This article is reprinted with permission from the CFIDS Chronicle, Vol. 12 No. 1 January/February 1999.
In an effort to provide Association members with answers to their many questions about Ampligen and the planned Phase III clinical trial, the The CFIDS 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.
Question (Chronicle): A 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.
2. Dr. Joseph John, 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.
Q: Can you provide results of the clinical trials conducted in Belgium? Where and when will this information be published?
A: 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.
Q: 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?
A: To 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?
A: Ampligen 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.
Q: We 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?
A: No 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.
Q: Based 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?
A: Since 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.
Q: Ampligen 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?
A: Long-term 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 Hemipherx Director of Clinical Operations, Mr. David Walters, at E-mail address HEMXJMD@bellatlantic.net for further information