Ampligen (one of two drugs effective against CFS)

FDA accepts antiviral drug AmpligenR for review as first-ever ME/CFS
(`chronic fatigue syndrome') therapeutic

ImmuneSupport.com

07-08-2008

July 8 – After 30 years in development and testing, the
experimental "antiviral/immune modulatory" drug AmpligenR has been
accepted by the FDA for review as potentially the first prescription
drug approved in the U.S. for treatment of ME/CFS – specifically for
certain patients with severe ME/CFS.

Delivered intravenously, typically twice weekly over a year or more,
AmpligenR (AMPLified GENetic activity) has been available in Belgium and Canada for ME/CFS and HIV treatment since 1996.

AmpligenR (polyI:polyC12U) - 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).

The drug's maker – Philadelphia-based Hemispherx Biopharma, submitted
a New Drug Application to the FDA in 2007, and had been asked to
answer a series of questions. The FDA's acceptance of the drug for
safety/efficacy review was based on receipt of the requested data.
The maker reportedly suggests it is also researching oral delivery of
the drug.

A "Who's-Who" of the world's leading ME/CFS specialists have
participated in AmpligenR trials over the years.
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To: All
From: Mike

At the last support group meeting (July 6th) we briefly discussed the
two new drugs approved by the FDA for the treatment of Fibromyalgia
(Lyrica and Cymbalta). I mentioned my feeling that the approval of
those drugs for FM pain made a huge difference in the perception of
the public and the medical community that Fibromyalgia was a real and
serious condition.
There is no similar FDA approved drug for the treatment of ME/CFS.
The one drug closest to approval is Ampligen. The article that I
posted says that Ampligen was "accepted" for study of efficacy and
safety by the FDA.
The process of getting approval by the FDA for a new drug is long,
expensive, and painstaking. Ampligen has been studied and tested for
over 30 years. Partly that's because the drug company making Ampligen
is very small and has a "checkered" history. They've almost gone
bankrupt a few times and they've never had success getting any drug
approved previously, that I know of.
Having said that, Ampligen is now on the FINAL step to getting FDA
approval. They've done the clinical trials, they've submitted an NDA
(a NEW DRUG APPLICATION), they've done everything the FDA wanted them
to do, to this point.
Now the FDA has to look at the results of the clinical trials done by
the drug company and decide if Ampligen is superior to placebo,
effective in treating CFS, what potential side effects there are, and
then render a verdict. This could still take months, but after 30 years we have never been this close to a drug approved specifically for CFS.
Mike
---

Ampligen, if approved by the FDA, would be a very expensive drug. I
think someone (Karen?) at the support group meeting said it ran a
couple thousand dollars per dose. That would still be the going rate
if it got approved by the FDA, so it's doubtful that many insurance
companies are going to jump to cover the cost of the treatments.

It is true that once a drug gets FDA approval, more doctors will feel
comfortable prescribing it to patients. If the drug begins to show
positive results, and doctors increasingly prescribe it, the cost of
the treatments will often go down. (Simple supply and demand, the
drug company doesn't have to charge as much per dose if they can sell
more of the doses.)

I don't think the average CFS/ME patient would be able to obtain
Ampligen at first. But I do think if the drug were to receive FDA
approval, that would be a huge psychological boost to patients with
this illness. As we've learned, the path to respect is slow, and once a drug is approved specifically for CFS it's another step to respectability. After all, how can there be a drug for an illness, if there's no illness? It MUST be REAL. RIGHT?

Keeping my fingers crossed.
Mike
* * *

According to "Osler's Web" (page 538) in 1991 Dr. Peterson observed "There's not a chance the Food and Drug Administration is going to approve this drug.  How can they approve a drug for a disease the NIH says doesn't exist?"

Dr. Cheney predicted they would delay approval as long as possible.  "They don't have the guts to kill it, because the patients will kill them.  But they don't have the guts to approve it, because Straus will kill them."

Straus is gone now, so maybe now, after decades of delays and excuses, we can finally get the best available drug to treat CFS.

As Mike points out, it's currently dreadfully expensive.  However, a lot of CFS patients were in good-paying careers before they were struck down.  If the government were willing to pay for me to get it for the first year or so, until I have enough improvement to go back to my former career, I would then be earning enough to cover the cost myself until retirement age.   Unfortunately, our government being pennywise and pound-foolish, I suspect that they would rather do without the next 20 years of my paying taxes on that good income than to pay the upfront costs of treatment that might get me back to work.