Treatment Optimism and Research Ethics: Interview With Nathaniel Pier, M.D.

Nathaniel Pier, M.D. has about 300 AIDS/ARC patients in his private practice in New York. He also works with New
York's Community Research Initiative (CRI), a project of the PWA Coalition. The CRI has obtained State and Federal approval to design and conduct clinical drug trials, providing unique opportunities for persons with AIDS to have input into clinical drug research.
We have spoken with Dr. Pier several times in the last two months, about the growing optimism on AIDS treatments, and about problems in research policy. We recorded and transcribed parts of these conversations and interviews.
We have learned that physicians in private practice are more free to speak openly if they want to, than scientists who
usually need permission to make public statements, and who dare not offend the institutions which control research funding.

Growing Optimism On AIDS Treatments

"The point we've reached in late 1987 is really quite an optimistic one. A year ago when we were just starting out with
AZT we could say that whether or not AZT works there's a profound change here, which is that we do have a tool which is a first step in treating people. And it forced the government and Otis Bowen at the meetings in June (the III International
Conference on AIDS, in Washington, D.C.) to admit that people who are already affected by AIDS should not be thrown on the dunghill of humanity, that it was worth trying to save them, that in fact medications could be developed or should be developed that would treat them. And that's led to a profound change in the way people approach AIDS.
"Part of the optimism here is that we're beginning to bias the process in favor of good news. We're beginning to have
more than one option. It's not just AZT now, it's AZT plus a number other drugs that are in phase II testing that may work.
"This perspective must be brought out. Patients must hear that although they have a serious problem that may result in them getting very ill or even dying, it's equally reasonable for them to hear that scientific progress has advanced
significantly to the point where we can now introduce variables into the course of their disease that may mean they will not die. That, coupled with the fact that it is clear that there are long-term survivors, and that this is not a universally
fatal, 100-percent-everybody-dies disease, makes it from a medical person's point of view a much more manageable disease. If you look at the 30-35 year old age group who have kaposi's, one in three is living more than five years. This is better statistics than many cancers. And somebody who presents in that fashion should not be told that they have a universally fatal illness.
"We must appeal to other physicians to stop the gloom and doom stuff. I know people are dying of AIDS, but when you're talking to an individual patient who is perhaps being diagnosed for the first time...they shouldn't necessarily feel that they're obligated to get sick and die to prove the CDC correct, or the New York Times for that matter.
"As far as practical things, if you ask the doctors in New York City about mortality rates, most of them, in fact everyone
I talk to will tell you that the mortality rates have gone down considerably, partly because of AZT, some believe, partly
because we're getting better at treating diseases that occur in AIDS, partly because of pneumocystis prophylaxis. But it is clear that AIDS is not the profoundly, "Oh, shit, the patient's got six months to live" kind of thing that it was a year or two
ago. And it's clear that physicians who are very skilled at dealing with AIDS do not lose patients in the kind of numbers
that the same physicians did two years ago, or that other physicians who are not very skilled at dealing with AIDS are
losing their patients."

The Wrong Way to Treat AIDS

"Recently some Harvard-trained physicians visited me to look at my practice and see how I deal with AIDS, because I've had so few deaths. They were astonished. What I do they do not do. They never heard of resources like AIDS Treatment News or Project Inform. And as a consequence their patients don't get the full spectrum of counselling, their opportunities for choosing their therapies are limited, and they don't do as well.
"It should be clear to the medical community that if you make a concerted effort with each individual to discuss
therapeutic options that may be appropriate for them, such as participating in a trial of an experimental drug, or trying
alternative therapies, they may do better, and survive longer and in better shape with a better quality of life. That's the
message that's being lost. If you ask the infectious disease specialist, who may be the typical doctor that is asked to
consult on an AIDS patient, what to do, it's AZT or nothing. That's a disaster. They don't take a look at what options the
patients have, they don't counsel the patients as to what other alternatives there are, and if the patient doesn't do well on
AZT they don't offer them anything else.
"If you look at medical literature you see statistics like 80 to 90 percent of AIDS patients develop dementia. These
reports come from a skewed population. These papers are written by neurologists who see end-stage AIDS patients in
tertiary care centers. They don't see what I see or what the usual clinical doctor sees. I have 300 patients with AIDS or
ARC in my practice, and maybe one of them has dementia. And why? Because I see the healthy group of AIDS patients. The people who write papers in tertiary care centers see a biased population. They see the disasters, they see the ones who haven't done well. And so naturally they publish these horrendous statistics of how poorly AIDS patients do. But if they come and they see my practice and they see what I see week after week, of basically healthy people who are out there in society, who are working, who are not demented, who don't have severe peripheral neuropathies, who very occasionally have problems that are AIDS related but are basically doing OK, they
would write very different papers. There's a serious bias in the medical literature also as to what is being reported, and
it's giving people a distorted picture of what is going on with AIDS. They're not seeing the people who are doing well, the people who are surviving long, the people who have good quality of life, because those people don't go to those centers and therefore they don't get papers written about them.
"The bottom line is that if a physician takes the time to look at each individual and talk about what their options are,
in terms of taking standard orthodox therapy, in terms of enrolling in an experimental treatment program, or in terms of
looking at alternative therapies that may in fact have some value, like AL 721 or naltrexone, you can not only treat a
patient and get them into a state where they can have some hope and some idea that there's something to do besides wait for the bomb to drop, that in fact if one of these therapies works, you've done an enormous good."

Research Policy and Ethics: Pneumocystis, DHPG, Ampligen, Lentinan

"The people who are affected by the research, by the disease, are demanding to know what the timetable is, and they want explanations.
"AIDS has forced the American medical scene to look at aspects it's never looked at. Like the bioethics of drug
testing. Is scientific data proprietary? Should the private drug companies, which the FDA constantly points to as being in partnership with the government, should they have proprietary interest over scientific data, like do their drugs work, or
should the people who are actually paying for the government have the right to demand that these companies make their scientific data public? What about the degree of time it takes to get scientific data published?
"A good example of that is pneumocystis prophylaxis. In New York we've been using pneumocystis prophylaxis for a year, some have been using it for 18 months or longer. And in June when we spoke with Dr. Anthony Fauci (of NIH) about pneumocystis prophylaxis, people from ACT-UP and the PWA Coalition, he told them that they were fools, that there was no study that showed that pneumocystis prophylaxis was of any value and that we might be putting people in danger by doing it. In fact, five months later the New England Journal of Medicine published an editorial which ssentially states that prophylaxis for pneumocystis should be standard therapy for people with AIDS. If we were wrong it would have done little harm. But we were right and therefore many people's lives were saved.
"If a therapy is generally deemed safe and we employ it, if we're wrong we haven't lost much, but if we're right we've
done a great good. In drug therapies where there is a track record in human beings of safety, we needn't wait for efficacy to be proved before we give the individual the opportunity to choose to use or not use such a therapy. This doesn't mean that good rigorous studies couldn't or shouldn't be done; it's just that the number of people affected is so large and the time so precious that we need to construct a system whereby both the needs of the individual to choose therapies is acknowledged and accepted as well as the need for researchers to do rigorous science.
"If DHPG has clearly been demonstrated to help people with CMV retinitis, it seems completely illogical not to approve it for use, even though the studies haven't been done. And to punish people, to punish the patients by letting them go blind or die, in order to make the companies do the tests right, is just not logical or humane, and it should call into question why this decision is made and who is making it.
"I know of a patient in a small town who went blind in one eye and was told by an ophthalmologist who diagnosed him as having CMV retinitis that there was nothing to do about it. In fact within a few hours of his getting me on the phone he was in an appropriate hospital in Miami receiving DHPG therapy. It's disturbing to me that the medical establishment feels it has no responsibility to offer to people with no other options therapies that have at least shown some efficacy. One can't exactly say that this ophthalmologist was committing malpractice, but certainly in the name of humanity, in the name of this man's vision, he should have known that in fact there was something to do besides letting him go blind.
"The FDA continues to say that the DHPG rejection was an advisory committee decision that has nothing to do with whether the FDA will or will not approve it. What the FDA hasn't done is put down a set of guidelines or rules that will allow persons who are interested to determine whether or not a drug will be approved. Exactly how many trials need to be done, what kind of trials, how many patients, etc? In a meeting with FDA Commissioner Frank Young that a number of concerned people had a couple of weeks ago, what he said is that it was completely up to his judgment, that there were no hard and fast rules; all you could say is that if a company wanted the drug to be approved, it would just have to apply and the FDA would use its judgment. But he refused or was unable to give out any guidelines. Of course if he did that it would allow people with AIDS to determine whether and how the FDA was influencing research policy.
"A good example is that the Ampligen study that's being done now in New York City at St. Luke's/Roosevelt Hospital
apparently forbids any pneumocystis prophylaxis. This is a major ethical point; if you have a therapy that is of accepted
benefit, is it ethical to deny this therapy to people at risk, because it might interfere with your endpoint for drug tests?
This is an major question, and I asked the fellow who spoke about the Ampligen study who designed this protocol, and was there any input into this protocol by persons with the disease, did they voluntarily say yes, we will agree not to take this life-preserving therapy in order to advance science?
"It is clear that if the FDA has decided that, say, the endpoint for the efficacy of a drug that is designed to prevent
people from going on to develop full-blown AIDS is whether or not they develop PCP, and therefore pneumocystis prophylaxis cannot be given, this is an major ethical question. I don't think that most people who are in genuine risk of developing PCP would in fact agree that this is an acceptable endpoint. And since Ampligen is one of the most hopeful drugs that we have, the exclusion of the ability to prevent PCP and the exclusiveness of Ampligen, the lack of number of slots in the trial, is coercive, is basically saying to a patient, "You do what we say, or die".
"I would still like to know why lentinan hasn't been tried. I would still like to find out what goes on in the AIDS
drug selection committee, why they meet so infrequently, why the minutes of their meetings aren't published, and why people aren't watchdogging them from Congress or other public agencies, and why they don't allow proprietary data that's submitted to them by private companies to be examined if it's relevant to the health and well being of people. Like the studies of lentinan, why Bristol Labs does not allow them to be published, it's completely unethical. And I think that people with AIDS should rise up en masse, and say that you can't do this to us anymore.
"These questions of ethics in human research are profound, and it's a service to humanity that the community of people with AIDS is performing by calling them into question and forcing the medical establishment to examine them. And we can be proud of the fact, and look forward to the resolution of some of these issues in favor of the people who are suffering. This would include not only AIDS, but also other grave illnesses like cancer and multiple sclerosis. It's always the case that in war like this, episodes of heroism and rapid progress occur even in the face of disasters. In this war the people who are suffering most are also offering the most and making the profound changes that will benefit everybody.
"This is not just a call for expressing anger, but a call to organize behind dramatic change in the way that medical
research is done, and organize behind a further dethroning of the cult of medicine. The medical authorities must also be
asked to be responsible to the public that benefits from their efforts and also pays for their work. We're asking that these
people acknowledge the fact that the consuming public does have the right to do this, even though they might not have the knowledge to make medical judgement. We don't want to condemn the sincerity of any individual's efforts to combat AIDS. What we're asking for is a two-way street.

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