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Adrienne Dellwo

Better Chance for Ampligen the Second Time Around?

By December 21, 2012

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The potential chronic fatigue syndrome (ME/CFS) drug Ampligen (rintatolimod) is about to go up for FDA approval again, but are its chances any better this time? It has elements both for and against it.

Three years ago, the FDA gave Ampligen's manufacturer, Hemispherx Biopharma Inc., a "not now" response in what's called a Complete Response Letter, requesting more data to prove the drug's effectiveness. Hemispherx submitted more data, and the new decision is due in just over a month.

However, it's not the data that makes some Ampligen watchers think the drug will make it through this time - it's a changing political climate surrounding ME/CFS.

Some financial blogs, which have been skeptical about Ampligen until recently, now say they think approval is highly likely. Here's a quote from investment blog Seeking Alpha:

"Patient advocacy groups, U.S. Senators, and the U.S President have all pushed for an FDA approved therapeutic option for ME. Let's be clear on this: there is no alternative to Ampligen at this time nor will there be any time soon. Hence, this could be the last chance to get an ME therapy approved for several years. It's also important to note that upon accepting Hemispherx's New Drug Application last July, the FDA agreed that the review will focus on the drug's efficacy, not safety. So the FDA does feel comfortable with the drug's safety profile, and this will have a major impact on the final outcome." -Seeking Alpha, Dec. 12, 2012

The FDA, at an ME/CFS stakeholder meeting in September, talked about the possibility of getting a treatment approved quickly:

"FDA's 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. For example, you may have heard the term, 'the accelerated approval process.' This is in FDA's regulations...this process is set up to increase the availability of experimental drugs and biological products based on findings that are somewhat preliminary, but thought to predict clinical benefit." -Transcript, FDA ME/CFS Stakeholder Meeting, Sept. 13, 2012

To qualify for accelerated approval, the FDA requires that the drug:

  • Treats a serious or life-threatening disease (which the FDA now admits ME/CFS is);
  • Provides meaningful and measurable therapeutic benefit to patients over existing treatment.

That last bit is really important, because currently ME/CFS has no existing FDA-approved treatment. Any proven benefit to any number of people could be considered meaningful and measurable.

However, the talk of a rosier outlook came before the Dec. 20 meeting of the FDA's Advisory Committee. That committee gives recommendations to the agency, and the agency usually follows them - but it has no obligation to do so. The committee voted 8-5 against Ampligen. It's important to remember, though, that the committee also recommended against Lyrica (pregabalin) for fibromyalgia, and it was approved. Why? Because, at the time, no other drugs were approved for fibromyalgia. So will the committee's vote mean anything?

We won't have to wait long to find out. The FDA's decision is due Feb. 2, 2013.

Do you think some of these factors will help Ampligen become approved? Is it dead in the water? If Ampligen were available and affordable, would you try it? Leave your comments below!

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Comments
December 21, 2012 at 9:25 am
(1) George says:

How many times do we have to see these outcomes to finally understand that HEB is a fraud and Ampligen does not work? Trust me, in the early 1990′s I had some hope, but 20 years of lies by the company and proposals to use Ampligen to treat almost every disease known to man makes one change his mind.

December 21, 2012 at 10:07 am
(2) Steven says:

@George: But what if it is not a fraud? Though I don’t think it’s a cure for everything, but if it helps for those suffering from CFS?

December 21, 2012 at 11:03 am
(3) George says:

The FDA told Hemispherix in 2009 that another trial was needed. What did the company do, nothing. Instead, HEB reanalyzed very old data using a different methodology and different end points. That is totally unacceptable. Secondly, I always smile when I read that the efficacy of the drug was based on treadmill tests. Anyone with CFS knows that this illness varies from day to day and claiming the drug works based on slight improvement in treadmill test results is just ridiculous.

That is why the CFS community should be very angry with the company management, for providing false hope to very sick people, all the while paying themselves handsomely.

December 21, 2012 at 1:02 pm
(4) Rudy says:

Its not that Ampligen does not work, its that the Hemispherx grossly has mismanaged the drug, and refuses to do additional trials. The testimony of CFS patients at the FDA panel yesterday was powerful and moving. While the trails have been poorly done and analyzed, patients have been taking the drug for years and have seen amazing results.

December 21, 2012 at 2:09 pm
(5) Rachael says:

I agree with George. As anyone who knows who has this illness, ME/CFS is so much more than increasing exercise stamina. “Ampligen-treated patients with chronic fatigue syndrome entered the study with a mean, baseline treadmill exercise duration of 576 seconds. After 40 weeks of treatment, mean exercise duration rose to 672 seconds, or a 16.7% improvement. The placebo patients began the study with a mean, baseline treadmill exercise duration of 588 seconds and ended at 616 seconds, or an improvement of 4.8% That works out to a placebo-adjusted improvement in exercise duration for Ampligen of 11.9%..”1

So, while this is all very interesting what will it do for other symptoms of ME/CFS (feelings of malaise, sore throat, nausea, low circulating blood pressure/volume, severe headaches, muscle pain, inflammation, irritable bowel, problems regulating body temperature, anxiety, insomnia etc).

Souce:
1 Hemispherx’s Ampligen Rehash Unlikely to Impress FDA – Sept 29, 2012

http://www.thestreet.com/story/11720256/1/hemispherxs-ampligen-rehash-unlikely-to-impress-fda.html

December 21, 2012 at 4:29 pm
(6) Terry says:

The people that testified for ampligen are real people with real results, we have nothing else, and I would take it in a second, without hesitation. I have already lost ten years of my life. The consequences of doing nothing, when they could be helping some, are to great. Approval would cause a ripple effect to other RX companys to start, and they would. Ampligen is not the wonder drug, or cure, but for the people it helps, it their life.

December 21, 2012 at 4:48 pm
(7) George says:

I have had CFS since 1987. Some really bad years and others half way decent. I understand people are desperate for a treatment and Dr. Carter knows that and has played it to the hilt to become a very rich man. Ampligen MAY work for some…MAY….but I think it would be dangerous to advocate approval of this drug with the limited, and poorly, conducted trials that have been conducted.

December 21, 2012 at 6:54 pm
(8) Mary says:

the problem seems to be in the poorly designed studies and the sheer expense of the drug, rather than a problem with the efficacy of Ampligen.
So yes, I would try it. If it doesn’t get approved, hopefully someone will get the funding to be able to do some better-designed studies.

December 22, 2012 at 12:09 am
(9) Maryhelen says:

I would try this drug. I heard they have had success with it in Europe for FM

December 22, 2012 at 4:37 am
(10) Kathryn says:

I will take Ampligen if it is approved without hesitation. In fact, I want to be first in line!!! I have been suffering from ME/CFS since 1972 and I am housebound as I watch the world continue living their lives. This is NOT what I would call “living”. I long to get back some part of what I have lost because of this horrible illness—HOPE for that is the only thing that gets me through another day. If I can’t have hope for a better quality of life, I think I’ve suffered too many years already. I do not wish to stay alive in this condition until I die of old age, I’ve had enough suffering!!! By the way, I am already 58 years old and I haved been robbed of any way to live but in misery for over 30 years! I HOPE I can get Ampligen. I HOPE for a way for a better life than what I have….that’s all I have to hold on to, HOPE for a better quality of life.
Also, some of the patients have definitely benefited from taking Ampligen. It isn’t “may have benefited”, They DID benefit! That is a fact! Do not make statements that you assume are true. George, get your facts straight! I want a CHANCE for a better life!

December 22, 2012 at 2:40 pm
(11) Edana says:

I have had ME/CFS and Fibromyalgia for most of my life and I am now 66 years old and literally housebound unless my husband takes me out in his truck which will carry my Power Chair. I can walk, for about 100 steps. I have no idea what it would be like to not have a sore throat. Between the two illnesses I am in constant pain, even taking very strong pain medication which does help, but does not get rid of enough of the pain for me to ever be even somewhat comfortable, If I leave the house for something such as a doctor’s appointment, it takes at least two days in bed to recuperate. Would I try the Ampligen? In a heart beat. What do I have to loose? That it will not work or have side effects that are intolerable and I am back to where I am now? Bring it on!

December 22, 2012 at 7:53 pm
(12) Sandy says:

George, I wouldn’t say that it would be dangerous. The committee found it to be safe. There are a lot of patients who claim they have their life back because of Ampligen, though they admit it doesn’t seem to help everyone, as many other approved drugs. Due to the fact that it was found safe, if it helps just a portion of people , it’s better than nobody getting help.

December 23, 2012 at 12:08 am
(13) Teresa says:

Will someome tell me what Ampilgen is? Is it a pain medication? In the last three years, I have been on and off the new designer drugs for Fbiro/ ME/ CFS..I am back on two pain relivers again, which helps the Asteo Arthritis, on neck and all over back. I am 62, been in pain nearly all my life, but just kept working and pushing through the pain. Ignoring the pain, brings on more pain.. House bound!, yes, more and more every month. I have to say I am glad I am not the only one here that is House Bound too. Some days, I get so guilty about been ill, I try to talk myself out of pain, and get some housework done..then, back in bed for two days to two months. I am glad it is winter time, so I cannot work in my flower beds..lots of reprecusions there.. thanks for info, I will keep watching for more information. Terri.

December 23, 2012 at 1:22 am
(14) linda popaj says:

this isn’t living.I will try just about anything.I have been suffering like the rest of you for years.I just hope medicare will pay for it.

December 24, 2012 at 5:22 am
(15) Jac says:

Dr. Carter founded interferon and was robbed the spoils of this drug by the big pharmaceuticals…

He didn’t have enough clout to protect himself from these thieves. Upon developing Ampligen, he surrounded it with over 600 patents. It’s understandable he’s somewhat paranoid under these conditions.

@George states that Dr. Carter enriched himself with this story… I invite him to attempt creating a new medical protocol and go through what Carter and his team have been through for the last 20 years!

Asensio is a known money launderer, and The Street has it’s followers of profit mongers. They are the ones who profited from this.

The problem at the FDA is the innovative nature of Ampligen. For 20 years no one knew where to classify it. It was shuttled between departments as a hot potatoe. ME/CFS wasn’t even recognized as a sickness!

Dr. Carter is NOT crazy and he’s NOT dishonest. He’s spent 20 years of his life to present to the world another approach to medicine. As usual, the establishment has a difficult time to accept change.

I hope approval is accepted. My cousin committed suicide (mother of 2) at the age of 32 because of this disease.

December 24, 2012 at 7:21 am
(16) David says:

I don’t have CFS, but I feel for everyone here. I will make a mention that those patients that represented Ampligen at the advisory panel did see results. The ones that were very interesting were the people who went through an ABAB(minus final B in some cases) test through their own due to issues with receiving it and then not being able to get it, and then receiving it again. This is pretty powerful in my opinion as it shows cause and effect. Someone who takes it and receives benefit, stops and goes backward, takes it and receives benefit again is a very showing tell. Now it may not work for everyone, but if it does work for just a few I think its worth it. Also lets not forget if the FDA actually approves something for the disease it gives a baseline for future drugs as they can compare their drug to Ampligen for approval.

December 24, 2012 at 9:15 am
(17) Martin says:

The reality is the FDA is killing the American pharmaceutical market and spending more time covering their butt than helping a company along. If there were so many problems how come they held an advisory meeting? Why didn’t the company get a chance to answer all those questions before coming to the table. Years and years of safety records? What about the weird safety questions — once again CFS patients get shoved aside. Heartless couple with their own version of the science and “all the power”
.

December 25, 2012 at 9:59 am
(18) Thommy says:

I hope president Obama will help all sufferers of CFS and Ampligen will get approval in the next year.Merry Christmas from Switzerland

December 26, 2012 at 12:22 am
(19) John says:

Yes I was on ampligen for a year and was seventy percent better. I could only afford it for a year. January twenty-first 2012 was last infusion. Three months later I was sick again. I would be well enough to work on Ampligen without it I’m disabled. I’m still waiting for disability hearing almost two years. If it ‘s approved I still couldn’t afford it. hemispherix will make it super expensive even with insurance. They have their stockholders and profit margins to maintain. Oh, it was $50,000 to move from Houston to Charlotte and a year away from my family. Maybe Hemispherix will take that into consideration …… Nah this is America the country I honorably served during the gulf war we help other nations not our own….

December 26, 2012 at 12:52 am
(20) Alexis says:

Hemispherex is doing everything they can to make have Ampligen approved by the FDA. I know personally every single person working for hemispherex, and they are dedicated to making the patients lives better. Im the grandaughter of the president of Hem and just wanted to let you we are doing everything in our power possible to have this drug approved.

December 26, 2012 at 9:52 am
(21) John says:

There is no doubt Hem is doing everything to get approval. Unfortunately if approved it legitimizes ME as a disease and you and I know government doesn’t want that. Then they have to pay out more disability and big pharmacy loses billions in treating our symptoms. There should be consideration made on how much Hem decides to charge us for Ampligen. Even if its half of the $25,000 a year it costs for the drug trial it’s too much! Will see how hospitable you’re grandfather is if Ampligen the only thing that allows me to function normally is approved! I’m not very optimistic considering how much the drug trial costs. If Hem sold Ampligen to Phizer 20 years ago we’d all have Ampligen, know what subsets respond better and adjustments on how much and how often you need to take Ampligen. It’s not you’re fault you’re grandfather put Hem before a million sick people. He knows how the game is played and we have all suffered because of it. He would have made a fortune selling to Phizer but we all know that ME victims are in Hem’s best interest… Please!!!!!

December 26, 2012 at 10:29 am
(22) John says:

Alexis,
I apologize to you this wasn’t an attack on you or you’re grandfather. It’s sad that I know it works, in six months I saw a 23 year old young man with a degree from a prestigious university and bound to a wheelchair. I was heartbroken for him he could barely speak. Six months later he was out of the chair, speaking and playing his guitar again. His plans were to go to law school. I know what Ampligen did for Him and me. You can’t imagine what it’s like to have you’re life given back to you then stolen three months later. Please try and forgive me Alexis !

December 26, 2012 at 10:51 am
(23) Rachael says:

“If Ampligen were available and affordable, would you try it?”

****************
I hope Ampligen will help ME/CFS sufferers who believe that their case of ME/CFS had a viral cause, in which their body’s own immune system is now incapable of eliminating without the help of antimicrobials, such as Ampligen.

I, however, don’t believe Ampligen will do anything for my autoimmune/neuroimmune illness (ME/CFS), which occurred because of a genetic predisposition towards autoimmunity and an environmental trigger (in my case aspartame poisoning and extreme aerobic exercise). I would not consider trying Ampligen because I don’t believe my case of ME/CFS had a viral cause; I believe Ampligen would do me more harm, that good.

December 27, 2012 at 8:43 am
(24) Alexis says:

John,

I know it wasnt an attack to me or my grandfather, it just frustrates me when I see people saying negative things about him and the Hem crew. He has spent his whole life working getting to this point, and the past 20 years on Ampligen. Its heartbreaking when people say things like its a fraud. Its certaintly not about the money like people like to say. He IS dedicated to making peoples lives better, and has Hem standing right with him with that.

December 27, 2012 at 10:04 am
(25) John says:

Rachel, what other option do you have? You lack the courage to at least try? Maybe it ‘s worth the risk unless you have a better option.

December 27, 2012 at 10:25 am
(26) John says:

Alexis,
I think we’re all frustrated and everyone is not a responder. I saw it first hand, a lady had been there for 6 months and she had to stop it wasn’t helping her. The first day I was infused I felt so much better and the next day my chronic muscle pains in my legs were gone. I was so astonished I called my wonderful nurse to tell her. She said I could go home now I was cured lol. The first six months you have to fight through the battle Ampligen is giving the human herpes virus 6 A and God knows what else. I could feel the battle moving from my gut to my brain and other parts of my body. It was no picnic but I was going to finish a year of treatment no matter what. The last six months I got stronger and stronger ending up 70% better. The people on the FDA committee can’t understand any of this because their not sick. There should be a slot on the panel for someone who got better and knows it works. The lab work proved it. My hhv6a was gone and I hated having to stop taking it. I pray that it’s approved for the subset of people it helps and find something to help the others. If its not approved the government is simply doing it to keep big pharmacy happy and deligitamize the disease. They don’t want to pay us disability. That’s why I’ve been waiting two years for a hearing. There hoping will go away one way or another, it’s criminal. I wonder if this wasn’t like the Tuskegee experiment except they are trying to control population with bio weapons such as viruses.

December 27, 2012 at 11:06 am
(27) Rachael says:

John, I just don’t think Ampligen would work for me. In fact I believe it would make me profoundly sicker. It has nothing to do with courage. Now, if they were talking about approving Rituximab for the treatment of ME/CFS I would be much more interested. The reason being is that in my case, I think that ME/CFS is an autoimmune illness; an immune system in overdrive; in need of calming. You can read some of my comments at the link below to see where I coming from:

Is Chronic Fatigue Syndrome (ME/CFS) an Autoimmune Disorder?
http://phoenixrising.me/archives/6190

I still hope for people like yourself Ampligen will be approved. I just don’t think we are all suffering from the same illness, even though our symptoms may be very similar.

All the best
Rachael

December 27, 2012 at 11:21 am
(28) John says:

Rachel,
I’m with you and would try the cancer drug also if I had $70,000 but not many of us do. God bless and good luck!

December 27, 2012 at 1:46 pm
(29) John says:

Rachel,
A little FYI you have too many run on sentences. I can help if you want to learn English grammar.

December 27, 2012 at 2:36 pm
(30) Rachael says:

John, Yes, the illness ME/CFS often leads to problems putting our thoughts together either verbally, or on paper. I sometimes take a very long time to write a comment because of the cognitive problems that come with the illness. I don’t think lessons from you is going to help me with this issue. Seems, you have some cognitive problems yourself, since this is the third time you have spelled my name wrong.

December 29, 2012 at 1:14 pm
(31) George says:

Dr.Carter once proposed using Ampligen in cigarettes. Doesn’t that tell you all you need to know?

December 30, 2012 at 10:02 am
(32) Thommy says:

George,
please,this is not the right place here to malign the management of Hemispherx.So please stop your charges here.

December 30, 2012 at 10:04 am
(33) Thommy says:

There is some great hope here.Just take a look at this :
http://www.ipetitions.com/petition/ampligen/signatures

December 31, 2012 at 9:41 am
(34) KAL says:

This is rather late in the game, but Terri – Ampligen effects the immune system. If issues with the immune system were to be causing your pain then yes Ampligen might help, but not directly as it is not a pain medication.

The reason the authors of the studies used exercise is because ME and possibly patients with CFS have what is known as post exertional malaise lasting 24-hours or longer unrelieved by rest aka PEM. If you don’t have that you may not have ME or possibly CFS. A drug that helps patients who are exercise intolerant be able to exercise is effective – assuming all the patients in the study had PEM. If they had lupus or depression, but not ME or CFS then the drug probably won’t help them and it will skew the statistics. That is why if a drug only works for a well-defined subgroup then it is pointless to study it in people not in the subgroup unless they are controls or in a separate arm for comparison.

Exercise is also an objective endpoint. One that can be measured objectively. This is far better than a questionairre asking if patients “feel better” whatever that vague term means.

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