AIDS TREATMENT NEWS No. 051 - February 26, 1988
The biweekly newsletter AIDS Treatment news began as volunteer
research and writing for an AIDS archiving organization in San
Francisco. In little over a year it has grown to a circulation
of over 3,500 almost entirely by word of mouth--an unexpected
public response which illustrates the critical dearth of
practical treatment information felt by patients and physicians
alike. Researching treatment articles for AIDS Treatment News
has provided an unusual opportunity to hear what this community
would like to see happen, and where it sees the obstacles now.
People react to an AIDS diagnosis in different ways. Some
resign themselves to dying and begin to prepare for death.
Others ask their doctors to make the medical decisions for
them, without their personal involvement. I do not have
contact with these people and do not know what they feel about
treatment research and access issues.
But very many persons with AIDS or other HIV infections choose
to involve themselves in decisions about their health care.
They often become experts in the disease and potential
And most of these people come to feel abandoned and betrayed by
society. They believe that many physicians, researchers, and
officials have been quick to write them off as already all but
dead--despite all the unknowns about this disease which make it
impossible for anyone to be sure that death is inevitable. The
projected deaths of at least a quarter of a million Americans
seem to have been accepted with surprising equanimity and
surprisingly little sense of crisis or mobilization.
Oddly enough there seems to have been no professionally
conducted survey asking the persons most directly affected by
AIDS what they thought about the issues of treatment research
and access now before this Commission. Certainly the people I
know have never been asked how they see the situation, what
problems they find in the institutional response to the
epidemic, and what improvements they would suggest.
A Wish List
Since we have no scientific survey information on what people
with AIDS would most like to see done, we did the next best
thing and interviewed Nathaniel Pier M.D., a physician with a
large AIDS practice in New York City. We have found his
statements about what is needed to be as close as anyone's to
the beliefs of the persons with AIDS with whom we have
communicated while writing AIDS Treatment News.
Dr. Pier proposed above all "That anybody diagnosed with HIV-
related disease or immunodeficiency be given a full assessment
of their situation and be allowed to choose to receive a
therapeutic regimen or decline it. Theoretically, all five
hundred thousand persons infected in New York should be allowed
access to some form of therapy if they wished. To satisfy
scientific needs, they could be enrolled in formal protocols.
Otherwise clinicians should be allowed to use empirical
regimens, with patients properly monitored.
"This way everybody would be given the optimal chance to save
their lives and nobody would be allowed to twist in the wind.
Furthermore, we could look at the results--and get a sense of
what works much more rapidly than under the current system.
"Persons could use single drug treatments, or rational
combinations based on the best judgment of experienced
physicians. "What we propose here is what is already done with
cancer patients. Almost no one diagnosed in the United States
today with cancer is denied an opportunity to participate in
potentially lifesaving therapy. There is in place a widely
accepted system for providing these experimental and
established therapies to cancer patients. This system advances
our knowledge of the treatments for this disease but is also a
humane and compassionate way of caring for patients.
"To the argument that there are no AIDS treatments except AZT
because no others have proven effective, we would answer that
we are currently capable of choosing safe, rational approaches
to therapies. In addition, people are using therapies anyway.
Our proposal would allow them to do so under supervision, so
this can be done safely and the data developed can be
critically evaluated and thereby be helpful to others instead
of remaining anecdotal."
"It is clear that the best hope for people with immune
deficiency or at risk for the illness is the rapid development
and dissemination of safe and effective therapies. Until this
goal is achieved, the most humane approach to dealing with AIDS
and AIDS-related problems is to give people access to
supervised therapeutic protocols. The main problem, therefore,
is to develop such a system--a system that would allow rigorous
scientific analysis of therapies and still incorporate anyone
wishing to try to help themselves with experimental therapies.
"The present system for developing AIDS therapies has been
painfully slow in starting. Access is so severely limited that
the majority of people affected by this disorder are left
without intelligent recourse.
"In addition it is unclear where the leadership for determining
priorities in therapy development is coming from. It is also
unclear how the decisions for prioritizing the various
therapeutic approaches are being made. For people with AIDS it
is unclear who is setting the timetable and who is supervising
the large-scale effort to develop therapies.
"For the individual who must make decisions there is no
centralized method of gaining access to the information that
will allow him or her to choose the best course of action.
"How does the present system work? As an example, we submit
correspondence relating to a potential therapy for AIDS which
first was recognized in 1984. In spite of prominent AIDS
researchers acknowledging the potential benefit of this
therapy, no clinical trials on humans with HIV infection have
been initiated since then. In addition, you will see that
letters to the people vested with protecting the public health
have gone ignored and unanswered. This has left the impression
that they are inefficiently and callously dealing with this
very important issue. We do not believe that this is truly the
case. Nevertheless the letters have gone unanswered and the
trials have not materialized."
Lentinan. Dr. Pier's statement above concerns his two-year
attempt to get this drug considered. We should point out that
lentinan has long been used for cancer treatment in Japan, with
complete safety. And a letter to The Lancet (October 20,
1984), signed by seven scientists including Robert Gallo M.D.
describes its use in the successful treatment of two patients
which retroviral infections (one with HIV, the other with
In four years nothing has been done. Examination of Dr.
Pier's correspondence with governmental authorities clearly
illustrates the frustration and difficulty he experienced in
attempting to get this potential treatment considered on its
merits. Now we have heard that NIH has put lentinan into its
highest priority category for investigation--without major new
information, essentially on the basis of what was known four
years ago. However the drug-selection process is secret so we
only have hearsay and have not been able to confirm that
lentinan has been placed into the high priority category, or
that it was done without new information.
AL 721. The unhappy story of the repeated failures to test
this drug properly and make benefits available is presented at
length in the back issues of AIDS Treatment News, submitted
into the record of this hearing.
In yesterday's hearings one of the Commissioners asked FDA
Commissioner Dr. Frank E. Young if a therapy might be developed
to help overcome drug abuse, which is becoming so important in
the spread of AIDS. AL 721 was in fact first developed
primarily for that purpose. Theory, laboratory, and animal
studies have suggested that it might be effective in reducing
the symptoms of opiate or alcohol withdrawal, thus helping
abusers to overcome their habits permanently. However to our
knowledge no human study has been done--not even a small,
quick, inexpensive pilot study which would give some sense of
whether it was worth proceeding with this potential medical
intervention against drug abuse.
Trimetrexate. The important news about the approval of the
first AIDS-related treatment IND has failed to acknowledge a
major concern. Theory and laboratory studies suggest that the
trimetrexate with leucovorin therapy now approved for
pneumocystis pneumonia (when standard therapies have failed)
would very likely also work against cryptosporidiosis, a severe
and often fatal diarrheal illness of persons with AIDS.
Cryptosporidiosis presently has no satisfactory treatment.
We have heard that even a leading gastroenterologist has been
unable to obtain trimetrexate for compassionate use for
treating cryptosporidiosis. We have also learned that the
manufacturer, Warner Lambert, has no plans to develop the drug
for this condition.
The result is that this drug, already proven so safe in persons
with AIDS that almost none of the (pneumocystis) patients had
to have the therapy terminated, will never be tested for
cryptosporidiosis under the current system, despite the immense
benefit the discovery of a successful therapy for this
opportunistic infection might bring.
Salk Polio Vaccine. The "old" Salk killed-virus polio vaccine
(not to be confused with Salk's current work on an HIV vaccine)
has recently been tried as a possible ARC or AIDS treatment.
Although it is far too early to be sure it is effective, this
therapy has generated considerable excitement among the
physicians who have seen the results. In addition, according
to an overview article which appeared in The Wall Street
Journal on January 27, 1988, this possible therapy has also
attracted unusual attention from some NIH scientists, who have
determined that persons receiving repeated treatments with this
vaccine have produced neutralizing antibodies against the AIDS
virus. We have heard that Dr. Pitts is now collaborating with a
university and a county board of health on a formal study,
approved by his IRB--but that Dr. Pitts and a colleague must
pay for the vaccine out of their own pockets.
We have also heard two reports that Connaught Laboratories
Inc., the only company able to sell the Salk polio vaccine in
the United States, has recently made it difficult for
physicians to obtain supplies for use in treating AIDS--even
though it is perfectly legal for physicians to use it for that
purpose. One internist told us that the company refused to
ship the vaccine unless he signed an affidavit that it would
only be used to immunize against polio. And we also heard that
Dr. Pitts' group had to th reaten a lawsuit in order to obtain
supplies for the study cited above.
We have been unable to confirm these reports because Connaught
has refused to discuss them. It is widely believed in the AIDS
community that companies do not on their own resist the
development of new markets for their products. It is generally
presumed that these cases reflect fear by the company of making
enemies at the FDA, which may fear damage to the regulatory
process from the development of a public demand for a drug
outside of normal channels. Bureaucratic interests may be best
served if the usefulness of a valid AIDS treatment is never
discovered in the first place. Patients' interests differ.
All this in conjecture, of course, as in these cases no one
talks, and unless an insider reveals information nothing can be
The polio-vaccine case is not at all unusual. In case after
case, too numerous to list here, deliberate roadblocks and
obstacles have impeded patients in obtaining treatments, and
prevented research which could serve as early pilot studies to
indicate whether or not an idea deserved further, more formal
1. That either the Commission or another body investigate the
problems cited above, and dozens of similar ones which we
can bring forward, to find out what did happen, if there are
indeed roadblocks to treatment and treatment research, and
how these roadblocks could be overcome.
2. That the Commission arrange for a survey to ask persons with
AIDS, ARC, and asymptomatic HIV infection what they think
about current public policies regarding the epidemic, and
how those policies might be improved.
3. That the Commission ask the FDA to provide guidelines to
researchers outlining what studies would be required to
qualify a drug for treatment IND or for approval. These
guidelines should specify when it is and is not ethical to
use placebos in persons with life-threatening disease, or to
withhold use of previously proven therapies such as
4. That the Commission recommend the creation of a public,
computerized and printed registry of all human trials for
treating AIDS and related disorders. This registry should
include pertinent information about each drug, and the
protocols, in language that can be understood by a lay
person. Registration in this database should be required for
all government-funded protocols, and voluntary registry of
all others should be encouraged.
5. That the Commission recommend steps to make access to
therapeutic trials equally available to all qualified
persons. A system must be established for insuring fair
access to everyone in need. A lottery might be suggested.
6. That the Commission suggest the creation of a confidential,
voluntary registry of individuals affected by AIDS and
related disorders, whereby these individuals can be notified
automatically when there are new trials for which they can
qualify. (This system could also help researchers recruit
for their trials.)
7. That the Commission recommend the immediate expansion of
funding for experimental trials organized and run at the
community level, using the resources of private and
community physicians, such as the Community Research
Initiative in New York, and the County Community Consortium
in San Francisco.
8. That the Commission encourage the current attempts to share
and disseminate reagents, materials, and scientific data
within the scientific community, to speed the discovery of
safe and effective therapies for AIDS.
9. That the Commission recommend the development of a system
such as compulsory licensing which would prevent proprietary
restrictions on data and access to drugs from impeding
development of AIDS treatments.
10. We urge the Commission to recommend that individual
patients and their physicians be allowed to choose to use
safe experimental therapies under supervision, even before
efficacy has been confirmed, if informed consent is