PETER JENNINGS: [voice-over] San Francisco, May 23, 1989.

MARTIN DELANEY, AIDS Activist, Project Inform: What I have in here is the envelope for each of you, containing the trichosanthin that's come from China. Project Inform is not the supplier of this, but I am acting as the courier to get it to you guys from the people who brought it into the country.

JENNINGS: [voice-over] An AIDS activist distributed a new experimental drug. He did so outside the doctor's office to get around the law that prohibits a doctor from supplying a drug to these men for a clinical trial.

Mr. DELANEY: This is not an FDA-approved clinical trial. The protocol did not go to FDA in advance. It was not approved by traditional institutional review boards.

JENNINGS: [voice-over] Martin Delaney is one of the most influential AIDS activists in the country. For years, he's been challenging the Food and Drug Administration to speed up its drug trial procedures.

Mr. DELANEY: But I think it's been shown time and again in AIDS, when you've got a crisis, a national emergency going on, you have to take a shorter path to your goal.

DEMONSTRATORS: [chanting] No more dying! No more dying!

JENNINGS: [voice-over] For years, activists have been demanding that the agency loosen up its regulations and allow access to experimental drugs, anything that might prolong life. One of the drugs they wanted was this one: trichosanthin, better known as Compound Q, the Chinese abortion and cancer drug. It was tantalizing because it killed HIV-infected cells in the test tube. The FDA promised human trials, but for activists like Martin Delaney, trials administered by the FDA would take too long. So Delaney joined forces with Dr. Alan Levin, a San Francisco immunologist who was prepared to conduct an accelerated trial illegally.

Dr. ALAN LEVIN, Immunologist: Now, it's not a simple treatment and it's not a safe treatment. And the odds are very good someone's going to die of this treatment. I mean, we're not kidding ourselves, this is not just a magical panacea.

JENNINGS: [voice-over] Dr. Levin was prepared to put his reputation on the line because many people had imported it from China and were taking unsupervised doses. Levin asked for volunteers from among his own patients. He and his staff chose three who had advanced cases of AIDS--

CLARK HUSEMAN, Physician's Assistant: Do you understand that Dr. Levin does not guarantee that trichosanthin treatment will slow down or stop your AIDS?

TANDY BELEW, AIDS Patient: Yes, I do.

JENNINGS: [voice-over] -- one of them, 41-year-old Tandy Belew.

Mr. HUSEMAN: Do you understand as well that this is not necessarily a cure for AIDS?

Mr. BELEW: I do.

Mr. HUSEMAN: Okay.

Mr. BELEW: I'm in my seventh year of dealing with this and I'm tired of all these symptoms. And I'm willing to try -- you know, I'm willing to take the risk to wipe it out completely. Of course, my main hope is that it is going to eliminate the virus completely and I really believe that it is. In terms of hopes for myself, I'm a survivor. I feel like that now and if it doesn't work for me, that's not the end of the road for me. But I think it may be for a lot of people. So many people are counting on this drug.

JENNINGS: [voice-over] Day One. The patients receive their first infusions of Compound Q. The trial consisted of three doses over three weeks.

Mr. HUSEMAN: I can tell you that we're going to see benefit or lack of benefit in hours, certainly in days.

JENNINGS: [voice-over] Dr. Levin was looking for an increase in T-4 cells, the white blood cells that fight disease. He was worried about side effects. He was most concerned about MTD, the maximum tolerated dose. That signals when the drug is about to overpower the body. Besides Tandy Belew, the other patients were 39-year-old Norman Watkins and 41-year-old Ron Fischer.

Dr. LAWRENCE WAITES, Physician: We needed people who had tried previous therapies and who had failed those previous therapies.

JENNINGS: [voice-over] Dr. Larry Waites assisted Dr. Levin.

Dr. WAITES: They have tried virtually everything and everything is failing them. And they are at the point now where they are trying this as literally -- it is their last resort.

JENNINGS: [voice-over] They waited anxiously for the drug to take effect.

Mr. BELEW: I was thinking about so many of my friends that have died and I felt their presence.

NORMAN WATKINS, AIDS Patient: And I just thought of all the people that have been so good to me in my life. That was the main thing in my life, was just seeing -- closing my eyes and seeing these wonderful people that had been so kind to me and so nice and so caring through this whole thing.

RON FISHER, AIDS Patient: I'm going to survive this and get over it. I'm committed to it. I believe that I'm going to survive it and get over it.

JENNINGS: [voice-over] They were kept overnight for observation. The activist and the doctor were anxious, too.

Mr. DELANEY: Okay, let's say the drug worked. Let's say it literally knocked out all these infected cells. Would the immune system recover? Does it have the capacity?

Dr. LEVIN: My guess -- I'm going to speculate. The guys with high suppressor cells will respond 'cause they have a lot of bone marrow reserves. The guy with low total T-cells and low suppressor cells might have more trouble. My sense is the guys whose bone marrows have been damaged by AZT won't do as well as the guys whose bone marrows haven't been damaged by AZT. I'm speculating again that there's going to be a dramatically clear result one way or the other.

Mr. DELANEY: Yeah. Yeah. Hope you're right.

Dr. LEVIN: And I'm also speculating we're going to run into some big trouble with some guys.

JENNINGS: [voice-over] The next morning, the first set of results were encouraging.

Dr. LEVIN: In the main, everybody did very well. In the one patient that we have the data on right now, we see a, I would guess, about a 35 percent increase in the white blood cells in 12 hours. That's pretty impressive. Medically, scientifically and ethically it should be made available to every HIV-positive person in this country by the end of the year, if it works. And we'll know that within six weeks to a month, two months.

JENNINGS: [voice-over] A very accelerated trial. Across town at San Francisco General Hospital, Compound Q was undergoing an official FDA trial and it would take at least 18 months under the direction of Dr. Paul Volberding.

Dr. PAUL VOLBERDING, Director, AIDS Program, San Francisco General Hospital: Well, even though there was some prior experience with this type of drug in China, there wasn't any experience that any of us had a direct involvement with. And we expected that the drug would be more toxic in the setting of AIDS. So when we designed the initial trials, we said, "We don't really know what dose to use. We don't know what side effects to expect, so we'll do a very careful study" -- it's called a "dose-escalating" study where we start out with very low doses of the drug and build up as we get more comfortable with the drug.

JENNINGS: [voice-over] Patients in Dr. Volberding's trial initially received only a fraction of the dose that Dr. Levin was administering to his volunteers.

Dr. VOLBERDING: That's the traditional way that we've used new drugs for the first time in people. So the plan for this drug is really comparable to the plan for other drugs that we've used in the past.

JENNINGS: [voice-over] Dr. Volberding did not know that the secret trial was taking place. On the seventh day of his trial, Dr. Levin was optimistic.

Dr. LEVIN: In every patient, we've seen at least a doubling if not a tripling of the white blood cell count. And these people have run very low white blood cell counts for years or for at least the year that we've been tracking them. So something very dramatic is happening to them. There are a lot of questions as to whether to double the dose next time or even to increase it by ten percent. We just don't know, so right now, to be on the safe side, we're just going to expand the population at this dose level and monitor them.

JENNINGS: [voice-over] So the next day, Norman, Tandy and Ron received their second infusion, the same amount as the first time, while four new volunteers began in the trial.

Mr. DELANEY: All right, well, that's still--

Dr. LEVIN: Five-thirty.

Mr. DELANEY: How long is that? So that's a week later and he's still got a white count in the normal range.

Dr. LEVIN: So more than -- almost twice the white count.

Mr. DELANEY: That's remarkable.

JENNINGS: [voice-over] On the eighth day, the experiment took a turn for the worse. After his second infusion, Tandy's mental capacity was seriously impaired.

Mr. BELEW: I'm scared, very, very much scared right now. I've been here all day, since, oh, what, 8:30 or 9:30, something like that. And the doctor wanted to see what's happening with me. He gave me some medications and they're not really seeing much difference. It's still difficult. And it's scary, you know. I'm pretty scared by all of the--

Dr. LEVIN: Well, Tandy, as you saw, had, you know, an episode where he has a lot of confusion and he has difficulty gathering words and has organic brain syndrome. And of course, most of our guys have described some level of feeling confused and inability to think well. He happens to be the worst. He's had the worst reactions of any of the guys as regards his problems with the drugs.

JENNINGS: [voice-over] At this point, Dr. Levin speculated that Compound Q might be killing brain cells, leading to temporary dementia or even coma. Then Levin's assistant, Clark Huseman, learned that Norm was also ill. The drug was taking over his body. It appeared they had reached maximum tolerated dose, MTD.

Mr. HUSEMAN: Norm was due to come in today anyway for blood work, to be checked. The friend is saying just how ill he is and how difficult it is for him to get around. And he expected to be in here anyway today. So that's what this telephone conversation was about. He has to come in, we have to check him out and if he can't walk -- he has trouble walking anyway, even before this started -- we have to get him in by ambulance.

JENNINGS: [voice-over] A worried Dr. Levin made a decision.

Dr. LEVIN: Well, that's MTD, friend. I mean, I'm not going any further with anybody.

Mr. HUSEMAN: So what does this mean for Thursday group? I think it's too soon to say right now.

Dr. LEVIN: You mean, in terms of the second infusion?

Mr. HUSEMAN: Second infusion.

Dr. LEVIN: Yeah. No second infusion on anybody as far as I'm concerned.

Mr. HUSEMAN: No second infusion on Thursday at this point?

Dr. LEVIN: At this point, right. Hello?

RECEPTIONIST: Hi, I'm sorry. Can you take a call from Martin Delaney on 65?

Dr. LEVIN: All right. Okay. Hi, Marty. We're hitting the wall. Well, Tandy has marked changes which are chronic and he's got atrophy and dirty white matter and -- well, he's got AIDS dementia. Yeah, in his case, we have to wait and watch. Norm Watkins is in here with a temperature of 103.2 which is coming down. Yeah. The problem with him is we don't know what antibiotic to use because he's been on all of them.

[on camera] I really don't like hurting people, but you know, obviously these guys had no alternatives and so -- I just don't want to be the one that accelerates their demise. But you know, we might be able to pull them through. Who knows?

JENNINGS: [voice-over] Hours later, Levin wanted to cancel the trial. Delaney wanted to proceed. They argued.

Dr. LEVIN: The next question, though, is do we do a second infusion on the second group?

Mr. DELANEY: Yeah. Well, see, on that one, I'm more inclined to think yes, but maybe rethinking the dosage question. I know some of those guys are very upset at the thought of not doing it, too.

Dr. LEVIN: But they're so sick.

Mr. DELANEY: Yeah. Well, maybe -- what about making some individual choices, then letting them call it? I mean that's sort of -- they're calling it anyway, so what would be the difference here?

Dr. LEVIN: 'Cause I'd feel guilty if I hurt them, no matter what they say.

Mr. DELANEY: Yeah. See, there's nothing in any of the lab work I've seen right now that suggests a problem with anybody. I mean, the only stuff in the lab work suggests some fairly significant benefits, in fact. Now, it's too early to make any kind of conclusion from that, but I don't see any evidence of harm in any of the blood work, do you?

Dr. LEVIN: I just see the evidence of harm walking through the door.

Mr. DELANEY: Yeah. Clinically. Right.

Dr. LEVIN: Right.

JENNINGS: [voice-over] By this point, the trial was no longer a secret. Word had leaked to the FDA, but the agency did not intervene. Former FDA official, William Vodra.

WILLIAM VODRA, FDA Chief Counsel for Drugs, 1971-1979: Ten years ago, the FDA might have been more inclined to say, "We're going to bring enforcement action against you to stop you from doing this research without prior approval of the FDA."

JENNINGS: [voice-over] The FDA has a long history of changing policy. It was created in 1938 by President Roosevelt after a patent medicine killed 107 people. It was the polio epidemic that first pushed the government to an all-out effort, bending its own rules to develop a vaccine. Then Thalidomide, the drug that caused terrible birth defects. So President Kennedy stiffened its regulations and made it harder to get drugs approved. These same rules were in effect in the early 1980s. Drugs had to be proven not just safe, but also effective, a cautious approach that seemed to work for most drugs, but the agency was unprepared to deal with the sudden onslaught of the AIDS epidemic and the highly politicized gay community.

Mr. VODRA: The FDA, I think, is intimidated by the AIDS community. The AIDS activists have sat in at FDA headquarters, occupied the desks and so forth. They've assaulted the FDA's integrity and honor and I think the FDA, frankly, is on the defensive. It's not been about to articulate and defend well its own position about what the law requires and why the requires and why that's in the public interest.

JENNINGS: [voice-over] By the end of the second week, Norman was unable to leave home and come for a checkup. Compound Q had apparently further weakened his immune system. Dr. Levin went to him.

Mr. WATKINS: All three of us got sore and tired and fatigued and I've had trouble with fever for two years, so I don't know if it's from another infection or from the drug itself. But this has been a bad week, so if I die, I was going to die anyway, you know. Probably. It's hard.

JENNINGS: [voice-over] Tandy's condition was troubling.

Mr. BELEW: And I was just doing okay for a while and then just completely lost it. Completely lost my mental capacity. Very, very out of it. Very much out of it.

Mr. DELANEY: Well, this comes back to the question that always come up with these and that is, do you treat the very sick people who desperately want to get treated 'cause they feel they have to or do you try to treat people at an earlier stage of the disease?

Dr. LEVIN: Well, this is the nature of a Phase One trial, right? I mean, that's what you do in the first place, establish MTD on the basis of the sicker patients.

Mr. DELANEY: But so often, they're the wrong patients to try treating. I mean, that's the history of this disease, has been that every drug that's come along, it's worked better at earlier stages, rather than later.

JENNINGS: [voice-over] Then, both Tandy and Norman stopped taking Compound Q. For the next two months, the trial continued with healthier patients. Altogether, 19 men were taking Compound Q. Then in late June, Ron Fischer died of pulmonary cancer. Days later, another patient died and suddenly, the news was everywhere. The secret that had been debated only behind the closed doors of the FDA was suddenly very public.

Dr. VOLBERDING: [news clip] Well, I think, first of all, the Project Inform study is playing more than a passive role. They're encouraging, by being involved, patients to use drugs out of desperation that haven't been tested for safety.

Mr. DELANEY: [news clip] Well, we face a situation here that has been typical throughout the AIDS epidemic, in that patients are gearing up for large-scale importation and distribution and use of this drug because it appears to be so promising.

JENNINGS: [voice-over] Six weeks later, the FDA, which had known of the trial almost from the beginning, felt obliged to respond. It sent a letter to Martin Delaney.

ELLEN COOPER, M.D., Director of Antiviral Products, FDA: The letter basically told Mr. Delaney that, you know, we certainly did not approve of the protocol, that it was illegal.

JENNINGS: [voice-over] But the letter had a curious turn of phrase. Although it said, "We feel that you should discontinue any further unapproved experimentation," it closed with, "We look forward to working with you," an unprecedented invitation to an AIDS activist.

Mr. DELANEY: I never expected FDA, you know, to come down on us and lock us up. I think they understood the good faith and intentions in what we were doing and that once they had the opportunity to look at how we did it, I think they could understand that we were scientifically responsible as well.

Mr. VODRA: The AIDS drugs are treated as a wholly separate category and the FDA is developing separate rules for those drugs. The long-term question is to what extent, when they get those rules in place, those rules will become the rules for drugs for arthritis and ulcers and diabetes and asthma and so forth.

JENNINGS: [voice-over] By the fall, Dr. Levin had assessed his findings from the trial and claimed that the drug might keep the AIDS virus from reproducing. And so he thought his experiments were justified.

Dr. LEVIN: I've got to tell you that the obligation between a doctor and a patient is above any law of any land, period. And if the Nazis say, "Throw this Jew in an oven," as a doctor, I would be obligated to say no. And I don't care what any regulatory affairs, any governmental institution tells me. If this is going to save a life, I'm going to do it. And I'll tell you that I can -- I'll do everything that's scientifically, medically and morally correct and somebody else has to make it legal.

JENNINGS: [voice-over] But by the end of the year, it was clear that Compound Q was not the miracle cure. Norman Watkins died of complications from AIDS. Compound Q may have contributed to his death. By then, one of the patients in the San Francisco General trial had died as well. Dr. Volberding had a different view of Dr. Levin's efforts.

Dr. VOLBERDING: We felt quite strongly that this was not a good idea, to do this kind of a study in a non-academic setting without the review, the external review of institutional review boards, the FDA and a drug company sponsor. And I still feel that that's true. I think what's changed, probably, is that we appreciate again that our goals are the same and that we've learned, too, over time that we really do have to look at our way of approaching things, to make sure that we're not slowing things down. It's not fair for us to criticize someone else if, in fact, we are part of the problem.

JENNINGS: [voice-over] But he also had some good news. Tandy Belew had rallied.

Mr. BELEW: I actually thought I was dying. I woke up one morning, I said to myself. "Tandy, this isn't working. I think you're dying. Stop your medications. Stop everything right now." So I did. I stopped it all and in two days' time, I knew I was coming back.

JENNINGS: [voice-over] In December, Tandy began taking Compound Q again.

Mr. BELEW: I was disappointed when it wasn't like the miracle cure right away. It wasn't the magic bullet. But continued with it and I think it's working. I see other friends of mine that are doing quite well on it.

JENNINGS: So, is Compound Q effective against AIDS? There are still no clear answers. The official FDA trial isn't over yet. Martin Delaney and the FDA are working together now. Delaney is planning yet another study of Compound Q, but this time, with official approval.