Ampligen "Death Blow" Another Tragedy for CFS Sufferers

In a shocking move, the FDA yesterday refused to approved Hemispherx's drug Ampligen.  It represents a devastating blow for Hemispherx who have in vain attempted to garner approval for Ampligen for over 20 years.  In their response letter, the FDA essentially demands that Hemispherx begin their clinical trials from scratch.  They site initial clinical trials fail to convince the review panel of Ampligen's efficacy, and that new clinical trials be conducted testing different doses for at least six months, on at least 300 patients, compared to the previous study which enrolled just 230 patients.  The FDA also goes on to cite unresolved manufacturing problems.  Considering that the previous study took 6 years to complete, approval doesn't appear likely any time soon, and likely won't be approved in other jurisdictions, as other nations have followed the guidance of the FDA, with a handful of exceptions of drugs that have gained approval by the European Medicines Agency before FDA approval is granted.

For persons suffering from ME/CFS, FMS, and Gulf War Syndrome it shows that government agencies continue to recognize these conditions as a real illness.  And research dollars earmarked for CFS/ME research gets misappropriated by CDC bureaucrats as a slush fund, where not one meaningful study has come out of it.  And it appears recent developments have not shifted the groupthink mentality of the CDC - but it will come a time when they will find themselves standing pretty well alone if replication studies on XMRV are in line with those of the Whittemore-Peterson institute, which I have reason to believe will be.

I believe that psychologically Ampligen represents a huge setback, but in the end it will amount to no more than a bump in the road.  Tests on antiretrovirals are going ahead full steam in pharmaceutical labs as we speak, and I've dug up a couple of other compounds that have demonstrated activity against other retroviruses: Lamivudine and emtricitabine - both have a far greater safety profile than Zidovudine (AZT), posing a low risk of anemia, nephrotoxicity, hepatoxicity, and pancreatitis.  I'm not particularly fond of Non-nucleoside Reverse Transcriptase inhibitors, due to their unfavorable side effect profile - distressing rash in as much as 20% of patients, and severe liver damage seen with Nevirapine and Etravirine - making patient compliance and adherance to treatment an issue in itself.  I also don't favor a single drug regiment - I would like to see a combination pill of either Lamivudine or Emtricitabine with raltegravir.

When Can Patients Expect Approved Therapies?

The availability of effective therapies for ME/CFS, FMS, and Gulf War Syndrome is still quite a ways off. Pharmaceutical companies have expressed a great interest in screening currently available HIV therapies, and compounds that did not pan out for treating HIV.  Likely it will be four years before the FDA approves any existing drug for treating XAND disorders - By next summer a number of drug candidates that work against XMRV will be selected for Phase II clinical trials (A Phase I trial is not necessary for a new indication), followed by a larger scale Phase III trial, which will likely not be completed well into 2012, with approval in 2013!  For new chemical entities, it will be even longer - at least that long for protease inhibitors (purifying the protein, doing X-ray crystallography studies, and finding small-molecule inhibitor candidates), then starting from scratch with phase I studies - what is going to turn out to be about a 10 year process, and likely the FDA won't grant accelerated review.

It's not to say that when results from ongoing studies begin to surface, that doctors won't begin the label of practicing current HIV drugs off-label to XAND patients.  However, such patients will have to shoulder the cost of treatment themselves, as insurers will be very reluctant to cover a drug which is not indicated for a particular disorder by the FDA.  High drug costs in the United States, which has been decried as criminal by AIDS activitists, will present a formidable barrier to most people.  In some countries with publicly funded health care systems, these drugs will be even harder to obtain: HIV patients have programs where drugs are given free of charge at clinics, and are not available in community pharmacies.  In some jurisdictions like British Columbia, which has a publicly funded drug plan, despite being approved for use by the federal government, the drug plan will do time-consuming studies which last on the order of 2 or more years, further delaying availability of treatment - so much for universal health care!