A Wasting of Time

An existing drug offers hope for AIDS wasting syndrome -- if you can get it

He never even noticed the weight loss, former ballet dancer Matthew Sharp says. Every day he'd look in the mirror and see the same lithe lines, not realizing that he was slowly turning to skin and bones. It took his friends to tell him that he might be starving to death.

"I was diagnosed with AIDS wasting," says Sharp. Wasting is a late-stage AIDS malady, a sign that the body has started to consume its own muscles in a misguided effort to stay alive. It is almost always fatal. Yet Sharp's diagnosis occurred more than two years ago. Now, thanks to a controversial AIDS drug, Sharp says he is back in shape.

"I was eating again. I had an appetite again, which I didn't have before. I was sleeping well. I had boundless energy," he says. "I even went back to ballet class and did a performance. I used to be a ballet dancer. All in all, I regained my life."

Sharp says he was saved from the perils of AIDS wasting by the so-called human growth hormone. It is not a new drug. More than a decade ago, South San Francisco's Genentech won Food and Drug Administration (FDA) approval to manufacture human growth hormone for treating short-statured young children. In fact, human growth hormone was the first bioengineered hormone ever brought to market, and it marked a brave first step for the then-nascent biotech industry.

But despite the concerted efforts of San Francisco AIDS activists, human growth hormone has remained mostly beyond the reach of those suffering from AIDS wasting. The drug costs $150 a day -- or about $54,000 a year. And Serono, the drug company that has proposed marketing the hormone as a treatment for AIDS, has been unable to win FDA approval to bring the drug to market for that purpose. Its most recent attempt, in March, resulted in a rare FDA split vote, as an advisory committee ruled 8-7 against Serono, saying the company had not proved that the hormone was both safe and effective as an AIDS treatment.

Now, however, the FDA is on the brink of granting accelerated approval for the hormone.

"I think we are very close. We're coming down to the wire now," says Gina Cella, director of community relations for Serono. "We've had many discussions with the FDA. We've provided them with new, additional data."

For its part, Serono has apparently agreed to exchange full approval, which it initially wanted, for accelerated approval, which comes with some strings attached. That's essentially what the AIDS activist community has been pushing for: access to the drug while tests continue, rather than waiting until all the results are in, a process that has already stretched on for eight years.

"Just very recently, like within the last day, Serono has agreed to apply for and to accept accelerated approval if it's offered," says Ron Baker, director of treatment education at the San Francisco AIDS Foundation, and who testified on behalf of the drug's approval before the FDA in March. "That will grant approval to a drug more quickly than under normal circumstances, but it comes along with some restrictions."

"Really we want to give this drug the best opportunity for approval," Cella says.

"We've been hearing rumors that any day now they're going to be reaching approval," says Sharp, a member of ACT UP Golden Gate and director of Healing Alternatives, the largest AIDS resource library in the nation. "We've come a long way, and it's a big victory for ACT UP Golden Gate and for a lot of people."

The road toward FDA approval for the hormone as an AIDS wasting treatment has been rocky indeed. AIDS wasting is a specific, late-stage syndrome marked by the body's erroneous decision to consume proteins instead of fats for energy. The body thus strips itself of muscle and cannot process food properly, resulting in starvation and the marked loss of weight. Reversing AIDS wasting means more than just gaining weight -- the body must begin to process nutrition properly again, and actually add lean body mass, or muscle.

In 1992, Genentech conducted trials of the hormone as an AIDS wasting treatment and then abandoned the idea of using human growth hormone as an AIDS drug. The Genentech trials were Phase I and Phase II tests, which are both relatively preliminary levels of the FDA-required process to bring a drug to market. The final pre-market test of a drug is called a Phase III trial, and successful results in that phase are required for FDA approval. Genentech's tests didn't get that far.

"We found no evidence of efficacy," Genentech Corporate Communications Officer Kathleen Rinehart says.

That's the void Serono entered. Serono, which is the Massachusetts-based U.S. subsidiary of Swiss drug company Ares-Serono, manufactures and distributes the hormone in 60 countries worldwide. But in trying to comply with the FDA requirements, Serono fell short, proving conclusively in one crucial trial only that people gained weight, rather than that the weight they gained was measurable lean body mass.

"It was supposed to confirm the drug safety, and they looked at weight gain, but they did not look at lean body mass," says the AIDS Foundation's Baker. "This was an inexcusable oversight on the part of Serono, not to have measured lean body mass in this Phase III trial."

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