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FDA declines to approve Hemispherx's Ampligen new drug application

Discussion in 'General ME/CFS News' started by Lynn, Feb 4, 2013.

  1. JohnnyD

    JohnnyD Senior Member

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    SOC, they've been looking for a partner since at least 2009. No takers, partner wants approval first.
  2. JohnnyD

    JohnnyD Senior Member

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    Ampligen may have been invented decades ago, but it real terms it is a very recent drug and development is in its infancy. They now know, much of, the "Mechanism of Action". Toll Like Receptors, TLRs (the human body has about a dozen) were discovered around 2005. These are gateways to various aspects of the immune system. Ampligen is a TLR-3 agonist. TLR-3 is the viral gateway. Ampligen is the only specific TLR-3. Other TLR-3's activate other pathways, which they think makes them toxic. Much, but not all, of the discovery work about how ampligen works was done by the late Ralph Steinman, 2011 Nobel prize winner... who experimented on himself trying to cure his pancreatic cancer.
    Waverunner and Xandoff like this.
  3. Xandoff

    Xandoff Michael

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    Big Money, Big Pharma, their rules ............our misfortune.
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  4. Firestormm

    Firestormm Senior Member

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    Hi Jonny, could you do me a favour and shove a link in your reply to the HEB published accounts? Thanks. I figured I'd take a gander at what's what in the books. It's been a while since I did that sort of thing, but thought I'd enjoy seeing if I still can.

    I won't address your 'concern' about the FDA but I will say that they have recognised the seriousness of our disease. This was again apparent in their open letter reply posted I think above.

    And let us not forget who the members of the review committee comprised. At least one of those voting 'no' on both major counts was a (as some might now say) 'previously' supportive member of the community I seem to recall.

    These were not 'out to get us' or to snaffle our only hope. HEB are by your account 'cash rich'. Their executives following the review committee decision awarded themselves additional bonuses!

    As patients we are biased. Naturally. There's rancour in the States against federal institutions, perhaps less so in the UK, though I don't know maybe not when push comes to shove and patients are wanting the latest drug regardless of expense as has happened with MS and Cancer recently for example.

    With Ampligen I cannot see why they could not be more selective in their cohorts. If HEB know this drug of theirs inside and out and back to front - after decades of study - then why the hell can't they tell who is most likely to benefit?

    Surely 'immune profiles' would assist in this sub-categorising of patients from the CFS community? And quite how a drug for CFS could possibly also be marketed as being of use in AIDS let alone HIV is quite beyond my ability this evening to understand; but again, HEB must surely know.

    What is it about those patients who have taken Ampligen in Clinical Trial that has allegedly seen them 'recover'? Why is it so hard to ascertain this fact? Can't they see a 'before' and 'after' profile and compare to the controls?

    If Ampligen was 'good' for HIV/AIDS then surely they can explain why it is 'good' for CFS? Wouldn't that imply an aetiology? Leading to a biomarker of some description?

    There is one aspect of the rehash of data I think it was that sticks in my mind and that is the extra minute Ampligen permitted patients on the treadmill.

    I might be incorrect in my recollection of this - but bloody hell - is it any wonder better evidence was asked for? Again. What were HEB thinking? Did they honestly expect - after all these years - for the FDA to roll over and stamp it approved based on nothing new?

    I read Cort's article this morning, very briefly, and I see that he has also lambasted the FDA with nothing said about HEB in terms of criticism.

    I need to read his blog-thing again tomorrow and reply. HEB made money out of the speculation before the committee decision. They did not release the concerns raised by the FDA into the public domain. They pocketed $8 million from this speculation so far as we know... some shareholders are prosecuting them.... and you've said they have $40 million sitting in cash....


    But they don't have enough for a full clinical trial?!
  5. dannybex

    dannybex Senior Member

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    That was my thinking as well. Surely, some patients have improved dramatically, like Bob Miller and Mary Schweitzer. Yet Hemispherx thought they could get Ampligen approved based on a study that showed (I think less than) a minute improvement on the treadmill? For a drug that costs $2,000 a month?
  6. Waverunner

    Waverunner Senior Member

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    And that's the problem. Maybe there are a few patients, who get helped by Ampligen or other drugs. The only way to find out, is trial and error but if you limit trial to only certain drugs for certain people and if you impose gigantic costs for drug approval, you will lack trials and drugs and this is what we see today.
  7. JohnnyD

    JohnnyD Senior Member

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    Hi Firestormm,

    Here is a link to HEB's SEC filings.
    http://finance.yahoo.com/q/sec?s=HEB SEC Filings

    If you want details on current clinical trials, you can go here:
    http://clinicaltrials.gov/ct2/results?term=ampligen&Search=Search

    I agree it is a BIG step. But I'm a little jaded since it has taken them 20 years to do this. (I often wonder what the tipping point was?). and until they translate that into some real action - and I'm not just talking about stakeholders meeting, or meetings to teach patients how to advocate, I'm talking about meaningful action.

    If you are speaking of Komaroff, he represents conventional medicine. We're not talking about a conventional disease. The Aids epidemic would have gotten nowhere had there not been big challenges to the conventional.

    HEB is a small biotech, and they are far from being 'cash rich'. They've spent 200+ million on CFS. A 600 patient phase III trial for CFS would be very costly, literally taking all of their resources for a shot at what? Another gamble with the FDA? While I don't like high executive salaries, the fact is their salaries and bonuses are in line with industry. It is Carters invention - there would be no company and no drug without Carter. And hundreds patients would not have benefited... gotten a good portion of their lives back.

    The expense of the drug is a drop in the bucket compared to the cost of disability. 1-4 million patients equals 11-44 billion in lost revenue for the U.S. There is a doctor in Souix Falls, South Dakota who commutes to Incline Village, Nevada (1,500 miles) every week for ampligen infusions. This allows here to practice medicine 3 days a week. She's been doing this for a couple of years.

    Wow. Not sure where to start on this. Biotechs do not define the case definition. This is done by the CDC and other gov't agencies. And for CFS the Case definition is still evolving - see the CDC's "Multi-Center Assessment". for example, if my memory serves, in the late 1990's early 2000's when HEB did these trials 'Post Exertional Malaise' was not part of the case definition. It is now considered a hallmark. Having said that, the company spent plenty of it's money chasing XMRV testing and Chronix NGS testing. It's a challenging disease, a large unmet need - and yet no Big Pharma will touch it - but a small biotech who happened to be crazy enough to try is now being roundly criticized?? It sound to me like you have no rancour towards federal institutions. ;)

    You attribute way too much knowledge to a small biotech's abilities. Clinicians (and patients) would be the experts on immune profiles - someone like Dr. Peterson. You cannot see how a drug can be used for both HIV and CFS? Well, a retrovirus is still a top hypothesis for CFS - that's why Ian Lipkin, worlds greatest virus hunter is looking for them in CFS. This book is online: America's Biggest Cover-Up: 50 More Things Everyone Should Know About The Chronic Fatigue Syndrome Epidemic And Its Link To AIDS , here:
    http://www.fms-help.com/aids.htm

    Written in 1993, you'll find Chapter 46 most interesting.

    Who are you asking? Hemispherx says they met the endpoints of the two studies they submitted. They FDA agreed with them on the first study, yes the endpoints were met, but not the second study. the problem for the FDA is that for 15 years they've authorized a limited form of marketing called the Open Lable AMP511 in which hundreds of patients have been treated to positive effect. The leading advocacy groups, the patients, the patients families, the leading CFS clinicians - ALL know ampligen works for a subset of patients. In fact the FDA acknowledged that it words for a subset of patients at the AdCom and their briefing docs.

    Here is an interesting read for you from a former FDA commissioner, written one year ago. Where is this guy when you need him?

    http://www.raps.org/focus-online/ne...ugs-based-on-safety-prove-efficacy-later.aspx
    Wildcat, Firestormm and Valentijn like this.
  8. Firestormm

    Firestormm Senior Member

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    Thanks Johnny. Will take me a while to go through your points but I will. Just need a break following search for a home yesterday. Looking at property is rather draining - as is the thought of living independently once again. Although it's a pleasant prospect also.

    I saw this earlier from Research First (CFIDS): http://www.research1st.com/2013/02/...arch1st (Research1st)&utm_content=Yahoo! Mail

    A couple of letters embedded that they have sent. One to HEB suggesting applications for federal funding to keep this moving.

    I also noticed this from the HEB letter:

    It made me wonder exactly how many patients have ever taken and benefitted from Ampligen, either through this method (presumably not too many), or by being in specific trials.

    Thanks again for your points and for the SEC filing. I will take a look.
  9. barbc56

    barbc56 Senior Member

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    I could be misremembering but from the blogs I have read, it doesn't sound like patients are getting better. I understand Bob Miller did, but can anyone point me to a source for others? I think someone on here took Ampligen but didn't get better?
    Thanks.
    Barb
  10. BEG

    BEG Senior Member

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    Mary Schweitzer has also been helped by ampligen and has been very vocal about that and other ME/CFS issues at the CFSAC meetings. I'm not sure I've spelled her name correctly.
  11. JohnnyD

    JohnnyD Senior Member

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    Firestorm, from Hemispherx's February 4 PR:

    845 subjects who have received Ampligen®, including 589 subjects suffering from severe CFS and over 200 CFS patients who have received Ampligen® for at least one year or longer.

    Barb, plenty of CFS patients have gotten better... from modest improvement to dramatic That's why the Open Label trial (AMP511 referred to above) has stayed authorized for 15 years. And this is now a problem for the FDA as many many patients, family members, clinicians now know that ampligen works. Dr. Peterson has the most experience with ampligen... with CFS for that matter (in my opinion). From his testing and experience he can hit about 70% of those who will improve (from modest to dramatic). Of those patients, when they get off of ampligen, 1/3 continue to improve, 1/3 maintain the improvement and 1/3 decline and need to go back on again (Like Bob Miller). There is lots of patient testimony about ampligen to google on the net.
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  12. LaurelW

    LaurelW Senior Member

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    There's no real source for those who got better since Hemispherx doesn't release their data. I'm one of the lucky ones. But as far as treadmill duration being an endpoint for measuring the drug's effectiveness--Dr. Bateman said at the December FDA meeting that she has had some patients go back to work who still can't exercise. Obviously they need a better measurement. During the trial, we had to wear an activity monitor for a week two or three times throughout, and it seems that nothing ever came of that data. That would have been a better measure for me since the longer I was on the drug, the better I got and the more I was able to do.
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  13. BEG

    BEG Senior Member

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    Hi Laurel,

    What exactly was the activity monitor that you wore? Heart rate monitor? Step counter? Something else? Thanks, Brown-eye Girl
  14. Kati

    Kati Patient in training

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    Well, in my opinion, while Ampligen approval is important, we need to advocacte for biomarkers for our disease, mainly 10 folds increase in NIH research funds, and perhaps we'd be getting somewhere.

    I am curious to know if NK cell function improves in patients who are responding to Ampligen? Is it significant?

    The work of Simmaron, Phanu, OMI, Nova and the like needs to be put on steroids if you will.
    BEG likes this.
  15. LaurelW

    LaurelW Senior Member

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    I'm not sure. It was worn on the belt. Not a heart rate or step monitor.
  16. dannybex

    dannybex Senior Member

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    Hi Kati,

    I don't know if you've seen this study or not, but it suggests a possible biomarker -- something called a "CD-19 IgM lymphocyte" depletion/deficiency. I started a new thread on it:

    http://forums.phoenixrising.me/inde...l-to-81-of-cfs-patients-plus-biomarker.21745/
  17. Kati

    Kati Patient in training

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    No dannybex I didn't read it. Not planning to either because I have a strong bias against natural holistic health. At least I'm honest.
  18. dannybex

    dannybex Senior Member

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    Well, you're missing out on the results of the study -- 81% improved -- but of course that's your choice, and I respect it.

    I'm curious however -- why you're against anything natural? Nutrition isn't important in healing?
  19. Kati

    Kati Patient in training

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    dannybex, when I was 16 my mom was diagnosed with breast cancer. Her dr booked her for mastectomy and my mom never showed up. in the following 4years, she tried all of the natural therapies in the book including eating soup in the morning to cutting all sugar( because "it fed the cancer") to fasting to therapies to go to the root of her cancer, "her upbringing and her emotions". i watched all that, saw her become a skeletton while her tumor fungated out of her chest wall.

    She finally got convinced to do chemo by her siblings, and the grapefruit size, open tumor retracted and almost closed down, but to her it was poison and she stopped. She died.

    i am all for medical treatment and in my case I want to be treated agressively. i eat a healthy diet that includes meats and fish, fruits, veggies, grains, dairy and yes I eat chocolate and sweets once in a while.

    There is a market out there for natural products. i am not buying into it. I take the vitamins that have made a difference, from the grocery store, and dropped the ones who have not helped. i only take a few of them. iron, calcium/ magnesium, vit D, vit B, and B-12 (and before someone says it, I am awarethat Ca/MG and Iron are not vitamins)

    I don't detox and I don't do coffee enemas, and I refuse to juice or to buy organic just because it's supposed to be better for you.

    I do not believe that what I eat made me sick. I do not believe that what I eat will "heal me". Am sick. i need research, clinical trials and best evidence treatments
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  20. barbc56

    barbc56 Senior Member

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    Thank you, Kati. I wholeheartedly agree with you. My doctor has me on vitamins where I have a deficiencies and a couple that are shown in science based medicine to have some preventative effect, such as Vitamin D and calcium to help prevent osteoporosis.

    A balanced healthy diet is important but it does not have curative properties. Vitamins are important from the standpoint have a deficiency needs to be addressed. I like the analogy that a car can be tuned so it runs well but further "tuning" will not make the car run even better.

    Barb

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