drug to anyone who follows the carefully planned safety procedures.

Furthermore, ACT UP/Golden Gate is working with Healing Alternatives Foundation to monitor people on thalidomide, in order to gain important observational data that will help answer critical question on toxicities and weight gain. (This is the second Observational Data Base that ACT UP/Golden Gate has designed; the first was on d4T use.)

Recently AIDS activists met with FDA officials in Rockville, Maryland in a meeting called by the PWA Health Group in New York and Healing Alternatives from San Francisco, to alleviate the feds' concerns about underground thalidomide usage and to discuss the underground supply, procedures, and policies. The goals of the meeting, activists said, were to urge the FDA to be realistic about the potential of thalidomide and to push for compassionate use against wasting syndrome.

It was clear from the beginning of the meeting where the FDA stands: their opening statement was concerning unborn fetuses, and not access for people with AIDS.

Eventually, to stress the importance of access to thalidomide outside of placebo-controlled clinical trials, Curtis Ponzi, an attorney present on behalf of the buyers' clubs, held up the B.A.R. obituary section and said, "This is our control group."

Off-label, post-market

With all the research to be done on thalidomide, it would seem logical that the way to have access to it is through pharmacies, but given the paranoia and illegality of the drug, that may take a long time. Brenda Lein from Project Inform stated, "I'm hopeful that an NDA [New Drug Application] can be made for aphthous ulcers with expedited approval, because there is so much data in this area. This would effectively address access for people who could use the drug for weight gain by acquiring the drug off-label.

"However, if this course of action is to be taken, Andrulis [the company that makes thalidomide in the U.S.] must guarantee post-marketing studies, to ensure that the research gets done."

A few caveats: because there are women with AIDS who may want a shot at thalidomide therapy, we must acknowledge that birth defects are a very serious issue. But with appropriate labeling and warnings thalidomide could safely be prescribed like the acne medication, Acutane, which also can cause birth defects.

There are also hints that neuropathy may be a problem for thalidomide users, as well as rashes and the masking of serious infections like tuberculosis and MAC. But even with these risks in mind, given proper vigilant monitoring, thalidomide is probably no more dangerous than other drugs.

For a while at least, media hype, regulatory roadblocks, and superstitions will prevent access to many people who need thalidomide. This is sad, but somewhat typical, in the history of AIDS drug development.
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