Ampligen On the Clock: Hemispherx’s ‘Complete Response’ Means Ball is Now in FDA’s Court Now

August 2, 2012

Posted by Cort Johnson

Hemispherx's Ampligen Response to FDA means the clock is ticking for the drug

By end the January, 2013, at the latest,we will know if the FDA will approve Ampligen for ME/CFS...

Moving quickly, Ampligen’s producer, Hemispherx Biopharma,  filed its ‘complete response’ to the FDA’s request for data just 53 days after the agency agreed to relax its requirements for review of the drug.  Hemispherx’s ‘complete response’ means we will know by the end of January and perhaps sooner whether Ampligen becomes the first FDA approved drug for chronic fatigue syndrome (ME/CFS).

New Data

In its response Hemispherx provided new data derived from a 24 week extension period occurring after the 40 week original trial.  Hemispherx stated the data will show that patients who remained on the drug for a further 24 weeks showed further improvements in endurance (23% – 39% increase in treadmill duration) suggesting that the drug’s effectiveness increases over time.

Similarly, Hemipherx reported that patients who responded well to Ampligen showed significantly greater improvement relative to non-responders in areas such as vitality, general health, Karnofsky performance (functionality) and activities of daily living  the longer they were on the drug.   This is important because the original study did not highlight improvements in functionality and well-being. This data suggests Ampligen works ‘globally’ to improve both endurance and functionality.

FDA and Ampligen

It appears that the FDA has at least several possibilities;

  • it can approve the drug
  • it can reject the drug
  • it can give the drug ‘accelerated approval’ status – in this scenario Ampligen is not approved but  Hemispherx is allowed to market the drug and gather more data for a final attempt at approval
  • it can ask Hemispherx for more data – without the income derived from ‘accelerated approval’ this outcome would be difficult for Hemispherx.
Hemispherx’s Press Release suggests the company is gunning for accelerated approval status. While the FDA did recently substantially relax its requirements to consider Ampligen for approval its possible, perhaps even probable, they still want that large, multi-dose, placebo-controlled, double-blinded treatment trial originally required in their 2009 report. Hemispherx didn’t come anywhere near providing that kind of data but getting accelerated approval would give them the means to do so.
 The FDA has increasingly come under fire  to approve more drugs and, in particular, to approve more drugs for chronic illnesses and  the new law provided drug companies an opening to do just that.

New Law Presents Opportunity for Hemispherx and Ampligen

In their press release Hemispherx highlighted the fact that the 2012 Food and Drug Safety and Innovation Act (FDSIA)  law encourages the FDA to take ‘innovative and flexible approaches’ to assessing treatment for ‘serious’ disorders with ‘unmet’ needs.

In section 901 from the FDASIA Act below note the emphases on ‘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.

Indeed, recent history suggests a drug need be effective in only a small group of patients if if few treatment options are present.  With FDA analyses suggesting that 11 lupus patients needed to be  treated on Benylstra to find one the drug helped, and with the drug showing marginal effectiveness overall, but with the last FDA approved drug for lupus dating back 50 years, Benylstra was easily approved. (Benlystra significant helped those few patients which responded.)

Hemispherx’s production facility at New Brunswick will be undergoing ‘pre-approval inspection’ as part of the FDA review process. If the drug is approved or granted accelerated approval status Hemispherx should, hopefully, be able to hit the ground running.  Could the drug be widely available to ME/CFS patients sometime next year? Time will tell.

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46 comments

{ 46 comments… read them below or add one }

Firestormm August 2, 2012 at 12:31 pm

I'm not optimistic about Ampligen, but if it is approved by the FDA does it then mean that any physician is able to prescribe it or will this also depend on subsequent approval by someone else? Not sure how it works over the pond.

Of course in the UK we'd need NICE to also agree that it was an effective treatment option for CFS patients I would suspect before any doctor could prescribe it specifically.

Thus far it hasn't to my knowledge attracted comment from the UK authorities. I guess like in many other things we're awaiting the USA to take the plunge.

justinreilly August 2, 2012 at 3:20 pm

Great news! Very glad to see they are on the ball and there is new data showing such effectiveness! What a godsend it would be if it were approved, both for treatment and for our 'credibility.'

Yes, once FDA approval is granted then any doctor can prescribe it for any condition he sees fit. If it is approved, it will be approved specifically for ME, meaning that it can be advertised and marketed as treating ME (they won't be able to do so for any other condition, though a doctor would be free to prescribe it for another condition). And it will be possible, but not a sure thing i think, that insurance companies will cover it (they will not cover medicines for conditions for which the drug has not been specifically approved).

Firestormm, why are you not optimistic (about the drug itself or just approval?) about ampligen? i will probably start taking it in about a year with Enlander or Cheney once i'm done with gcMAF.

The stars seem to me to be fairly well aligned for approval, but i don't know a lot about ampligen.

Thanks, Cort for the info.

justinreilly August 2, 2012 at 3:21 pm

Great news! Very glad to see they are on the ball and there is new data showing such effectiveness! What a godsend it would be if it were approved, both for treatment and for our 'credibility.' knock on wood!

Firestormm, Yes, once FDA approval is granted then any doctor can prescribe it for any condition he sees fit. If it is approved, it will be approved specifically for ME, meaning that it can be advertised and marketed as treating ME (they won't be able to do so for any other condition, though a doctor would be free to prescribe it for another condition). And it will be possible, but not a sure thing i think, that insurance companies will cover it (they will not cover medicines for conditions for which the drug has not been specifically approved).

Firestormm, why are you not optimistic (about the drug itself or just approval?) about ampligen? i will probably start taking it in about a year with Enlander or Cheney once i'm done with gcMAF.

The stars seem to me to be fairly well aligned for approval, but i don't know a lot about ampligen.

Thanks, Cort for the info.

Cort August 2, 2012 at 3:22 pm

If it is FDA approved then any doctor in the US, to my knowledge, can prescribe it for ME/CFS. Whether the insurance companies will pay for it is another story. Cost of scale factors should, I would think, push the price down, though, I would think and approval would mean more money for studies which should make it harder for insurance companies not to cover.

Bob August 2, 2012 at 4:15 pm

Thanks for the info Cort.

MishMash August 3, 2012 at 12:01 am

Hemispherix makes Immunovir. It was prescribed to me. It isn't covered by any insurance, you have to order it from Canada, and I didn't notice any benefit after taking it for a long time. I hope Ampligen works better.

Firestormm August 3, 2012 at 1:49 am
justinreilly

Great news! Very glad to see they are on the ball and there is new data showing such effectiveness! What a godsend it would be if it were approved, both for treatment and for our 'credibility.' knock on wood!

Firestormm, Yes, once FDA approval is granted then any doctor can prescribe it for any condition he sees fit. If it is approved, it will be approved specifically for ME, meaning that it can be advertised and marketed as treating ME (they won't be able to do so for any other condition, though a doctor would be free to prescribe it for another condition). And it will be possible, but not a sure thing i think, that insurance companies will cover it (they will not cover medicines for conditions for which the drug has not been specifically approved).

Firestormm, why are you not optimistic (about the drug itself or just approval?) about ampligen? i will probably start taking it in about a year with Enlander or Cheney once i'm done with gcMAF.

The stars seem to me to be fairly well aligned for approval, but i don't know a lot about ampligen.

Thanks, Cort for the info.

Morning Justin,

Good to see you posting. Been a long while.

Thanks for that (and from Cort too). I wasn't sure of the process over there.

I think we have had several threads on Ampligen now and I expressed my concerns again I think when this last study was published. Those were based on that study, but I guess I am still feeling that Ampligen like (what I know of) GcMAF was something that searched for something to treat and I'm still not convinced of it's ability to do whatever it now says on the tin in the way that it says it. But if it's approved then we can all give it a whirl I suppose. Whether or not that approval would extend to the UK I really don't know. We'll see I guess.

Apologies for not being as enthused. I hope it lives up to all expectations :)

Bob August 3, 2012 at 6:18 am

Does anyone know if there are any simple stats to read on the results of any Ampligen studies that I can have a look at without having to read through whole papers?

Cort August 3, 2012 at 7:07 am
MishMash

Hemispherix makes Immunovir. It was prescribed to me. It isn't covered by any insurance, you have to order it from Canada, and I didn't notice any benefit after taking it for a long time. I hope Ampligen works better.

Revex makes Immunovir – http://www.rivexpharma.com/products_imunovir.html – Ampligen has undergone quite a bit of study…It's Peterson's best drug at the moment…I would be surprised if its not more effective than Immunovir…

Bob August 3, 2012 at 7:44 am
Cort

Revex makes Immunovir – http://www.rivexpharma.com/products_imunovir.html -

Thanks for clearing that up Cort. I didn't think Immunovir was made by Hemispherx .

Cort

Ampligen has undergone quite a bit of study…It's Peterson's best drug at the moment…I would be surprised if its not more effective than Immunovir…

But we need to see proper blinded medical trial data so we know what to expect from Ampligen.
Your recent long article said: "Dr. Peterson said that after going off Ampligen about a third of patients actually get better, a third maintain their improvement and a third eventually slide back."
This is good news, but it's not a controlled trial.

And does Peterson use it on all of his patients, or only a subset? (i.e. does it only work for a certain subset of CFS/ME patients?) I thought I read that Peterson only uses Ampligen on a small subset of his patients, but I just had a look for that info and couldn't find it, so maybe I've got that wrong.

satoshikasumi August 3, 2012 at 7:52 am

Ampligen's effects:
-improvement in VO2 max and increased ability to exercise on a treadmill
-ability to improve daily functioning, particularly in the most severely disabled and bedridden patients who do not respond well to symptomatic therapy or cognitive therapy
-reduced usage of medications that treat individual symptoms
-improved cognitive function- in the severely ill, who may have a form of dementia, this includes both improved working memory and normalization of performance IQ
-improved natural killer cell function
-control of herpesvirus reactivation- patients who have consistent positive PCRs for HHV-6 or EBV will have these tests come up negative shortly after starting Ampligen
-complete elimination of the defective 37kd form of Rnase-L and normalization of the overall level of Rnase-L in patients who have abnormal Rnase-L levels. (Rnase-L is part of the interferon pathway, our body's natural antiviral control system)
-some improvement is noticeable after 6 months, but it can take up to 18 months for full benefit
-dosage can be very tricky
-some patients do not respond to Ampligen
-Ampligen can cause flu-like symptoms like chills, headache, muscle pain, and increased fatigue for up to 24 hours after each infusion, but Ampligen appears to be safer than other drugs being considered to treat CFS, such as rituximab and valcyte

Cort August 3, 2012 at 9:25 am
Bob
Cort

Revex makes Immunovir – http://www.rivexpharma.com/products_imunovir.html -

Thanks for clearing that up Cort. I didn't think Immunovir was made by Hemispherx .

Cort

Ampligen has undergone quite a bit of study…It's Peterson's best drug at the moment…I would be surprised if its not more effective than Immunovir…

But we need to see proper blinded medical trial data so we know what to expect from Ampligen.
Your recent long article said: "Dr. Peterson said that after going off Ampligen about a third of patients actually get better, a third maintain their improvement and a third eventually slide back."
This is good news, but it's not a controlled trial.

And does Peterson use it on all of his patients, or only a subset? (i.e. does it only work for a certain subset of CFS/ME patients?) I thought I read that Peterson only uses Ampligen on a small subset of his patients, but I just had a look for that info and couldn't find it, so maybe I've got that wrong.

The original trial was blinded but it used one dose throughout which sounds like it was a mistake. Nevertheless positive findings regarding endurance and functionality were found. I think a study with multiple doses – each tailored to the patient – as Dr. Peterson does – would get much better results. They also need to figure out who really benefits; its going to be a subset – identifying that subset would be very helpful.

SpecialK82 August 3, 2012 at 9:58 am
Cort

If it is FDA approved then any doctor in the US, to my knowledge, can prescribe it for ME/CFS. Whether the insurance companies will pay for it is another story. Cost of scale factors should, I would think, push the price down, though, I would think and approval would mean more money for studies which should make it harder for insurance companies not to cover.

I believe any doctor could prescribe it, but I wonder in practice how many would when they don't understand the illness at all. Would they be comfortable prescribling something with potentially serious side-effects for an illness that is basically unbeleiveable? I think that will be an obstacle………but the fact that it is approved by the FDA would lend a ton of credibility to this illness, and I think over time, physicians will learn to take it seriously. Wouldn't it be fantastic if Hemispherx started running commercials for Ampligen on TV, CFS/ME would become household words just like has happened with Fibromyalgia.

Ampligen is administered by IV twice a week, so I'm not sure how a run-of-the mill Family Doc would administer without an IV suite. Maybe they could prescribe and then patients could go to their local hospital for infusion??

Also, as I understand it, Ampligen currently is made by hand in small batches. Hemispherx was having a significant problem (as of a couple of years ago) converting the process into larger scale production. I don't know that they have solved these issues……..let's hope so.

On the insurance front, surprisingly, I know of one big insurance company that was planning on covering Ampligen at the beginning of 2011. I happened to carry that insurance but I stopped infusions right before that time so I don't know for sure that it happened, I still think there were some issues being ironed out. I know that Hemispherx is pushing the insurance industry on it.

Firestormm August 3, 2012 at 11:46 am

Ampligen is administered by IV twice a week, so I'm not sure how a run-of-the mill Family Doc would administer without an IV suite. Maybe they could prescribe and then patients could go to their local hospital for infusion??

Thanks. I hadn't remembered/didn't know that. Yes that would be a considerable problem should this drug find it's way across to the UK I should think for General Practitioners anyway.

Sasha August 3, 2012 at 12:00 pm

Years ago I tried Vit C infusions and had to pay privately to go to some local place – a nursing home? Can't actually remember – to have it done several days in a row. Just needed a bed to lie down on (because I was bedbound) and that metal stand thing that you see on TV for the drip to hang on, and the bag of drip, and a nurse to stick the needle in. It didn't seem very complicated or to involve any big-deal equipment but maybe I just wasn't noticing. I'd be surprised if this wasn't something that could be done easily in a GP's treatment sideroom but perhaps that's just my ignorance speaking!

And if it was on the NHS (unlike my Vit C) we shouldn't have to pay privately.

Hope123 August 3, 2012 at 7:14 pm

As far as I know, Immunovir is not manufactured by Hemispherix but by Rivex, a Canadian company that is urelated. They do sound alike though.

My take based on reading and talking to people is that Hemispherix probably works for subset of CFS patients but many will likely need to take it regularly and continuously for a benefit. But what that subset is is not known publicly and is not in the papers; perhaps some docs have a sense of who would benefit. The docs that I know who use it do not suggest it for everyone.

More generally, I have problems with the outcome measures that Hermispherix uses (statistically significant does not equal clinically significant) and safety issues. The company is not as forthcoming with the latter as they could be; there may be more behind the story than what is out in the public eye. Regardless of efficacy, safety does need to be considered.

Overall, I think there will be better drugs than Ampligen, safety and efficacy-wise, in the near future and I would caution people to not push for a "political victory" without regard to safety.

Cort August 4, 2012 at 5:23 am
Hope123

As far as I know, Immunovir is not manufactured by Hemispherix but by Rivex, a Canadian company that is urelated. They do sound alike though.

My take based on reading and talking to people is that Hemispherix probably works for subset of CFS patients but many will likely need to take it regularly and continuously for a benefit. But what that subset is is not known publicly and is not in the papers; perhaps some docs have a sense of who would benefit. The docs that I know who use it do not suggest it for everyone.

More generally, I have problems with the outcome measures that Hermispherix uses (statistically significant does not equal clinically significant) and safety issues. The company is not as forthcoming with the latter as they could be; there may be more behind the story than what is out in the public eye. Regardless of efficacy, safety does need to be considered.

Overall, I think there will be better drugs than Ampligen, safety and efficacy-wise, in the near future and I would caution people to not push for a "political victory" without regard to safety.

I hope there are better drugs out in the near future but I would be surprised if there are significant safety issues with Ampligen as Dr. Peterson said in a recent interview with Llewelyn King, I think, it was…that it is safe and effective. He's been giving it to patients for over 10 years…

Some people seem to do well after Ampligen – they stay at their level of wellness – but others relapse. It can take a couple of years for them to relapse or it can happen more quickly…

I see Ampligen as a bridge to something better; once they can start studying it in more detail they'll know better why it works in some patients and not others; that will give researchers a leg up on the next drug…but its got to get in there first….

LaurelW August 4, 2012 at 11:11 am

I agree with Cort, it would be really nice if they could figure out who would benefit from the drug. But if it was covered by insurance, a lot more people would be able to try it and find out, in the absence of defining biomarkers.

Infusing Ampligen is no big deal, like Sasha said. You just need an IV pole, a nurse to do the infusion, etc. The most complicated part is that it has to be kept refrigerated until use and taken out an hour or so beforehand so it's not too cold. And a good IV nurse is pretty indispensable, but people can be trained to do that by taking a short course and practicing.

I also agree that Ampligen could be a bridge to something more effective. It works for me, but it takes a long time, and although the side effects aren't serious, they are like worsening CFS in the beginning, which usually wears off eventually. For me it takes about four months. And although I got a lot better last time I was on it and went back to my normal life, I never completely got rid of the post-exertional malaise, although it took a lot more to trigger it. The increase in energy and disappearing of other symptoms, such as pain and brain fog, made it totally worth it.

Does anyone know if, in the case of accelerated approval, the patients would still have to pay for the drug like they do now? Would it be under the auspices of a drug trial such as the current Amp-511? What would be the difference between accelerated approval and the current Amp-511?

Thanks.

Hope123 August 4, 2012 at 7:46 pm

It's not my area of expertise but my feeling is approval of Ampligen by FDA would not necessarily mean approval for reimbursement by US insurance companies. Private insurance companies are given leeway in whether they want to pay for something on not given their own review of the evidence. Most of the time, it takes more than one successful trial (and those trials often have to be larger, multi-center, etc.) for them to consider a drug effective and worth covering. Government insurance like Medicare are mandated by law if I remember right to cover at a least one drug for every medical condition but even then, they might require more evidence before coverage. But coverage by Medicare does influence private insurance coverage since private companies which have a contract with Medicare are required to provide AT LEAST what Medicare does in their plans (companies can choose to provide more).

I hope it does not happen but one of my concerns if if Ampligen is approved whether the idea would then spread among physicians, government, insurance companies, etc. that all people with CFS should be on Ampligen or have a trial of Ampligen. That would be dangerous for some people with CFS.

RE: Peterson. I don't know Peterson personally nor am I a patient of his and he doesn't talk much publicly so I only see/ hear what most people hear/ see. Outside of studies, many people who have side effects or aren't improving on a drug are lost to clinic follow-up (they stop going to the doc) so if they don't do well, their physicians will not know what happened unless the physician makes an extra effort to reach that person. So I believe what he sees/ hears is by nature limited through no fault of his. Also, drug companies are not bound to report all their information to clinicians. And finally, as I mentioned, I doubt he (or any ME/CFS specialist) recommends Ampligen to every CFS patient.

I also think if Ampligen were the true answer Peterson and some of the other docs that have been in this for a while and are dedicated to caring for patients would be much more outspoken and encouraging about the drug.

satoshikasumi August 4, 2012 at 8:38 pm

Hope123 says: "Overall, I think there will be better drugs than Ampligen, safety and efficacy-wise, in the near future and I would caution people to not push for a "political victory" without regard to safety."

I am frankly astonished by how skeptical some in the patient community are about Ampligen and how optimistic they are that something better will be invented in the "near" future.

I'd like to remind you that many of us have been ill 20 years or longer, and have heard the line that a treatment is just a few years away many times before. CFS is extremely difficult to treat and there are no known molecular targets. Drug companies are afraid to start. As of now, no drug company other than Hemispherx has a novel candidate drug even in phase I or II for CFS. When a target is identified, it will take five to ten years to go through the full clinical trials process. If Ampligen doesn't get approved and you are 20 years old and are disabled by CFS, that means you may be in your 30s before "something better" comes along.

The "near" future in scientific terms really means a long, long time in real human life terms.

And that something else may not be better. There is the idea of repurposing existing drugs like rituxan, but it is well known that rituxan can cause death. Existing antivirals carry the risk of organ damage, infertility, and death if they are not carefully monitored.

Ampligen should have been approved for CFS back in 1998, if not earlier. There was strong evidence then that it improved all the symptoms of the condition in the majority of treated patients with severe disability and as far as we know it is safer than any other known immunomodulator.

Firestormm August 5, 2012 at 2:57 am
Cort

I hope there are better drugs out in the near future but I would be surprised if there are significant safety issues with Ampligen as Dr. Peterson said in a recent interview with Llewelyn King, I think, it was…that it is safe and effective. He's been giving it to patients for over 10 years…

Some people seem to do well after Ampligen – they stay at their level of wellness – but others relapse. It can take a couple of years for them to relapse or it can happen more quickly…

I see Ampligen as a bridge to something better; once they can start studying it in more detail they'll know better why it works in some patients and not others; that will give researchers a leg up on the next drug…but its got to get in there first….

How can a doctor be prescribing a drug that is not FDA approved to patients for 10 years? Have I missed something only I thought Peterson was doing so in trials only, but regardless I would like to understand more about how such a thing could happen. Isn't the whole point of Ampligen trying so hard to gain this approval that they can then be offered to one and all legitimately? If Ampligen can be prescribed now, then what's the point? Sorry. I'm not familiar really with the way this works in the USA.

Firestormm August 5, 2012 at 3:30 am

Morning Sato,

satoshikasumi

I am frankly astonished by how skeptical some in the patient community are about Ampligen and how optimistic they are that something better will be invented in the "near" future.

I'd like to remind you that many of us have been ill 20 years or longer, and have heard the line that a treatment is just a few years away many times before.

Speaking personally and as someone having had this diagnosis now for 15 years, my scepticism is as a result of having this condition for 15 years.

I have grown used to false starts, false promises, false hope, and therefore I'll believe it when I see it/can try it myself once it has been approved.

Absolutely nothing wrong with scepticism but, don't get me wrong, I am at one level quite delighted to hear of any drug being manufactured specifically for my use – but Ampligen wasn't not originally.

If it works, it works, if it doesn't well I won't have lost any more sleep about another failed promise of help.

Generally, I am very very cautious about any drug that claims it can help such a diverse patient group even one that employs the 'best' criteria currently known.

That said and like I mentioned above, if it does find it's way into my doctor's list of approved thing to try, I shall most definitely take it for a work-out and myself too if the claims hold up.

Doogle August 5, 2012 at 11:43 am
MishMash

Hemispherix makes Immunovir. It was prescribed to me. It isn't covered by any insurance, you have to order it from Canada, and I didn't notice any benefit after taking it for a long time. I hope Ampligen works better.

No. Hemispherix does not/never made Immunovir to my knowledge. Immunovir was developed and produced by Newport Pharmaceuticals Limited and their subsidiaries. http://www.rivexpharma.com/products_imunovir.html

Snow Leopard August 5, 2012 at 12:02 pm

Here is hoping for ‘accelerated approval’ status!

Doogle August 5, 2012 at 12:36 pm
Firestormm

How can a doctor be prescribing a drug that is not FDA approved to patients for 10 years? Have I missed something only I thought Peterson was doing so in trials only, but regardless I would like to understand more about how such a thing could happen. Isn't the whole point of Ampligen trying so hard to gain this approval that they can then be offered to one and all legitimately? If Ampligen can be prescribed now, then what's the point? Sorry.

Ampligen has been used in the US for over 20 years outside of FDA approved double blind studies under Investigational New Drug (IND) status. "Treatment IND is submitted for experimental drugs showing promise in clinical testing for serious or immediately life-threatening conditions while the final clinical work is conducted and the FDA review takes place." http://www.fda.gov/drugs/developmen…estigationalnewdrugindapplication/default.htm

The length of time this has occurred is rather unique as far as I know, and is due to the political problems with the illness and Hemispherix. The FDA has not wanted to shut down access to the only drug that has ever shown promise for treating ME/CFS. Yet the FDA doesn't want to approve it for general marketing until they conclusively know the benefits of the drug clearly outweigh the risks.

A treatment IND only allows a limited number of people to be on the drug. I believe the treatment IND Hemispherix has from the FDA allows 100 persons. The sites giving the drug have to go though a lengthy approval process and theoretically the drug company can only recoup their cost of manufacturing the drug. Right now it costs approximately $15,000 per year for the maximum dose, 400mg. IV infusion costs are extra. Most Insurance will not pay for a drug given under a treatment IND because it's still classified as experimental.

So there are severe limitations for use of the drug under the present process hence the necessity to get full marketing approval.

jimells August 5, 2012 at 12:37 pm

Like satoshikasumi, I can't wait another sixteen years, or six years, or maybe even another six months for a real treatment. How many of us will be dead from this illness by the time the FDA gets off its ass and makes a decision?

My life has already been destroyed. If a treatment killed me quickly, that would be an improvement over dying a little each day. Right now my bank account and my ability to perform activities of daily living are in a race towards zero. My only income is food stamps, and the ground is rising up to meet me at a terrifying speed. Since I live in the US, there is no safety net and I have no parachute. But we have 'freedom': we're free to starve under a bridge, except the cops will run us off, since it is still a crime to be penniless in public!

My application for Social Security disability is celebrating its fifth anniversary. It's now old enough to enter school, except public education is also being wrecked. No one can tell me when I will get scheduled for an ALJ hearing. If I manage to live long enough to get a hearing, the chances of winning my claim are not good.

Meanwhile Governor 'Bully' LePage never misses an opportunity to insult and attack Mainers with no money. His latest plan is to 'reduce energy costs' by getting rid of the tiny fee on electric bills that funds a program to help poor people with their electric bills. Last year that program paid all of $120 to my account. An undeserved bonus for sure!!

satoshikasumi August 5, 2012 at 6:16 pm

I know I am being somewhat of a bulldog for Ampligen. But I am one of only about a hundred patients who has actually taken Ampligen during the past five years. We, the privileged few, know that this drug is unique. It gave me back my mind. My cognitive function.

The FDA knew Ampligen could dramatically reduce the severe debilitation associated with CFS back in 1998, when the phase II results were released. They chose not to fast track the drug for approval, in spite of the fact that these desperate patients had no other hope. At the time FDA made this decision, CDC and NIH officials publicly expressed skepticism about whether CFS was a real disease, and the view that it was a psychological or imaginary ailment was held by many doctors and government officials.

Up until just a couple years ago, the CDC website had a detailed statement advising doctors not to order tests that could document CFS patients' disability. They said not to order tests for herpesvirus infections, not to do immune panels, not to look at natural killer cell function, not to do tilt-table tests, not to do brain function tests, claiming that none of these things had any value in diagnosing or treating CFS.

But, the treatment section of the CDC site did and still does list psychotherapy: cognitive behavior therapy, and graded exercise therapy as the first treatments to be tried. Ask patients in the UK and EU countries how well that approach is working for them.

In the 14 years since, no other drug company has dared to even try to develop a drug for CFS, in spite of the fact that the profit potential is huge. With Ampligen, the FDA has repeatedly sent Hemispherx on fishing expeditions to try to find something wrong with the drug- a new rodent carcinogenicity study, a new evaluation of cytokines to look for evidence that the drug induces autoimmunity. Each test looking for the ghastly side effect comes up empty-handed. Ampligen has been used in humans with CFS for more than 20 years, and some individual patients have taken it for 10 years or more of their lives because nothing else works. No one has died from Ampligen.

justinreilly August 6, 2012 at 4:35 pm

Thanks for the info guys!

justinreilly August 6, 2012 at 4:36 pm

Thanks for the info guys!

SpecialK82 August 6, 2012 at 6:04 pm
jimells

My life has already been destroyed. If a treatment killed me quickly, that would be an improvement over dying a little each day. Right now my bank account and my ability to perform activities of daily living are in a race towards zero. My only income is food stamps, and the ground is rising up to meet me at a terrifying speed. Since I live in the US, there is no safety net and I have no parachute. But we have 'freedom': we're free to starve under a bridge, except the cops will run us off, since it is still a crime to be penniless in public!

My heart goes out to you jimells, your post has really touched me. I am glad that you are here sharing your frustration and pain with us, I hope that we can give you some comfort. Know that you are loved.

Bob August 12, 2012 at 6:14 pm

via @TomKindlon

The San Francisco Bay Area area practice of physician (Dr. Raj Patel) has been IRB approved and has started baseline testing patients for the FDA open label Ampligen treatment study protocol (AMP 511).

In a 1990-1992 placebo controlled study, approximately 80% of 45 CFS patients improved, and 50% improved significantly. A similar study in Belgium reported similar results.

https://listserv.nodak.edu/cgi-bin/wa.exe?A2=ind1208b&L=co-cure&F=&S=&P=11435

WillowJ August 12, 2012 at 11:27 pm

if approved by the FDA, any doctor would be free to prescribe Ampligen for any condition (off-label prescription is perfectly legal in USA).

Insurance coverage in the USA would work like this: most companies have a formulary listing covered drugs. There's probably a requirement that they have a comprehensive listing (and it would be to their benefit to do so, anyway), so it would be difficult for them not to include a drug for a condition which has no other approved treatments.
However, if they do not cover brand-name drugs on a specific plan, they might not have to cover it until there is a generic version (though it's possible they could make an exception; some insurances will do this but that's more likely if one has group insurance through someone's work).
Your doctor still needs to convince the insurer that the treatment is 'medically necessary' for you specifically (whether because you have a diagnosis it treats, or for some other reason).

In other countries such as Australia, the drug may have to be approved for a specific condition in order for insurance to cover.

Firestormm August 13, 2012 at 5:37 am
Bob

via @TomKindlon

AMP 511 Cost-Recovery treatment protocol requirements are expensive:

* The total cost of Ampligen to a participant is approximately $2,500/month.

* Ampligen is administered from 200mg or 400mg vials. The cost of the Ampligen alone, at the maximum therapeutic dose of 400mg twice per week, is $2,400 per 8 weeks, and is rarely covered by medical insurance. Some patients may require the lesser 200mg dose which is less expensive.

* IV Infusion costs, administrative costs, and testing are extra and estimated at $2,000 per 8 weeks and are generally not covered by insurance.

* Dr. Patel estimates it will take 18 months for most patients to achieve the full therapeutic and stabilization effects of Ampligen, and they should plan for that timeframe.

* Before being enrolled in the treatment protocol, patients will be
tested for biomarkers to determine the likely response to Ampligen.

However, Ampligen has been clearly beneficial for a subset of ME/CFS patients. Some of the
clinical factors that may identify these patients are:

* Acute Viral Onset

* 2-5A Synthetase/RNase L Up Regulation

* Depressed Natural Killer Cell Cytotoxicity

* Persistent Herpes Virus Infections (EBV, HHV-6, CMV, etc)

Ampligen often initially causes dose dependent flu-like symptoms that can be significant. This has contributed to patient reports of Ampligen's adverse effects.

These are not the only health risks from Ampligen.

Titrating the Ampligen dose may help minimize side effects.
Ampligen is administered intravenously twice per week, so people with fragile veins may have difficulty with infusions.
IV ports are sometimes installed.

Well at least it affords more insight into the clinical application of this drug and it's cost etc.

Firestormm August 15, 2012 at 3:39 pm

So the FDA have accepted the submission from Hemispherex. Share price was up 10% on the news it seems – or it was – remains 3 cents off it's 52 week high of 51 cents but if approved then…. who knows? Fancy a punt anyone?

http://www.virtual-strategy.com/2012/08/14/fda-accepts-complete-response-submission-regarding-ampligenr-new-drug-application-chronic

alex3619 August 15, 2012 at 7:48 pm
satoshikasumi

Hope123 says: "Overall, I think there will be better drugs than Ampligen, safety and efficacy-wise, in the near future and I would caution people to not push for a "political victory" without regard to safety."

And that something else may not be better. There is the idea of repurposing existing drugs like rituxan, but it is well known that rituxan can cause death. Existing antivirals carry the risk of organ damage, infertility, and death if they are not carefully monitored.

I agree we should have had Ampligen decades ago. It could have now become generic and hence cheaper. The delay in approval has robbed two generations of patients of a chance at some recovery, and will rob another two generations who do not have the cash to pay for it once it goes commercial.

However Rituximab is not particularly dangerous (especially if it can cure rather than treat), though not particularly safe either:

http://arthritis-research.com/content/pdf/ar3337.pdf

The death rate is about 3%. Much of that is from infections. Some of it is from a severe infusion reaction. However we have yet to see either in CFS or ME patients, unless I missed an announcement. We are a different patient set. On the other hand, so far not enough patients have been studied to reliably show even a 3% death rate. On the third hand, for any Moties fans out there, drugs have been withdrawn from market for death rates lower than this.

Bye, Alex

Tally August 16, 2012 at 3:37 am

They mention that patients with depressed natural killer cell cytotoxicity have better chances of responding to Ampligen. Is this lower number of natural killer cells, or is it a different thing?

Bob August 16, 2012 at 6:56 am
Tally

They mention that patients with depressed natural killer cell cytotoxicity have better chances of responding to Ampligen. Is this lower number of natural killer cells, or is it a different thing?

It's almost the same thing, Tally, but "depressed natural killer cell cytotoxicity" specifically means low function, rather than low numbers. But I suppose that low function can be caused by low numbers.

Bob September 28, 2012 at 6:24 am

Here's some info about the effectiveness of Ampligen.

A Double-Blind, Placebo-Controlled, Randomized, Clinical Trial of the TLR-3 Agonist Rintatolimod in Severe Cases of Chronic Fatigue Syndrome
David R. Strayer et al
March 14, 2012
PLoS ONE 7(3): e31334. doi:10.1371/journal.pone.0031334
http://www.plosone.org/article/info:doi/10.1371/journal.pone.0031334

And here is a helpful summary:

ME Research UK – Breakthrough Magazine:
Autumn 2012 issue, page 12, third column, under 'Maryland':
http://www.meresearch.org.uk/information/breakthrough/index.html

Rintatolimod trial
Clinical trials of non-psychological treatments
are quite rare in ME/CFS, so it can be
exciting when one comes along, particularly
if the most severely affected patients
are included. One recent example was
a phase III randomised trial in which the
TLR-3 agonist rintatolimod was compared
with placebo in 234 people suffering
with long-standing, debilitating ME/CFS
at 12 different centres across the USA.
After 40 weeks, exercise tolerance (total
achievable exercise time on a treadmill) was
significantly improved in the patients on
intravenous rintatolimod therapy (400 mg
twice weekly) compared with those on
placebo. The size of the improvement was
around 20%, equating to approximately
68 seconds of additional treadmill activity.
Indeed, the authors say that this level of
improvement represents approximately twice
the minimum considered medically significant
by regulatory agencies. Rintatolimod also
reduced dependence on symptom-relieving
drugs compared with placebo, and it seems
to have been well-tolerated with no serious
adverse effects definitely related to the drug.
Under its more common name, Ampligen,
rintatolimod has been well-known to ME/
CFS patients since the first preliminary report
of its potential usefulness in 1994. Ampligen
has known antiviral and immunomodulatory
properties, and these positive results
suggest that it may become part of the
therapeutic armoury of treatments for ME/
CFS, providing FDA approval is finally granted.

Nielk September 28, 2012 at 8:22 am

Tell the FDA what your experience with Ampligen has been.

http://www.cfscentral.com/2012/09/what-have-your-experiences-been-on.html?spref=fb

TUESDAY, SEPTEMBER 25, 2012

The Food and Drug Administration (FDA) will once again decide this winter whether the experimental drug Ampligen, which has helped many ME patients recover or improve, should be approved. Should the drug be approved, it will be an ME game-changer.

Oddly, little has been discussed on the boards about Ampligen this time around—perhaps because the drug’s been up for approval several times before—and that’s too bad, because an approved drug would help legitimize the disease, provide much-needed treatment for patients, and signal to other drug companies that the disease is worthy of effective medications—and not psychobabble.

Eighteen years ago, I wrote a piece on Ampligen for Philadelphia magazine called The AIDS Drug No One Can Have. Back then the drug proved remarkably helpful for both ME and HIV/AIDS. Many patients went from bedridden to returning to work and school and a few with ME whom I interviewed who’d been ill for only a few years completely recovered.

Then the FDA insisted on a formula change to make the intravenous drug easier to administer; it’s not clear if the new formulation is as effective, but many patients have continued to report improvement.

The FDA has tentatively scheduled the review process to begin December 20 and to continue to February 2.

In the meantime, the FDA wants to hear from patients by November 1 about their experiences with ME, including how drugs like Ampligen have helped in treating the disease. The FDA is also interested in learning about the emblematic endpoints that should be used in reviewing ME drugs for approval. Fatigue, for instance, is a a subjective and often inaccurate marker with ME. Improvement in post-exertional malaise would be a far more significant marker.

Patients can submit comments about Ampligen to the FDA directlyhere or send in comments and I'll post them on CFS Central and forward them to the FDA. Pressure needs to be placed on the government so that these agencies will be forced—kicking and screaming—to do the right thing by patients.

grosolo September 28, 2012 at 3:28 pm

I don't think Ampligen is the answer. It's not if you believe that ME/CFS has an autoimmune component or that it causes neuroinflammation (and ME definitely does cause neuroinflammation). If you look up the drug under its brand name, none of this info will come to light, but if you go to pubmed and look it up under its chemical name (Poly I:C, or poly-inosinic polycytidilic acid), you'll find lots of hugely discouraging studies, some of which even call Ampligen "neurotoxic."
I'm not saying this to be discouraging. I want Ampligen to work as much as anyone — I suffer. But I think there's a reason Ampligen wasn't rushed into duty two decades ago and I think the ME/CFS community might be better served going in other pharmaceutical directions.

http://www.ncbi.nlm.nih.gov/pubmed/18590811

http://www.ncbi.nlm.nih.gov/pubmed/21123556

http://www.ncbi.nlm.nih.gov/pubmed/21296697
http://www.ncbi.nlm.nih.gov/pubmed/21815968

Doogle September 29, 2012 at 11:51 pm

Reply to grosolo,

Sorry grosolo, but your information is missing a vital component. Ampligen is not simply Poly I:C as described in the links you presented. Ampligen is a specifically modified form of Poly I:C that retains TLR3 activity but is designed to have a much shorter half-life in the body.
http://patft.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=/netahtml/PTO/srchnum.htm&r=1&f=G&l=50&s1=4,024,222.PN.&OS=PN/4,024,222&RS=PN/4,024,222

This results in much less toxicity than Poly I:C. The toxicity of Poly I:C was known years before Ampligen because it was researched as an immune stimulant to fight cancer. It proved too toxic for even that use, and Ampligen was invented as a less toxic immune stimulant. Ampligen proved to be very efficacious in stimulating the TLR3 interferon system to fight viruses and also has some anticancer activity.

Snow Leopard September 30, 2012 at 2:45 am

Skepticism about whether doctors or insurance companies will prescribe the drug is a bit premature at this point, the first step is getting the drug approved in the first place.

Getting a drug approved for CFS or ME sets a major precedent and will benefit us even if we don't ever take it personally.

Hip December 10, 2012 at 10:41 pm
grosolo

I don't think Ampligen is the answer. It's not if you believe that ME/CFS has an autoimmune component or that it causes neuroinflammation

The apparent autoimmunity and neuroinflammation in ME/CFS are likely caused by one or more infections. Enteroviruses, for example, which are strongly linked to ME/CFS, are inducers of autoimmunity. 1 In general, infections precipitate the production of lots of autoantibodies.

So by wiping out viral infections with Ampligen, or with some other antiviral developed in the future, you will likely halt the autoimmunity.

Cort December 12, 2012 at 10:36 am

I don't think Ampligen is 'the answer'; I think it's like part of an answer; pushing forward to a bigger answer…Its definitely a big help….a big big help for some people…a smaller help for others and it doesn't work for others but that's what you'd expect from anything that works in this disorder – until subsets are teased out.

grosolo December 18, 2012 at 1:15 pm
Hip

The apparent autoimmunity and neuroinflammation in ME/CFS are likely caused by one or more infections. Enteroviruses, for example, which are strongly linked to ME/CFS, are inducers of autoimmunity. 1 In general, infections precipitate the production of lots of autoantibodies.

So by wiping out viral infections with Ampligen, or with some other antiviral developed in the future, you will likely halt the autoimmunity.

If the medicine in question causes brain toxicity — even while stimulating the immune system — it's still a very flawed treatment. Look up Poly inosinic poly cytidilic acid on pubmed. Scientists now administer Ampligen to mice in order to test other medicines under the conditions of neurotoxicity. IE — they're using Ampligen to ruin the brains of mice in order to see how other drugs can fix the brain. That says a lot…And these are the non-Hemispherx researchers — the ones who have no stake in this…

I don't mean to be a kill-joy. I want everyone to be able to benefit from whatever they can. But there has been so much research that has been glossed over. The whole brand name Ampligen, not to mention the recently made-up fake generic name rintatolimod — which no one uses — are modes of obfuscation. Because if you look up the drug under that name, you get very little information. But if you look up studies under its chemical name of poly inosinic poly cytidilic acid, you get so much more…and a lot of it is ugly and scary.

grosolo December 18, 2012 at 1:19 pm
Doogle

Reply to grosolo,

Sorry grosolo, but your information is missing a vital component. Ampligen is not simply Poly I:C as described in the links you presented. Ampligen is a specifically modified form of Poly I:C that retains TLR3 activity but is designed to have a much shorter half-life in the body.
http://patft.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=/netahtml/PTO/srchnum.htm&r=1&f=G&l=50&s1=4,024,222.PN.&OS=PN/4,024,222&RS=PN/4,024,222

This results in much less toxicity than Poly I:C. The toxicity of Poly I:C was known years before Ampligen because it was researched as an immune stimulant to fight cancer. It proved too toxic for even that use, and Ampligen was invented as a less toxic immune stimulant. Ampligen proved to be very efficacious in stimulating the TLR3 interferon system to fight viruses and also has some anticancer activity.

This patent form just says that polyriboinosinate and polycytidilate were modified to YIELD Poly IC.
So they didn't modify Poly IC to create a less toxic form. They modified other drugs to create Poly IC. So anytime you see a study coming out now that uses Poly IC, it's using Ampligen, plain and simple…

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