A Twisted History – Twenty years ago advocates were lobbying for ‘fast-track’ status for the new wonder drug they hoped would cure the disease. Ampligen never had a chance of being fast-tracked given the patient population wasn’t dying in large numbers but no one ever thought 20 plus years later the fate of the drug would still be unclear. Ampligen’s manufacturer, Hemispherx Biopharma, has been seeking FDA approval on and off since 1988 but the only patients currently able to use the drug are patients in the drug trials or very ill patients who have been able to use it on a ‘cost-recovery basis’. Ampligen has been available for use in both Belgium and Canada (under its emergency drug release program) since 1996.
With Friends Like This… Hemispherx Biopharma, the small company that produces the drug, and it’s founder, Dr. William Carter, have Ampligen (poly I:poly C12U) have been embroiled in lawsuits throughout much of its existence. Throughout his career Dr. Carter has been a lightning rod for controversy with dismissals, accusations and lawsuits dotting his time with different companies.
Some of the allegations have been remarkable; one sick chronic fatigue syndrome (ME/CFS) patient sued Carter claiming he required him to buy $1,000,000 in Hemispherx stock to order to participate in a study. (He later dropped the lawsuit). Dr. Carter has twice sued former employers (once successfully) to get his job back. Patients in the chronic fatigue syndrome drug trials successfully sued after Hemispherx denied them the drug after the trial ended. (For a hilarious and often heartrending ‘fictional’ account of the early Ampligen trials read Patient 002 by Floyd Skloot.)
With a 30 million dollar infusion from DuPont, Dr. Carter and Hemispherx began clinical trials in Ampligen in chronic fatigue syndrome (ME/CFS) in 1988. Trouble ensued and in 1990 Dupont settled (what else) a lawsuit with Hemispherx and gave up all claims to the drug. Possibly hemorrhaging money after a major financier died, Hemispherx cut back a 48 week trial to 24 weeks and, according to one report, applied for fast-track limited drug approval at the FDA. As the media spread accounts of a wonder drug that significantly helped many patients, the FDA, citing toxicity concerns and calling the data before them ‘incomplete and inadequate’ (and not happy at all about the media attention) nixed the fast track status and told Hemispherx to stop making unproven claims. Hemispherx then turned its attention away from getting approval for the drug in the U.S. It would be almost 20 years before Ampligen would finally near the end of the treatment trial road at the FDA.
Despite producing a drug that has substantially helped some chronic fatigue syndrome (ME/CFS) patients, Hemispherx has gathered few friends in the past 20 years. In the early 1990s Kim Kenney (McCleary), now the President of the CFIDS Association of America , said “Ampligen is a good drug in the wrong hands”. Daniel Hoth, then head of the National Institutes of Health’s AIDS drug program, went further when he told the Wall Street journal that “no professional drug company with any degree of professionalism would ever develop Ampligen the way it was developed by HEM.”
Indeed the drug trial has been going on for so long that Hemispherx is reportedly tied to an obsolete definition for the disease (Holmes) and an out of date statistical package. Advocates and physicians have questioned what kinds of patients are in the studies, the markers Hemispherx is using to chart the patients’ progress, and the overall competence of the company.
Whatever mistakes have been made, Hemispherx has its advocates as well. Ampligen is still the only drug that has undergone clinical trials for this controversial disease. While many fervently wish a different company had taken over the drug years ago, others applaud Dr. Carter’s tenacity for sticking with it through thick and thin for over 30 years. That the drug is still alive and may, in fact, be on the edge of approval, may be something of a miracle given its history, the disease it treats, and the high failure rate of drugs in general.
Recently Hemispherx appears to have gotten its financing and legal house in order. In 2009 Carter reported that all legal issues had been settled leaving the company free to focus on the drug itself. Ampligen has completed Phase II and III clinical trials. After having missed the deadlines in 2005 and earlier in 2007, Hemispherx filed its ‘New Drug Application’ (NDA) with the FDA in October, 2007. The FDA bounced back the application with questions in early 2008. Hemispherx replied and after both the drug and the company went under further reviews the FDA passed the drug onto the final review.
The Ampligen Series on Phoenix Rising
- Ampligen – What it is, how it works, what doctors say, etc.
- Twisted History – Ampligen’s long and tangled road with ME/CFS
- Dr. Lapp Interview – Kelvin Lord interviews long-time Ampligen provider, Dr. Charles Lapp, on Ampligen and other treatment options
- The Ampligen Blogs – Follow Kelvin Lord as he documented his year-long experience with Ampligen
A Big Blow – In what journalist (and Hemispherx critic) Adam Feuerstein called a ‘staggering blow’, in Dec. 2009, the FDA told Hemipherx that it could not approve Ampligen’s use in ME/CFS until the following issues have been settled. Hemispherx needed to
- Show credible evidence of Ampligen’s efficacy using expanded studies that are at least six months long
- Show that Ampligen is safe; ie that it does not cause autoimmune disorders or heart problems (prolonged qt intervals)
- Produce studies in which patients are on more than one dose regimen
- Produce studies in which at least 300 patients are on doses intended for the market
- Follow the FDA’s ‘recommendation’ that rodent studies be done to examine carcinogenicity
- Provide additional data on quality control issues
- Fix Inspection issues at one of Ampligen’s facilities
The FDA asked a lot for a small company when it asked for an expensive 300 person study and Hemipherx stock prices plunged 43% on the news. A strong skeptic, journalist Adam Feuerstein noted that Hemispherx has unsuccessfully attempted to prove the drug works in a ‘dizzying’ array of diseases (hepatitis B and C, smallpox, HIV, ebola, avian flu , swine flu- HINI) and predicted the drug would never be approved. (Shortly after the XMRV Science paper, in an ill-fated move, Hemipherx embarked on studies attempting to prove Ampligen efficacy in treating XMRV infected patients.) Feuerstein suggested the company might not be able to financially meet the FDA’s demands.
2012 Study – Feuerstein may have been right. While a 2012 study did provide more evidence of efficacy and more safety data, it was based on an analysis of data from a 1998-2004 study. The FDA, however, rejected Hemispherx’s claims of Ampligens efficacy in earlier studies. This study may help but while it was larger study (@ 200 people) it wasn’t able to met the FDA’s requests for a 300 person study or to examine the effects of multiple dose ranges or do rodent studies.