January - February
Q & A on Ampligen
Hemispherx’s David R. Strayer,
In an effort to provide Association
members with answers to their
many questions about Ampligen and the planned Phase III clinical trial, the Chronicle asked Hemispherx
Biopharma, manufacturer of Ampligen, to respond to some of the most frequently asked questions. David
R. Strayer, MD, Medical Director for Hemispherx Biopharma, provided the following answers to our questions.
We appreciate his response and look forward to keeping you up to date as the clinical trials proceed and
Hemispherx Biopharma moves Ampligen toward full Food and Drug Administration (FDA) approval.
phase 3 clinical trial that is both double-blind and placebo-controlled and involves a significant number
of patients is the ultimate test of a drug's safety and effectiveness before it is approved by the FDA.
What is the status of such a trial for Ampligen?
Answer (Dr. Strayer):
The phase 3, randomized,
placebo-controlled trial of Ampligen for the treatment of CFS/CFIDS has already started to enroll patients.
Ultimately, over 200 patients will participate at approximately 8-10 clinical sites throughout the United
States. The patients must meet the eligibility criteria of the study and will be selected by the investigators
at each site. To date, investigators at the following six clinical sites have agreed to participate:
1. Dr. Paul Cimoch, Irvine, Calif.
New Brunswick, N.J.
3. Dr. Robert Keller, Hollywood, Fla.
4. Drs. Ronald Moss and Alex
Mercandetti, San Diego, Calif.
5. Dr. Daniel Peterson, Incline Village, Nev.
6. Dr. Richard
Podell, New Providence, N.J.
Additional clinical sites will be added
in the near future. The trial
is expected to continue through the end of 1999. The first phase includes 40 weeks of double-blind, placebo-controlled
treatment. In the second phase, patients who complete the first phase will receive 24 weeks of open-label
treatment with Ampligen.
you provide results of
the clinical trials conducted in Belgium? Where and when will this information be published?
Since this study is continuing
to enroll patients, no decision has been made as to when and where the clinical results will be published.
Results on 44 patients studied by Professor De Meirleir suggest increases in physical performance and
cognition after 24 weeks of Ampligen which was statistically significant as presented in Cambridge, Mass.,
at the American Association for Chronic Fatigue Syndrome conference, Oct. 10-12, 1998.
How many patients have received
and are currently receiving Ampligen in the United States under the current cost-recovery program? Will
more patients be able to obtain the drug through this program?
date, 36 patients have received
Ampligen in the United States under the cost-recovery program. Currently, 22 patients are receiving treatment.
It is estimated that within several weeks, approximately 50 patients will have received Ampligen under
this particular program. The direction of the program is under discussion with the FDA.
Q: Under what conditions
is Ampligen available
to patients in Canada and other countries?
is available to CFS/CFIDS
patients in Canada under an Emergency Drug Release Program administered by the Canadian Health Protection
Branch (HPB, similar to the FDA in the United States). Physicians in Canada request authorization from
the HPB to treat with Ampligen on a named-patient basis. Patients in Austria and Belgium may also be treated
under a similar program. In some instances, Americans have relocated temporarily to other countries in
order to obtain Ampligen treatment.
have seen reports that
clinical trials scheduled to take place in the United Kingdom have been canceled, but that Ampligen is
to be made available there on a named-patient basis. How will this program work?
clinical tests are presently under
way in England. The company is in active discussion with various British physicians specializing in CFS/CFIDS
research to initiate a clinical program.
on the information
you now have about Ampligen in treating CFIDS from previous clinical trials in the United States and Belgium,
as well as from the current cost-recovery program here, what can you tell patients about the safety and
effectiveness of Ampligen? Have you been able to identify any patient characteristics or laboratory findings
that correlate with a particular response to Ampligen?
Ampligen is presently an investigational
drug and is not formally approved by the FDA under the new drug approval regulations for treating CFIDS,
no claims can be made by Hemispherx Biopharma regarding the safety and/or effectiveness of Ampligen for
treating CFS/CFIDS. To date, Ampligen has been generally well-tolerated in CFS/CFIDS patients and no special
patient characteristics or laboratory findings have been shown to correlate with a particular response
to Ampligen. Interested parties are encouraged to review relevant transcripts of the recent CFS research
meetings and to speak with clinical investigative groups which are conducting the ongoing clinical tests.
was first administered
to CFIDS patients in the United States in 1989 and 1990. Has your company continued to follow the clinical
status of those patients? If so, what have you learned about the long-term effects of Ampligen treatment?
follow-up data on patients
who had received Ampligen and then discontinued treatment for periods of time ranging up to several years
was presented at the AACFS meeting in Cambridge last October. The results as presented at the meeting
suggest that the improvements in physical performance and cognition continued during the follow-up period.
Interested parties are encouraged to review the full relevant transcript in order to develop an independent,
in depth, perspective.
We [at Hemispherx] recommend
interested parties contact the
above referenced clinical investigators or the Hemispherx Director of Clinical Operations, Mr. David Walters,
at E-mail address HEMXJMD@bellatlantic.net for further information.