NEWSBRIEF: (Updated, Dec. 14, 2010) One year after the FDA rejected Ampligen (poly I:poly C12U), manufacturer Hemispherx Biopharma Inc. has asked for and received another year to resubmit data on its New Drug Application. The company now has until December 2011 to modify its 2009 application.
In late 2009, the FDA issued a Complete Response Letter saying Ampligen would not become the first ever drug approved for chronic fatigue syndrome -- at least, not yet. The letter listed several things the company could do to increase its odds of approval and gave them a year to do get those things accomplished. Now, Hemispherx says it needs more time because new information about chronic fatigue syndrome -- coming out of XMRV experiments -- could dramatically change the way clinical trials of the drug are designed, conducted and interpreted.
- Learn More: Ampligen
Some people have criticized Hemispherx's continued attempts to get Ampligen to the market. The drug has been in development for more than 3 decades and has been tested as a possible treatment for a host of different diseases. However, some people with chronic fatigue syndrome say it's the most effective treatment they've tried.
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The problems between Hemispherx (the makers of Ampligen) and FDA are, unfortunately, all too common with small companies.
FDA was created to work with an oligopolistic pharmaceutical industry (what we all call “Big Pharma”). For years, the companies were allowed to operate as effective monopolies by way of the patents granted on their new products – with a lot of those profits being plowed into research and development of new products. The system worked well.
And then FDA granted the industry permission to advertise directly to consumers. Economic studies have shown that the money that used to go into R&D now goes into glossy magazine ads and tv commercials for Viagra.
In the meantime, university professors looking for a way to profit from their discoveries were picked up in the 1980s by a movement to find “Venture Capital” for small, innovative companies. That’s what Hemispherx is – a venture capital biotech.
Unfortunately, FDA is not set up to deal with these small companies. The first double-blind done by Hemispherx around 1991 had really robust results – but FDA did not trust either the company or the disease (with CDC and NIH both insisting our disease had nothing that such a potent drug could fix). Hemispherx was sent off to do more studies to prove Ampligen was nontoxic, which they did, and then they had to run another double-blind, starting in 1998.
This second one was not nearly as successful as the first, because in the first one, Hemispherx had pretty much stuck to physicians working with cluster outbreaks (the purest form of the disease we have to work with in research).
In the second study, they used a simple treadmill test and a set of (frankly) Reeves-like questionnaires to diagnose the disease and then test its efficacy.
Some doctors already knew what they were doing and didn’t put anybody in the study who didn’t have biomarkers and other strong evidence of disease – but other doctors included in the study really didn’t have the foggiest notion how to diagnose the disease, and the questionnaires weren’t much help.
It has also not helped that the “placebo” – a sugar pill, as they say, in most studies – had to be IV saline – admittedly, only 400 ml administered twice weekly, but that could be enough to make some people feel better temporarily. The placebo wasn’t really a placebo, in other words.
Some of the sites – such as Salt Lake City and Incline Village – had very robust results, while others (such as one large study outside Philly) had terrible results.
FDA now complains about the studies with weak results, and the small placebo effect that was recorded at some sites.
It is within FDA’s power to approve Ampligen for fast-tracking (1997 law) – there is plenty of evidence that Ampligen meets that law’s requirements. But FDA says the company has never submitted the right forms. The company says they have.
I just pray that it can remain in the category of phase III trials, because without this drug (which I have to pay cash for, but am only allowed to as long as FDA says Hemispherx is making progress towards approval) – without this drug I become horribly sick. I am petrified of losing it again – I have lost it before and it means returning to living hell for me.
I know it does not work for everyone, but it does help many of us, and as a community we should stick together – evidence that an immune modulator makes patients better is good for all of us (as opposed to yet another CDC study insisting we’re all nuts).
It is also in everyone’s interests to get a chance to find out if Ampligen helps patients with XMRV (which I have tested positive for).
The evidence we already have – that it helps patients with low natural killer cell function and/or the 37kDa Rnase-L defect and/or active HHV-6 is shoved aside by CDC as part of their campaign to portray our disease to the public as having “no tests and no treatments.” But XMRV is getting public attention, so we need a chance to see if it is a fourth biomarker that predicts success with Ampligen – since CDC keeps telling FDA the other biomarkers are rubbish.
It’s not just us – FDA makes it hard for a lot of small companies with new medical products. How do you design a double-blind study for artificial skin? Yet they kept a good product off the market for years because they wanted testing that was impossible to do.
Let’s all hope that FDA continues to grant extensions for Ampligen, because we will no longer be allowed to get it, even by paying for it, if they are denied.
And I do not want to return to where I was a year ago, for a fourth time in my life.
Mary Schweitzer
Mary, I can’t imagine what you are feeling. It’s like being released from jail and then fearing that you have to go back. Not only are we stuck in a medical nightmare but we are also victims of politics and the CDC. No one understands how desperate our situation is.
You know what I think.
Thanks for writing this, Mary. I’m going for #3 starting next Monday. (thanks to my sister and her husband) Yep, starting Ampligen again after 7 years off. I’ve hit bottom….well, perhaps not bottom, as just when I feel that it can’t possibly get any worse, it does, and then, I remember……..best to forget those days, right? This will be the first time I’ve had to make a long drive and a bit frightened with this winter weather so it looks like those two days a week will be my only activity, at least for several months and then “life” returns!!!!!!! and just in time for spring and summer. HOORAY. Can’t wait.
Do you feel we really could have our lifeline taken from us?
I know, I know…..always the possibility, and we’ve dealt with this for years although now with THE virus, it seems less does it not? See you soon.
I’ve been so sick every time I do the Guinea Pig
Routine, I’m afraid I’m going to have to stick
with my medical status quo for now, until a
new drug isn’t new anymore.
My Fibromyalgia is getting worse by the year; I
think I know why, but I didn’t see that choice on
the poll.
In the meantime, I’m going to stick with the
medications I have, as they have alleviated
SOME of the debilitating pain. But, the Fibro
Fog slogs on, unabated.
I’m REALLY looking forward to the day (if it
ever gets here) when I can leap out of bed
and say gleefully (rubbing palms back and forth),
“What do I WANT to DO today?”