What's Wrong With AIDS Treatment Research?

Update January 1986: This article was published in the San Francisco Sentinel last May. Naturally much of the information, especially about AZT, has changed since then. But unfortunately, most of the public-policy concerns are all too relevant today. JSJ

Any review of AIDS research must distinguish between the basic science studying the biology of the disease, and the applied work to develop practical treatments and make them available to patients.

The basic research has performed very well, compared to the time required to understand diseases in the past. This scientific success, however, does not reflect competent Federal management or public policy. Instead, the scientific achievements have stemmed from the great progress in biology in the last two decades - and from the dedication and sacrifices
of individual scientists who for years managed to carry on their work without proper support.

While public policy for basic research has been merely poor, that for treatment development has been abysmal, amounting to a system of human sacrifice which could hardly be worse even if deliberately designed to have people die. Community organizations must development independent expertise on treatments, which they have not done so far. To rely solely
on official institutions for our information is a form of group suicide.

Here are some examples. The first one concerns the most promising treatment now under development.

(1) Azidothymidine (AZT). The general public, and even most AIDS organizations and activists, do not yet realize that we already have an effective, inexpensive, and probably safe treatment for AIDS.

The scientific and newspaper reports on AZT have been buried in an avalanche of other AIDS stories. The press, evidently advised not to raise "false hopes", has reported the news but not highlighted its importance. As a result, doctors do not need to deal with the anger of persons who know they are being left to die unnecessarily. And public officials do not
need to deal with political demands for a change.

In the only published clinical study of AZT (The Lancet, March 15, 1986), 19 subjects with AIDS or ARC gained an average of five pounds each during a six to eight week course of treatment. 15 of the 19 showed increases in the number of helper T-cells. Other improvements included two cases of nailbed fungus infections which cleared up with no anti-fungal
treatment, six patients who developed normal skin-test reactions when they had none before, and six who had an end to night sweats or a greatly improved sense of well-being. At the highest doses tested, AIDS virus disappeared from the blood. Only KS did not improve, perhaps because the treatment was not continued long enough, or because specific treatment for KS may
be needed in addition. Side effects were minor.

Eleven of these 19 patients had AIDS - six with PCP, four with KS, and one with both. One of the most important results of this study is that it showed that the immune system can re-build itself if the virus is stopped, contrary to what many doctors had believed. It is not necessary to write off persons who have already developed AIDS.

AZT does cross the blood-brain barrier.

The drug is not a cure. It will probably have to be taken indefinitely, and we don't yet have proof of long-term safety or effectiveness. The study did report on the condition of the patients later, four to eight months after entry to the study; except for one who was taken off the study due to advancing KS and who later died, they were generally in good shape, although
two cases of PCP, and some other opportunistic infections, developed several months after the patients had been taken off AZT.

So what's happening now? The study ended with the conclusion that long-term, controlled studies would be needed. Yet during the several months which have elapsed since the benefits of AZT were known, very little clinical work has been done. Large-scale studies are at least two more months away. If all goes well, your doctor might be able to get AZT in about
two years.

Of course there are risks in using a drug before the effects of long-term use are known. There are also long-term risks from untreated AIDS.

We should point out that ten thousand people are expected to die of AIDS in the next year. And with deaths doubling every year, a little math shows that a two-year delay between when a treatment is known to work and when it becomes available means that three quarters of the deaths which ever occur from the epidemic will have been preventable.

Incidentally, a biochemist can easily make AZT. The only reason it's not already available through a grey market is that people don't yet understand its importance.

(2) AL 721. AZT represents a shining public-policy success compared to what has happened with AL 721.

AL 721 has been around for years and is known to be safe for humans. And at least since November 1985, it has been known to prevent the AIDS virus from infecting human T-cells in the laboratory. But it still hasn't been tested on any person with AIDS or ARC, to our knowledge; there are rumors that secret tests, with only a handful of patients, may have started
in April 1986.

AL 721 is composed entirely of substances found in ordinary egg yolk. As such, it could legally have been sold as a food supplement - not as an AIDS medicine, but in fact made available to patients immediately.

Instead, its licensee, Praxis Pharmaceuticals of Beverly Hills, CA, treated AL 721 as a high-tech AIDS drug, using it last year to raise 3.7 million dollars in the stock market. The company itself predicted that it would be at least four years before the FDA approved any commercial sale.

Thus one of the most promising treatment leads available was lost to years of red tape. It's hard to get investors to put millions into a food supplement.

Praxis has cloaked it's AL 721 work in secrecy. We don't know how well they're handling it, but what is known isn't encouraging. Meanwhile, the licensing forms a legal barrier which prevents anyone else from touching AL 721.

Would a promising treatment be handled this way in any other deadly epidemic?

(3) A little arithmetic shows that an AIDS cure will be much more profitable in a few years than it would be now. In fact, today it would cause a big loss to the company which developed it. There is no commercial incentive to develop an AIDS cure too early.

Today it costs at least 50 million dollars to get a new drug through all the testing required for Federal approval. Divide 50 million by the ten thousand people in the U.S. who now have AIDS, and you can see that a company would have to make five thousand dollars clear profit on each person just to pay off its development expenses - not including interest,
insurance, and costs of operation.

But in a few years, the picture will change dramatically. With cases doubling every year, we will have a ten-fold increase within four years. That's ten times the potential profit.

Of course it would look bad for a company to hold up an AIDS treatment just to make money. Here the Federal bureaucracy provides a service to industry, withholding new- drug approval and forcing the private company to take the profitable path.

Of course the government shouldn't look bad, either. So under the spotlight of publicity it makes the right decisions. For example, the FDA approved testing of HPA-23, no longer one of the most promising treatments but the one Rock Hudson went to Paris to receive, in an unheard-of five days.

(4) Doctors genuinely care what happens to their patients. But doctors don't rock the boat. Very few will speak out or otherwise step out of the context which the larger system has given to them.

Doctors seldom initiate treatment trials. Even at the leading AIDS hospitals, doctors run trials when drug companies bring them something to test, not before. This fact is important because, as we have seen, drug companies have very different incentives than doctors in this matter.

It's also important because it virtually guarantees that a treatment which cannot be patented or licensed exclusively to some company - such as preventing ARC from developing into AIDS by eliminating suspected cofactors - will not be tested seriously.

(5) What can be done? Plenty.

The most important thing we can do is to build awareness - within AIDS organizations, within the high-risk communities, and among the general public - of what is going on and where the problems are. So far, community-based AIDS organizations have been uninvolved in treatment issues, and have seldom followed what is going on. No wonder the gay community and the general public don't know, either.

With independent information and analysis, we can bring specific pressure to bear to get experimental treatments handled properly. So far, there has been little pressure because we have relied on experts to interpret for us what is going on. They tell us what will not rock the boat. The companies who want their profits, the bureaucrats who want
their turf, and the doctors who want to avoid making waves have all been at the table. The persons with AIDS who want their lives must be there, too.

Organizations with treatment expertise can find out exactly where the delays are. They can analyze the current plans to centralize Federal AIDS research. They can identify and publicize important leads which have been dropped. They can expose gross mis-allocation of funds, such as attempts to inject homophobia, racism, or other political hobby horses into
AIDS research policy. When black markets develop, they can receive samples and have them tested by reputable laboratories, to protect people against fraudulent or inferior products. We have not had this kind of advocacy so are, and we have suffered for it.

We must of course talk with everyone, co-operate with everyone, be willing to work together. But no longer can we rely entirely on government-sponsored organizations or on the medical-industrial complex for our understanding of what is happening with treatment research and development. The people there may have their hearts in the right place, but they must
follow the dictates of a system which fails to follow up opportunities, does not make saving lives a high priority, and is not always directed in good faith.