Komaroff and his Ampligen Vote

Anthony Komaroff voted against the FDA approval of Ampligen. Now there is some huffing and puffing in the ME/CFS blogosphere about this (which I will not dignify with a link): Some are writing that he is removed from the ME/CFS patient population (and that could as well be from Mars, not from Earth). Some paint him as a sort of underhanded malicious villain (and presumably bought by someone).

Personally, I think this is utterly stupid.

There is an article posted on "Medscape Medical News" (via) that sheds some little light on Komaroffs decision:

Anthony Komaroff, MD, Simcox/Clifford/Higby Professor of Medicine at Harvard Medical School, Boston, Massachusetts, voted no on both efficacy and safety. "As a physician who has cared for many of these patients for nearly a quarter of a century, nothing would please me more than solid evidence of an effective therapy, but I think there are enough questions about the conduct of the studies that it does not meet the standard of adequate evidence."

Even if I don't know the details behind his decision, I think his explanation is reasonable. It is reasonable to vote agains a drug, if one thinks that there are questions about the conduct of the drug studies. And my impression is that Anthony Komaroff never says or does anything without having the facts to back him up. If he says the FDA should not approve Ampligen, we should better listen.

And just because there is no currently approved drug for ME/CFS, that does not mean we should accept badly done drug studies.

What if Ampligen does not work as we hope? What if its efficiency or safety are actually worse than what the patient anecdotes suggest? In that case an approval could do us some serious harm. Directly, by harming patients who receive the drug and by costing money needed for proper research. And indirectly because "we" lobbied for an ineffective and/or dangerous drug. This would play right into the hands of the psychobabblers.

And what is really awful, that we see like in 2010 with the XMRV-fraud the same stupid behavior by "patient advocates" and "patient activists", who treat critics like villains. Case in point in 2010 was John Coffin who (rightfully) criticized Mikovits, who said nothing but the truth (and that in a very polite and unassuming manner), and who in turn was portrayed by "patient advocates" and "patient activists" as if he were the baby-eating-anti-christ himself.

And now it seems that Komaroff is the target of this misguided anger.

So, let me tell all those stupid "patient advocates" and stupid "patient activists" out there:
It is you, who are malicious. By ignoring valid criticism. By ignoring the valid reasons behind the criticism. By ascribing malicious motives to critics, motives that only exist in your head.

With friend like these "patient advocates" and these "patient activists", we don't need enemies.

CFS, the cash cow that keeps on giving!

Not enough that we have Simon Wessely making a living out of treating CFS patients the right way (wink, wink), not enough that we have the CDC having their hand on CFS research money, not enough that we have the NCI having their hand on CFS research money, not enough that we have a lot of good friends like Harvey Alter, Judy Mikovits, Michael Maes and Kenny De Meirleir, no, we have a good old friend with Hemispherx having their hands on CFS research money!

http://www.thestreet.com/story/11730475/1/hemispherx-atm-withdrawals-raise-a-red-flag.html
…
Hemispherx used a Friday night SEC filing to disclose the sale of 10.9 million shares of stock at an average price of 92 cents per share. After expenses and commissions, Hemispherx net $9.5 million from the sale.

You said this stock sale was stealthy. Why?
…
An FDA advisory panel is scheduled to review the chronic fatigue syndrome drug Ampligen on Dec. 20. Presumably, a positive panel vote will push Hemispherx shares much higher than where they trade today. Yet, Hemispherx is rushing to raise money now. That's not a good sign.

Maybe Hemispherx wants to raise a small amount of money now and then plans to raise more money later, hopefully at a higher share price if the Ampligen panel votes to recommend approval.
…
Ha! That's funny. The FDA rejected Ampligen as a chronic fatigue syndrome therapy in late 2009. Yet for the past three years, Hemispherx has done nothing to advance Ampligen, including refusing to run a new clinical trial that FDA asked for. Hemispherx has plenty of cash to develop Ampligen. The problem is that management is hoarding cash to pay its outrageously high salaries, not to spend on Ampligen.

How much does Hemispherx CEO Bill Carter earn?

Carter was paid $1 million in salary in 2011. With bonuses, stock options and other perks, his total compensation for the year was $1.5 million. Carter's salary alone has doubled since 2009 -- the year that Ampligen was rejected.
…

I can hardly believe that!

It must be a lie!

Surely the invisible hand of the free markets will intervene! Let us all pray to the invisible hand of the free markets! Capitalism, in whom I trust, praised be your name, blessed be your trickle-down-economy!

Ampligen ("Rintatolimod") Phase III study published

Finally!

[Update: This was not a new study, just a re-analysis http://phoenixrising.me/archives/8882]

A Double-Blind, Placebo-Controlled, Randomized, Clinical Trial of the TLR-3 Agonist Rintatolimod in Severe Cases of Chronic Fatigue Syndrome

David R. Strayer 1*, William A. Carter 1, Bruce C. Stouch 2, Staci R. Stevens 3, Lucinda Bateman 4, Paul J. Cimoch 5, Charles W. Lapp 6, Daniel L. Peterson 7, the Chronic Fatigue Syndrome AMP-516 Study Group¶, William M. Mitchell 8*

1 Hemispherx Biopharma, Inc., Philadelphia, Pennsylvania
2 BCS Consulting, Philadelphia, Pennsylvania
3 University of the Pacific, Stockton, California
4 Fatigue Consultation Clinic, Salt Lake City, Utah
5 Center for Special Immunology, Fountain Valley
6 Hunter-Hopkins Center, Charlotte, North Carolina
7 Sierra Internal Medicine Associates, Incline Village, Nevada
8 Vanderbilt University School of Medicine, Nashville, Tennessee

Abstract

Background
Chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME) is a severely debilitating disease of unknown pathogenesis consisting of a variety of symptoms including severe fatigue.

The objective of the study was to examine the efficacy and safety of a TLR-3 agonist, rintatolimod (Poly I: C12U), in patients with debilitating CFS/ME.

Methods and Findings
A Phase III prospective, double-blind, randomized, placebo-controlled trial comparing twice weekly IV rintatolimod versus placebo was conducted in 234 subjects with long-standing, debilitating CFS/ME at 12 sites.

The primary endpoint was the intra-patient change from baseline at Week 40 in exercise tolerance (ET).

Secondary endpoints included concomitant drug usage, the Karnofsky Performance Score (KPS), Activities of Daily Living (ADL), and Vitality Score (SF 36).

Subjects receiving rintatolimod for 40 weeks improved intra-patient placebo-adjusted ET 21.3% (p = 0.047) from baseline in an intention-to-treat analysis.

Correction for subjects with reduced dosing compliance increased placebo-adjusted ET improvement to 28% (p = 0.022).

The improvement observed represents approximately twice the minimum considered medically significant by regulatory agencies.

The rintatolimod cohort vs. placebo also reduced dependence on drugs commonly used by patients in an attempt to alleviate the symptoms of CFS/ME (p = 0.048).

Placebo subjects crossed-over to receive rintatolimod demonstrated an intra-patient improvement in ET performance at 24 weeks of 39% (p = 0.04).

Rintatolimod at 400 mg twice weekly was generally well-tolerated.

Conclusions/Significance
Rintatolimod produced objective improvement in ET and a reduction in CFS/ME related concomitant medication usage as well as other secondary outcomes.

Ampligen approval granted extension by FDA

PHILADELPHIA, Jan. 11, 2012 (GLOBE NEWSWIRE) -- Hemispherx Biopharma, Inc. (NYSE Amex:HEB) (the "Company" or "Hemispherx") announced today that the Food and Drug Administration ("FDA") granted an extension for the Company to modify its New Drug Application ("NDA") in response to a Complete Response Letter ("CRL") received November 25, 2009 for the Chronic Fatigue Syndrome ("CFS") therapeutic indication. The extension will remain open while Hemispherx submits an amended NDA.

Dark clouds over Hemispherx and Ampligen

It looks like there are several problems with Hemispherx, the maker of Ampligen:

http://messages.finance.yahoo.com/Stocks_(A_to_Z)/Stocks_H/forumview?bn=27865

(Sorry, I don't link to yahoo, you have to copy the link manually)

Seems like the investors are not happy with the results of Ampligen. If one reads posts on that forum, it seems like efficiency and safety of Ampligen are a bit lacking in comparison to what they are supposed to do. Especially the posts by "deepdeepdeepthroat" are interesting:
http://search.messages.yahoo.com/search?.mbintl=finance&q=deepdeepdeepthroat&action=Search&r=Huiz75WdCYfD_KCA2Dc-&within=author&within=tm

By the way, even Jamie Deckoff-Jones MD gets mentioned:
http://messages.finance.yahoo.com/Stocks_(A_to_Z)/Stocks_H/threadview?m=tm&bn=27865&tid=440024&mid=440051&tof=17&rt=2&frt=2&off=1

Furthermore, Hemispherx is being traded at $0.19 per share (as of the time of writing), which would make it a penny stock in my view – YMMV. Then again, we have seen this since the eighties…

Don't believe anything you have read in the internet, but never hand-wave away this kind of criticism. Patients like Mary Schweitzer swear by Ampligen and I think it would be shame if would loose it. Yet I would like to see some solid studies from them.

Hemispherx/Chronix announce results in their CFS DNA-sequencing study

The aim of this recent CFS study was to find signature DNA sequences from patients with CFS compared to healthy controls with respect to their diagnostic predictive value, as well as, to potentially provide new insight into CFS biology. DNA extracted from serum samples of CFS subjects and normal healthy controls was sequenced and compared to the human genome. A total of about 10,000 high quality sequence reads were generated from each serum sample and four genes were identified by Multivariate Regression that separated CFS patients from the normal control group with a c-value of 0.95.

These results support additional studies with a larger CFS cohort using Massively Parallel Sequencing platforms with the aim of reduction to validated clinical assays for the diagnosis and evaluation of CFS and to explore whether the technology can be used to identify how different persons with CFS will respond to Hemispherx's experimental drug Ampligen.

Note: The blood test for CFS is still experimental in nature and has not been evaluated by any regulatory agency. It is currently limited to investigational use.

Previously, Chronix utilized Next Generation Sequencing (NGS) to generate sufficient DNA sequences to provide the statistical power to identify alterations in blood DNA from patients with breast and prostate cancer vs. normal healthy controls.