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Ampligen approval granted extension by FDA

snowathlete

Senior Member
Messages
5,374
Location
UK
Maybe it is difficult to prove its working, for whatever reason, and a test would help them prove it. If you can test for the illness, then treat and test again and its gone, then thats pretty solid(so long as the test is solid) and maybe thats cheaper than doing a massive trial and trying to show the results prove hte drug works. Perhaps because of the trouble in picking for sure, people who have the illness without doubt?

Just an idea.
 

Firestormm

Senior Member
Messages
5,055
Location
Cornwall England
Everyone loves a comeback story with a happy ending, but Hemispherx's second attempt won't turn out any happier than the first. FDA is likely to reject Ampligen again because Hemispherx ignored the agency's demand to run a new clinical trial in chronic fatigue syndrome.

Instead, Hemipsherx has spent the past three years doing essentially nothing but re-analyzing data from the old Ampligen phase III trial completed in 2004. FDA reviewed the original study already, deeming it "lacking credible evidence of efficacy of Ampligen."

It would be highly unusual -- unprecedented, even -- for FDA to reverse itself and find a retrospective rehash of old Ampligen data more compelling this time around. FDA has been facing political pressure to speed new drugs to market, particularly for diseases like chronic fatigue syndrome with no current treatments. But this hardly means FDA is going to throw out its drug-review rulebook.

FDA has scheduled an advisory panel for Dec. 20 to review Hemispherx's Ampligen resubmission. The agency is expected to issue a final approval decision on or before Feb. 2, 2013.

Ampligen's long odds haven't deterred speculators from bidding up Hemispherx's stock price, just like they did in 2009. At Wednesday's close of 90 cents, Hemispherx shares are up more than 220% since July 11 when the company announced plans to resubmit Ampligen for FDA approval.

For those that don't remember, the phase III study enrolled 234 patients with chronic fatigue syndrome, randomized to treatment with Ampligen or a placebo. The study's primary endpoint was improvement in treadmill exercise tolerance at week 40.

Hemispherx announced positive results from the Ampligen study in May 2004, claiming that Ampligen patients demonstrated a 17.4% improvement in treadmill exercise tolerance compared to a 4.3% improvement for placebo patients -- a net difference of 13.1%. The result was statistically significant with a p value of 0.047.

However, in May 2006, results from this same study were presented at a medical meeting showing a 12.9% difference in treadmill exercise performance between Ampligen and placebo that was not statistically significant...

Continues...

A postscript: Omitted from this column is a retelling or discussion of Hemispherx management's questionable actions and misleading statements made during the previous Ampligen review cycle in 2009. I covered Hemispherx extensively during this period, so feel free to go back and read my prior stories:
Hemispherx's CFS Drug Is a Long Shot
Hemispherx Builds False Hope on Old Data
Hemispherx, in FDA Limbo, Seeks More Cash
Hemispherx Hasn't Called FDA on Ampligen Review
Hemispherx Cops to Ampligen Delay
Hemispherx's Ampligen Dealt Fatal FDA Blow
--Written by Adam Feuerstein in Boston.

27 September 2012: The Street: http://www.thestreet.com/story/11720256/1/hemispherxs-ampligen-rehash-unlikely-to-impress-fda.html
 

Firestormm

Senior Member
Messages
5,055
Location
Cornwall England
There's a more positive analysis of Ampligen, Hemispherex and the need of a drug for CFS/ME here:

Can Hemispherx Find Success In The Chronic Fatigue Market?

Seeking Alpha: October 1st 2012: http://seekingalpha.com/article/898091-can-hemispherx-find-success-in-the-chronic-fatigue-market

It's easy to give up on Hemispherx Biopharma (HEB) and its flagship drug Ampligen. The Street's Adam Feuerstein has done a good job trashing the company and the drug. Initially, I wasn't very impressed either, but then I read many accounts of patients who took the drug and raved about how Ampligen greatly changed their quality of life.

Ampligen (rintatolimod) (poly C12u), is an experimental immunomodulatory double stranded RNA drug developed by Hemispherx Biopharma of Philadelphia, Pennsylvania. Ampligen is a toll-like receptor 3 (TLR3) antagonist based on synthetic double stranded RNA (dsRNA). Although chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME) is the main indication for this drug, Ampligen is also in early clinical trials as a vaccine adjuvant for breast and ovarian cancers and influenza.

There is a real need to find a drug that treats CFS/ME. According to the Centers for Disease Control and Prevention (CDC), more than one million Americans have CFS/ME. Others estimate that the CFS population is closer to four million adults in the United States, a number that is 10 times larger than that of multiple sclerosis (400,000), a condition with similar functional impairment, also of unknown etiology, but with nine FDA approved disease modifying agents. CFS/ME strikes more people in the United States than multiple sclerosis, lupus, and many forms of cancer.

Some research has estimated that 14.3% of CFS/ME sufferers have severe disease. Such patients are house-ridden, bed-ridden and when ambulatory may need to be in a wheelchair. CDC studies have shown that CFS can be as debilitating as multiple sclerosis, lupus, rheumatoid arthritis, heart disease, end-stage renal disease, chronic obstructive pulmonary disease (COPD) and similar chronic conditions.

The more severely ill patients die prematurely, often from organ failure, cancer and heart disease or suicide. Recently, the CDC and Harvard School of Public Health published an analysis based on a group of patients in Georgia. They calculated the economic burden for a CFS/ME sufferer both in terms of additional healthcare expenditures and lost earnings that they estimated at $11,780 per year per person. If it is assumed that the CFS patient population that is currently under care was one million, then the annual cost to society would be $11.8 billion. The full cost assuming four million CFS sufferers would be $47.1 billion per year...

...This study was initiated on December 17, 1998 and concluded on August 16, 2004. Although only 234 patients were enrolled in this study, HEB claims over 1,000 patients have received Ampligen and 90,000 have been doses administered. Some patients have been on Ampligen treatment for over five years.

THE HEB funded study was co-authored by HEB scientists, as well as some of the most respected names in CFS/ME research: Lucinda Bateman, MD, Derek Enlander, MD, Nancy Klimas, MD, Charles Lapp, MD, and Daniel Peterson, MD.

HEB hopes that the FDA's recently expanded statutory authority under the FDA Safety and Innovation Act ("FDASIA") may be relevant to the potential approval of Ampligen. Although there can be no assurance how the FDA will implement the new FDASIA provisions, HEB cites a September 13, 2012 teleconference wherein Sandra L. Kweder, M.D., Deputy Director, CDER Office of New Drugs stated that the FDA considers CFS/ME to be a "serious and life threatening condition" and that the FDA is "committed to making promising drugs available to individuals with serious diseases as quickly as possible for the rapid development and review of these types of therapy."

Section 901 of the FDASIA Act stresses the importance of 'expedited review' and the need to target subpopulations:
…the FDA should be encouraged to implement more broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life threatening diseases or conditions… This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation…​
…Patients benefit from expedited access to safe and effective innovative therapies to treat unmet medical needs for serious or life-threatening diseases or conditions.​

All the Ampligen speculation has caused a 190% 52 week change in the price of HEB stock which has soared from a 52 week of 17 cents on November 28 low to a high of $1.10 on September 24. At the Rodman and Renshaw conference (September 9-11, 2012), HEB reported having a market capitalization of $115 million, about $20 million in cash, no debt, and a burn rate of $10 million per year.

There will be two audiences closely monitoring news from the FDA on December 20 and February 2. Speculators who want Ampligen to win FDA approval so they can make a lot of money, and those who want the drug available because it may substantially improve their quality of life. I am not trying to pump HEB. I am recommending that you do a simple internet search of "Ampligen, Chronic Fatigue Syndrome" as part of your due diligence. You may be surprised to read the support the drug has in the CFS/ME community, which after all, is the market for the drug.
 

Shell

Senior Member
Messages
477
Location
England
I would love to believe that both Ampligen and Rituxin could be genuine answers for us - but I'm both cautious and cynical.
I did love that link to TheGlass Mountain blog. She talks of taking a 30 minute walk!!!! That little sentence alone is so wonderful!
Nevertheless Firestorm's observations leave me thinking there isn't as much hope as we might like.

As for either of these drugs being available of the NHS, I just can't see it. Apart from the egos that would be damaged the NHS is crap at funding proper medical care and spends it's money of managers and faff.
 

Firestormm

Senior Member
Messages
5,055
Location
Cornwall England
Morning Shell,

Yes I am unsure of what would happen in the UK if the FDA approves this drug. Would the MRC (?) then need to do a clinical trial themselves for example? I doubt that FDA approval is sufficient for other countries but it would mean something.
 
Messages
646
Morning Shell,

Yes I am unsure of what would happen in the UK if the FDA approves this drug. Would the MRC (?) then need to do a clinical trial themselves for example? I doubt that FDA approval is sufficient for other countries but it would mean something.

FDA is US only - and then of course there would be a question of whether/what Insurance would cover in the US. For the UK a licence would be required either from MHRA or EMA, both have their own strict criteria to meet. Safety studies carried out, outside of Europe may often be accepted, so long as those studes meet the relevant critieria, but the fact of FDA approval would not of itself be material to either MHRA or EMA approval. For the UK there is then the further test in respect of NHS availability and although that woud not directly impact private treatment, it does in practie influence what private insurance may cover.

Patient group lobbying for drug approval has long been an aspect of US Pharmaceutical marketing, a practice that has spread to Europe http://www.nytimes.com/2009/10/22/health/22nami.html?_r=0 and http://euobserver.com/news/29934 . It's not unknown for lobbyists to use the internet to present themselves as patients and act as cheerleaders for drug approval campaigns, the FDA has likely become somewhat resilient to patient lobbying because of doubts about the validity of what/who is driving patient demand.

IVI
 
Messages
646
As for either of these drugs being available of the NHS, I just can't see it. Apart from the egos that would be damaged the NHS is crap at funding proper medical care and spends it's money of managers and faff.
That's not an unexpected perspective for those of us who have conditions which the NHS doesn't deal well with - but we are a minority in a system created to address the mass needs of the majority. The NHS is and always has been the most efficient deliverer of health care of any large developed country. Not wholly current but a simple presentation http://www.commonwealthfund.org/Pub...omparative-Performance-of-American-Healt.aspx - New Zealand does better on cost overall but population size and age (young and healthy migrant population has a a major impact, plus lack of large cities and industrialisation) changes the context of delivery somewhat.

IVI
 

Snow Leopard

Hibernating
Messages
5,902
Location
South Australia
Morning Shell,

Yes I am unsure of what would happen in the UK if the FDA approves this drug. Would the MRC (?) then need to do a clinical trial themselves for example? I doubt that FDA approval is sufficient for other countries but it would mean something.

National bodies such as the MRC, generally do an internal review based on existing scientific evidence (including all published RCTs, no matter where they were conducted).

If Ampligen proves effective to many patients in the USA, the UK will have no choice but to follow. The alternative is to continue to endure the high economic burden of ME/CFS.
 

Firestormm

Senior Member
Messages
5,055
Location
Cornwall England
National bodies such as the MRC, generally do an internal review based on existing scientific evidence (including all published RCTs, no matter where they were conducted).

If Ampligen proves effective to many patients in the USA, the UK will have no choice but to follow. The alternative is to continue to endure the high economic burden of ME/CFS.

I dare say that pending clinical trials in the UK, and subsequent approval, NICE would also have to stick Ampligen in it's Guideline. And then we'd have to ensure that PCT's agree to fund and GPs agree to prescribe.
 

Shell

Senior Member
Messages
477
Location
England
I hope it all goes through at some point. My main concern is my children, if this turns out to be either familial or contagious. I would hope they would get better treatment and hope of a cure than us lot.
 
Messages
646
I dare say that pending clinical trials in the UK, and subsequent approval, NICE would also have to stick Ampligen in it's Guideline. And then we'd have to ensure that PCT's agree to fund and GPs agree to prescribe.
NICE has a duty to consider overall costs versus benefits, so Ampligen at current costs would be very unlikely to meet inclusion in Guideline criteria unless it was demonstrably currative or at the very least full pre illness functionality was achieved while treatment was ongoing. Even then as you suggest, local commissioning and prescribing practices would be further hurdles, which would be significant if there were to only be a low level of treatment responders. Ampligen could be demonstrated as both a safe and an appropriate treatment for M.E/CFS and therefore receives MHRA approval and still not be available on the NHS because of cost/benefit or low response rate issues.

IVI
 
Messages
5,238
Location
Sofa, UK
I think the note of caution is probably realistic, sadly, and the NHS is unlikely to approve either Ampligen or Rituximab in the next NICE Guidelines review, even if the FDA has approved it by then, and until there is more trial evidence including about cost/benefit/risk analysis I don't really expect approval. We will be in the ridiculous situation that talk therapies (CBT/GET) that have been proven ineffective for the vast majority of patients will be approved and promoted thanks to manipulated research papers and a relatively large volume of (underwhelming) published literature ("lots of evidence"), while drug treatments that have been proven effective will be unavailable due to a smaller volume of published research and the fact that these treatments - albeit they actually work - cost more than psych treatments and there's not enough published literature on the cost/benefit analysis.

However, I think that FDA approval of Ampligen would at least oblige the NICE Guidelines review panel to review it as an option and report on that. I think that being forced to look at this evidence would be an eye-opener for some: double-blinded placebo-controlled drug trials that show significant improvement are evidence enough to disprove any thoughts of psychological causation and I think this is evidence that has the power to change individual perspectives on the nature of ME/CFS. I would therefore hope that being obliged to review evidence for Rituximab and Ampligen would force a change in perspective of the NICE guidelines and I would hope at least for a fairly prominent mention of strong evidence of immune or autoimmune problems in many ME/CFS patients.

FDA approval of Ampligen would really open doors and raise eyebrows and I think it could be a huge step forward for us. We can press for UK trials of both Ampligen and Rituximab, and for more money for ME/CFS research in related areas of medicine (immunology), and with a half-decent campaign these would be hard arguments to resist given the historical low levels of research funding.
 

Shell

Senior Member
Messages
477
Location
England
FDA approval of Ampligen would really open doors and raise eyebrows and I think it could be a huge step forward for us. We can press for UK trials of both Ampligen and Rituximab, and for more money for ME/CFS research in related areas of medicine (immunology), and with a half-decent campaign these would be hard arguments to resist given the historical low levels of research funding.

Mark
I would like to believe that if the FDA do approve Ampligen and Rituximab that those sitting in the comfy chairs at NICE would rethink their love of GET/CBT and the whole psychi label thing.
However CBT is very fashionable in the NHS, regardless of the need or efficacy of it Hence the wait of over 14 months even for people who actually need it.

The funding for Inferon Beta never happened for us did it?

Is there even a dim glimmer at the end of the tunnel that getting Ampligen and Rituxin approved in America could eventually get something just a little cheaper like Inferon Beta funded here???
Frankly, with my 16 years working in the NHS and watching my dh as he continues (now 25 yrs) to work in the NHS, I have very little hope.

As time goes on and patients on these drugs reach remission or dare I even use the word "cure", then perhaps...And when Wessley and White are retired and gone, and politics and egos are at rest, then, just maybe....
 

Firestormm

Senior Member
Messages
5,055
Location
Cornwall England
More news and opinion from The Street:

Hemispherx ATM Withdrawals Raise A Red Flag

8th October 2012: http://www.thestreet.com/story/11730475/1/hemispherx-atm-withdrawals-raise-a-red-flag.html

No rant, just a warning. 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.


Didn't Peregrine Pharmaceuticals (PPHM) raise money through an ATM facility last spring?
Yes, Peregrine executives were talking publicly about the greatness of its lung cancer drug bavituximab, while in private, the company was selling millions of shares of stock for pennies through its ATM facility. We know how the Peregrine story turned out. Hemispherx is borrowing from the Peregrine playbook.

You mean, Hemispherx shouldn't be raising money now if the company has confidence in Ampligen?
Exactly. 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.

Small-cap biotech companies dilute shareholders all the time. Hemispherx needs money to develop Ampligen.
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.

inShare
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.
Hemipsherx is using an ATM to raise money ahead of the Ampligen FDA panel because Carter uses the company and its shareholders as a different sort of ATM -- an automated teller machine.

Of course it's 'only' an opinion. Personally I'd be doing exactly what the company appears to be doing: SELLING ME STOCK NOW LOIKE :)

Not that I'd get 92 cents like they managed. Typical.
 

barbc56

Senior Member
Messages
3,657
@Firestorm
This makes me want to read Ben Goldacre's book, Bad Pharma.

What I like about Goldacre is that he doesn't use sweeping generalizations but uses facts to back up his claims. He uses critical thinking skills/skepticism when he is investigating a subject and goes through the process of how he comes to holding his claims.

The bolding beow is mine.

The book is also available on Kindle.

From the intro:
Medicine is broken. And I genuinely believe that if patients and the public ever fully understand what has been done to them – what doctors, academics and regulators have permitted – they will be angry. On this, only you can judge.

We like to imagine that medicine is based on evidence, and the results of fair tests. In reality, those tests are often profoundly flawed. We like to imagine that doctors are familiar with the research literature, when in reality much of it is hidden from them by drug companies. We like to imagine that doctors are well-educated, when in reality much of their education is funded by industry. We like to imagine that regulators only let effective drugs onto the market, when in reality they approve hopeless drugs, with data on side effects casually withheld from doctors and patients.

I’m going to tell you how medicine works, just over the page, in one paragraph[1] that will seem so absurd – so ludicrously appalling – that when you read it, you’ll probably assume I’m exaggerating. We’re going to see that the whole edifice of medicine is broken, because the evidence we use to make decisions is hopelessly and systematically distorted; and this is no small thing. Because in medicine, we doctors and patients use abstract data to make decisions in the very real world of flesh and blood. If those decisions are misguided, they can result in death, and suffering, and pain.

This isn’t a simple story of cartoonish evil, and there will be no conspiracy theories. Drug companies are not withholding the secret to curing cancer, nor are they killing us all with vaccines. Those kinds of stories have, at best, a poetic truth: we all know, intuitively, from the fragments we’ve picked up, that something is wrong in medicine. But most of us, doctors included, don’t know exactly what.

I've always kind of cringe at the work skepticism as it may have a negative connotation but this is far from the truth. A definition of scientific skepticism:

A scientific (or empirical) skeptic is one who questions the reliability of certain kinds of claims by subjecting them to a systematic investigation. The scientific method details the specific process by which this investigation of reality is conducted. Considering the rigor of the scientific method, science itself may simply be thought of as an organized form of skepticism. This does not mean that the scientific skeptic is necessarily a scientist who conducts live experiments (though this may be the case), but that the skeptic generally accepts claims that are in his/her view likely to be true based on testable hypotheses and critical thinking.

http://scepticism.askdefine.com/

That's my 92 cents worth.:rofl:

Barb C.:>)
 
Messages
25
Maybe it is difficult to prove its working, for whatever reason, and a test would help them prove it. If you can test for the illness, then treat and test again and its gone, then thats pretty solid(so long as the test is solid) and maybe thats cheaper than doing a massive trial and trying to show the results prove hte drug works. Perhaps because of the trouble in picking for sure, people who have the illness without doubt?

Just an idea.
Ampligen is a total hoax, it does not work...Like one doctor said to me one day 'I would not touch Ampligen with a 10 foot pole' you can all wait or scream for Ampligen but you could not pay me money to take this 'lie'...All their research are fabricated lies and I have heard this first hand from former patients...Hem do not care for patients they are cold hearted people interested in increases in company stocks...Nancy Kaiser was the first cfids patient on Ampligen and she is dead today and she told me monster stories about this corrupt company from day one...If Ampligen was such a great spectacular medicine then why was the drug never given the green light on cancer...The biggest investor ever in Ampligen was the inventor of the fax machine and he is dead today and used Ampligen for his cancer but died...Besides if radiation is the actual cause of cfids then the science will prove Ampligen is useless because it will be up to radiation experts now to look at data with regards to Ampligen and say whether it will work or not...It does not work 'PERIOD'
 

heapsreal

iherb 10% discount code OPA989,
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10,089
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australia (brisbane)
Its probably not everyones cup of tea but people have improved on it. Just like rituximab.

My doctor told me his work collegue did a small trial on patients in australia in the 1990s, some got very sick on it and never really recovered from this set back. I dont know anymore about it then that.

Maybe back then and probably now, they need to select the right patients for the right drug with proper subsets and testing to select these patients.

Now i think if ampligen gets approved and if it helps a large or small number of people, cfs/me is then taken more seriously and recognised by the medical community and the government. Hopefully it spurrs researchers and big pharma to develop more drugs to help treat cfs/me and also those ones who dont respond to ampligen.

Thats what im hoping comes out of ampligen being approved.
 

Snow Leopard

Hibernating
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5,902
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South Australia
As is, it might only be approved for a subset of patients (eg severe patients). If they can swing $10 million around like this, then surely there is room for more research/additional trial?
 

snowathlete

Senior Member
Messages
5,374
Location
UK
Another positive factor in our favour (though it may not be enough) is that the first drug that is shown to work, even if only a small improvement, would be considered against the fact there are no alternative drugs yet. NICE do bare that on mind. Of course the biggest thing is that adding it to the guidelines would break their current world view of ME/CFS as a psychological illness and that may stop it happening full stop.

Once in the guidelines I don't think we will find it too difficult to get prescribed. It's getting it in there that will be the hard part. Of course if Lipkin or others find a bio marker or causive pathogen then that would help. Either way the UK will drag their heels and be behind the US.

We in the UK will have to campaign like we have never campaigned before if we want to speed things up once a drug is FDA approved.