An FDA ruling was expected this month on the investigational “antiviral/immune modulatory” drug Ampligen® – potentially the first to be approved for treatment of ‘chronic fatigue syndrome’ (ME/CFS).
As of Feb 18, however, Ampligen’s maker (Hemispherx Biopharma) revealed that the FDA has delayed the decision date to May 25. At the same time, Hemispherx also announced that its Medical Director, Dr. David Strayer, will be presenting “new Ampligen clinical data” at the IACFS/ME Science Conference in Reno, NV, March 12-15.
Strayer’s presentation, scheduled in Session 1 of the Professional Meeting:
“Will include new data from the ongoing treatment IND/compassionate study (AMP 511) as well as the completed well-controlled pivotal Phase III study (AMP 516).
“Individuals suffering with CFS are known to be at greater risk than age-matched healthy populations with respect to certain catastrophic events including cancer, sudden cardio-vascular death and suicidal ideation. The new data will evaluate the potential of Ampligen… to mitigate certain of these events. Data utilized will integrate serially performed immunologic lab panels and EKGs, as well as physical performance scores and medical records (including concomitant medications used to alleviate certain symptoms of CFS).
“Ampligen®, an experimental product, may modulate nature’s primary gateway to immune response, mediated by receptors termed “toll-like receptors” (TLRs). Many other immuno-modulators may work downstream from the TLRs immune “gateways” at so-called “checkpoints.” Since “checkpoints” are not normally part of nature’s own immune-defense surveillance apparatus, these alternative “checkpoint” strategies may be associated with unintended consequences including significant toxicities.”
What Is Ampligen?
Ampligen (AMPLIfied GENetic activity) – an “orphan drug” under development & testing for 30 years and still allowed only in specific clinical trial settings conducted under U.S. governmental authorization – is termed “a nucleic acid drug,” designed to “modulate” the body’s immune system. Its mechanism of action in ME/CFS “is not entirely clear,” but it is thought to act on two enzyme systems so as to help the immune system destroy viral RNA and speed the death of virus-affected cells. In particular it may “downregulate” an anti-viral pathway which research suggests has become “upregulated” in certain ME/CFS patients (the 2-5 Synthetase/RNase L anti-viral pathway).
A “Who’s-Who” of the world’s leading ME/CFS specialists have participated in Ampligen trials over the years.