San Francisco Chronicle - Tuesday, June 27, 1989
Randy Shilts, National Correspondent

Organizers say their goal is to not only discover whether Compound Q is effective but to establish that the testing of such drugs can be conducted much faster than is currently done within the orthodox drug testing system. The so-called Compound Q is widely considered one of the most promising AIDS drugs ever tested because of unique HIV-killing properties it has demonstrated in the test tube.

"We want to demonstrate quite clearly that these studies can be done rapidly and that they don't need to go through the unconscionable delays we're still seeing," said Martin Delaney, the major organizer of the study. "I think we already have made that point," said Delaney, who is co-director of Project Inform, a San Francisco group that provides information on AIDS treatments. "We've already got several times as many people on the drug than the government has done, even with all its resources."

TWO HAVE DIED

Of 14 volunteers who enlisted in the study in San Francisco, two have died, said Delaney. One committed suicide for reasons unrelated to his involvement in the research, Delaney said. A 45-year-old San Francisco man suffered toxic side effects from the drug, but died on Saturday from causes that appeared unrelated to the drug, organizers said. Organizers say it is too early in the planned three-month study to draw any conclusions as to the drug's effectiveness.

The study started five weeks ago in San Francisco when private doctors administered to five volunteers the first injections of the protein trichosanthin, which had been smuggled in from China. The trichosanthin molecule is the active ingredient in the drug GLQ 223, which, in research at the University of California at San Francisco, selectively killed off HIV-infected immune cells while not affecting healthy blood cells.

POTENTIAL CURE?

If the drug, which is commonly called Compound Q, is as effective in people as it is in the test tube, researchers say it could potentially act as a virtual cure for the disease. However, some other drugs that were effective in the test tube later proved extremely toxic in people.

A UCSF-developed version of the drug recently started early clinical trials at San Francisco General Hospital to study possible toxic side effects.

Worried by official pronouncements that this early study could take as long as nine months, local AIDS organizers, coordinated by Delaney, began piecing together their own plan for an expedited study. "If we had waited that long, we would have seen supplies of trichosanthin starting to flood the underground and we'd have thousands of people taking this before we knew anything about whether it worked or its possible side effects," said Delaney last night in a telephone interview from Washington, where he was attending a meeting on AIDS drug development issues at the National Academy of Sciences. "It would be chaos to take the time to go through normal channels," he said.

FROM SHANGHAI

After obtaining a purified version of trichosanthin from sources in Shanghai, Delaney contacted attorneys and physicians to work out a plan for a study that would not violate food and drug laws but still provide data that would be scientifically rigorous enough to meet the strict demands of government regulatory authorities. "The protocol we put together is much stricter and scientifically more precise than an awful lot you would see coming out of the National Institutes of Health," Delaney said. "Doing fast science doesn't mean that it can't be done every bit as rigorously - it just means that you actually get the study written and you get it done."

Attorney Curtis Ponzi, a regulatory law specialist who advised a doctor working on the study, said last night that while a researcher who wants to conduct a study on a new drug must get approval from the Food and Drug Administration, doctors are allowed to monitor patients who chose to import their own drugs from other countries. BY THE BOOK

Patients were supplied their own drug by study organizers and then monitored by physicians who administered extensive physicals and blood tests. Ponzi developed a lengthy informed consent questionnaire that was read and signed by patients during a video-taped session. Since the study began, Delaney said, 15 volunteers in Los Angeles have started the three-shot regimen, as well as AIDS sufferers in Miami and New York City.

Patients in the San Francisco arm of the study were being monitored by Dr. Alan Levin, who has a large private practice of HIV-infected patients. San Francisco volunteer Robert Pitman, 48, said the drug trial represented an opportunity to take trichosanthin. "Even before I knew about this, I had already decided that one way or another, I was going to get this drug," said Pitman, who was a job counselor for teenagers before his AIDS diagnosis last year. "I was not going to wait the 1 1/2 or two years before it was generally available," he said. For the past two years, Pitman said he had watched his immune system disintegrating, even as he took AZT and other approved treatments.

FIRST INJECTION

Pitman took his first injection nearly two weeks ago and experienced what Delaney describes as typical side effects of fevers and muscle aches. A few days later, however, he said, "I felt better than I had in two years. I felt like a million bucks." Pitman took his second injection last Wednesday and is slated to take his third and final shot in the study tomorrow.

The drug appears to have more severe toxicities for AIDS patients suffering from dementia or severe neurological complications of the disease, Delaney said. One such San Francisco man slipped into a coma last week after his first injection of the drug. He came out of the coma on Friday and appeared to be regaining his health, Delaney said, but later that night he choked on his own vomit and died at Mount Zion Hospital.

"It clearly was not a direct effect of Compound Q," he said. "Everybody regrets that death. What's just as noteworthy is the 138 people who typically die every day from AIDS in the United States because there are no treatments. That's part of this tragedy too." No spokesman for the Food and Drug Administration was available last night to comment on the study.

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