Clinicians and Biomedical Experts Request Major Ampligen Review at FDA

  • Date: June 5, 2015
  • To: White House, HHS, Senate HELP Committee and House Energy Commerce Sub- Committee on Health, Drs. Woodcock, Chowdhury and Maynard
  • From: Clinicians and Biomedical Experts in ME/CFS
  • Re: Request FDA Facilitate 2-day Meeting for Treatment of ME/CFS

As clinicians and biomedical experts in Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), our goal for over the last four plus decades has been to advance the care and treatment of those patients suffering from this devastating illness. Critical in their care and treatment is access to approved treatments.

Recently the Institute of Medicine (IOM) released a report, “Beyond Myalgic Encephalomyelitis/Chronic Fatigue Syndrome: Redefining an Illness” (1) stating ME/CFS is a legitimate, serious and complex systemic disease that dramatically limits the activities of affected individuals. This disease is characterized by profound fatigue, cognitive dysfunction, sleep abnormalities, autonomic manifestations, pain, and other symptoms that are made worse by exertion of any sort. ME/CFS can severely impair patients’ ability to conduct their normal lives. The report states there may be as many as 2.5 million Americans affected at a cost upwards of $24 billion per year. This report is an example of a significant number of strides which have been made in our field; yet there are still no FDA approved treatments for this disease.

We are requesting your support to compel the FDA to facilitate a process with the sole purpose of providing conditional approval for the drug Ampligen®. We recommend a two day, facilitated process to examine how to address scientific and medical concerns of the FDA. We are in consensus in this request and commit to provide our time and expertise to this process along with the sponsor, Hemispherx Biopharma.

Ampligen is the only drug in the pipeline and has been evaluated in Phase II and III clinical trials in ME/CFS over the last two decades. Since 1997, it has been provided under an open label treatment protocol. To date hundreds of patients have been infused with more that 90,000 of doses of Ampligen and there are patients who have received more than 1500 doses over 12 years and remain on the drug.

Ampligen is a broad spectrum antiviral and immune modulator which has demonstrated that it provides substantial improvement in this patient population. In a 2012 peer reviewed article in PLOS ONE (2), the conclusion was Ampligen [rintatolimod] produced objective improvement in Exercise Tolerance and a reduction in ME/CFS related concomitant medication usage as well as other secondary outcomes. Experts have provided public testimony that approximately 25-40% of the patients, who are able to access the drug, have shown improvement in their daily function. At the 2012 FDA Advisory Committee Meeting, the committee voted 8 to 5 that the safety profile of Ampligen was adequate for the approval for the treatment of CFS. We know CFS is heterogeneous. We know there are those individuals with CFS who benefit from Ampligen.

So why has the drug not been approved? This is an extremely complex chronic illness and this disease has long been poorly understood by the scientific, medical and regulatory communities. The movement of Ampligen through five separate divisions of the FDA demonstrates the lack of understanding of the illness and of the drug’s benefits and risks. At a meeting of experts and patients with the FDA in March of 2014, FDA still mischaracterized CFS symptoms as adverse events.

FDA has been given the authority to provide access to promising treatments to patients with serious and life threatening diseases; specifically when there is a large unmet medical need. For example, two obesity drugs were denied approval (3) then through a collaborate process with the FDA, the drugs were granted approval with conditions. During the meetings, the experts were able to demonstrate a clearer understanding of the risk/benefit ratio and impact on quality of life of these patients. The FDA recognized the need, understood the risk/benefit and realized there was still more to learn. We believe the approval of the obesity drugs with conditions is a good model for the approval of Ampligen for ME/CFS with conditions.

With our medical and research expertise in this disease, our clinical trial experience and understanding of Ampligen, we will work collaboratively with the FDA to outline a path to approval with conditions that will answer any unresolved concerns. We will assist in the development of an appropriate REMS program to advance physician education and ensure proper administration of the drug.

We applaud the efforts by both the House and the Senate on their work to advance biomedical innovation, however, the patients cannot afford to have another decade pass. Currently, the quality of life of patients with ME/CFS can simply be measured in years of life lost – with no hope of recovery. If you review the thousands of testimonies provided to the Dept. of Health and Human Services, you would easily find these two phrases repeated over and over again, “I have lost my life to this disease and I have tried everything”. We request that you assist us in creating a collaborative pathway and allow access to treatment for those suffering this devastating illness.

Sincerely,

Dharam V. Ablashi, DVN, MS, Dip Bact.
Scientific Director of HHV 6 Foundation
Co-Founder of IACFS/ME
Santa Barbara, CA

Lucinda Bateman, MD
Clinician and Researcher
Fatigue Consultation Clinic
Salt Lake City, UT

Laura Black, MD
Physician
Hunter-Hopkins Center
Charlotte, NC

Derek Enlander, MD
Clinician and Researcher
ME/CFS Center
Faculty, Mt. Sinai Medical School
New York, NY

Kenneth J. Friedman, PhD
Associate Professor of Pharmacology and Physiology (retired)
Board Member and Treasurer, IACFSME
Fort Lauderdale, FL

Maureen Hanson, PhD
Researcher, Liberty Hyde Bailey Professor
Department of Molecular Biology and Genetics
Cornell University
Ithaca, NY

Leonard A. Jason, PhD
Professor of Psychology
Director, Center for Community Research
DePaul University
Chicago, IL

Nancy Klimas, MD
Clinician and Researcher
Director, Institute for Neuro Immune Medicine
Nova Southeastern University
Fort Lauderdale, FL

Andreas M. Kogelnik, MD, PhD
Clinician and Researcher
Director, Open Medicine Institute
Mountain View, CA

Charles Lapp, MD
Clinician and Researcher
Hunter Hopkins Center
Charlotte, NC

Gailen D. Marshall, Jr., MD, PhD, FACP, DFACCI FAAAAI
Department of Medicine
The University of Mississippi Medical Center
Jackson, MS

Daniel Peterson, MD
Clinician and Researcher
Founder/President, Sierra Internal Medicine
Incline Village, NV

Dr. Richard Podell, MD MPH
Clinician and Researcher
Podell Medical Practice
Somerset, NJ

Dr. William Mitchell, MD, PhD
Professor of Pathology, Microbiology and Immunology
Vanderbilt University Medical Center
Board of Directors, Hemispherx Biopharma
Nashville, TN

Connie Sol, PhDc
Clinical Exercise Physiologist
Institute for Neuro Immune Medicine
Nova Southeastern University
Ft. Lauderdale, FL

J. Mark VanNess, PhD
Professor of Health, Exercise Science and Bioengineering
University of the Pacific
Stockton, CA
Researcher, The Workwell Foundation
Ripon, CA

(1) http://www.iom.edu/Reports/2015/ME-CFS.aspx
(2) http://www.plosone.org/article/authors/info%3Adoi%2F10.1371%2Fjournal.pone.0031334
(3) http://www.nejm.org/doi/full/10.1056/NEJMp1211277

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