Deja Vu?
“I just try and not think about it, because it gets me furious” multiple sclerosis patient
The story sounds eerily similar. Citing questions regarding study quality and side effects, the FDA refuses to approve a drug for a condition for which few or no effective drugs exist. After hearing story after story of patients who have been dramatically helped by the drug, the patient community goes ballistic. They send letters and emails and gather 10,000 signatures on a petition asking the FDA to change their decision.
Astounded at the FDA’s rejection of a drug for MS, the patient community successfully fought back.
They argue they should be allowed to determine whether they want to accept the risk that comes with taking the drug.
It sounds very much like what played out in Ampligen and chronic fatigue syndrome (ME/CFS), but these event concerned a multiple sclerosis drug called Lemtrada.
Studies indicated the drug resulted in 50% fewer new attacks and reduced numbers of new lesions. The drug had already been approved in forty countries including Canada, Europe and the U.K. but the FDA said no go to it in the U.S. March of last year.
The FDA didn’t like the study design, though, and was concerned about the potentially dangerous, even fatal side-effects of the drug.
The chief advocate for the National MS Society said he didn’t have a clue what the FDA was thinking: “Trying to get a grip on what the FDA is thinking around this is quite puzzling and frustrating,” said Timothy Coetzee, chief of advocacy, services and research for the National MS Society.
The doctors on the ground couldn’t understand either as they were clearly seeing benefits. Seventy doctors using the drug in clinical studies signed a letter asking FDA to approve the drug. They called it “an important tool” in their fight against the disease.
Doctors using the drug were shocked at the FDA’s action
Some patients had had remarkable outcomes. One doctor who was headed for a wheelchair is able to walk and work full-time since he got an infusion in 2003. Another who would choke on his food and often needed a cane to walk is now able to ride his bike.
After Genzyme provided a re-analysis of its data, the FDA overturned its disapproval and approved the drug last December.
Ampligen
It’s hard not to think of Ampligen at a time like this. The doctors who have used it strongly promote it. Not everybody gets helped, but stories of major improvement and even recovery abound. It’s maker, Hemispherx Biopharma, provided the FDA with a re-analysis of its data as well. Even more so than MS patients, people with ME/CFS don’t have many good drug options.
But after highlighting concerns about safety in a drug the doctors using it found to be very safe, the FDA failed to approve Ampligen. This was despite the fact no other drugs have been in the FDA pipeline for ME/CFS for twenty years, and none are in sight now. The FDA’s call for expensive new studies essentially removed Ampligen as an option for people with ME/CFS. A drug that was clearly safe and clearly helped many people hit the dust.
After action from patients and doctors and a re-analysis of the data, the FDA approved the drug.
Since then the FDA has removed its major objection to approval – safety – leaving efficacy as the last remaining stumbling block. Even the FDA panelists who voted against approval, however, stated Ampligen was clearly having an effect. They simply wanted more study.
The reasons for the FDA’s rejection of Ampligen are fading fast. There are ways to conditionally approve a drug which allow more information to be gathered about its effects without small drug companies like Hemispherx Biopharma having to pony up vast amounts of cash. Why that’s not being done is unclear.
With the multiple sclerosis community successful in their attempt to get the FDA approval for a drug with potentially very significant side effects, it’s getting harder and harder to see why Ampligen, a drug with no significant safety issues, is not receiving at least conditional approval from the FDA for chronic fatigue syndrome.
- Next up – Advocates Request Congressional Hearing to Address Ampligen Rejection
The FDA approved Ampligen as a safe medication and it’s just sitting on the shelf. While millions suffer. It must be effective because it’s been in trials for two decades, or they would ‘ve stopped it long ago. Plus all of the patient reports of how much it helps them. I want a chance at it. There isn’t anything else out there like it. It’s not perfect, but it’s by far and away our best chance now. I think we need to do all it takes to get Ampligen approved!
Agreed – there’s so little downside of approving Ampligen even on a strictly regulated conditional basis – why not do it?
Can you help me out with one thing?
Not having FDA approval means what in this case?
Doctors can still prescribe it right? Is it more about insurance bureocracy?
Thanks
Btw. Can’t we do a petition?
I agree with Folk – time for an online petition [worldwide].
I would love to be part of what ever it takes to help push this drug through the bureaucracy. Signing, writing a letter or whatever you would suggest I will be a part of. Thank you for keeping us posted. Deb
I for one really tire of the “pro-ampligen case”.
The scientific case for its use is not great at all.
Cort, you still seem wedded to the notion that CFS is fundamentally a major disorder of the immune system?
Everyone is entitled to their opinion. I think the immune system is involved to some degree, but that the illness is not fundamentally related to an immune system issue.
I am all ears as to what you theory is.
It would be great if the FDA approved Ampligen for ME/CFS. I would like to try it, even if it mean financing it myself. I feel like I’ve tried all the «big» ME/CFS treatments, including gamma globulin, Imunovir, Valtrex, long term antibiotics, rituximab, and others. Ampligen and stem cell transplant is some of the last remaining options – I’m up for both!
PM – isn’t that a sign that it’s not the immune system?
Come on guys, it’s time to move on!
Why not “PEM” and immune system issues?
So what’s your theory? Cellular-a type of mitochondrial disease? Or autonomic, vascular? It is of course already coded as neurological but I think the immune system comes into play, with a possible viral assault. Viral reactivation and immune flare ups are well reported, so how do you explain these and some patients experiencing swollen lymph glands and viral symptoms throughout the illness? By the way, I’m not hung up on any one theory and ME seems to have systemic effects. However, illnesses are classified under one category.
With the microglia we’re blending nervous system and immune system together 🙂
Well based on my PERSONAL situation:
– Immune dysfunction (pretty solid testing to confirm)
– Long term activation of viruses (as a result?) such EBV, HHV-6, Enteroviruses, etc.
– Resulting inflammatory responses affecting the brain (abnormal fMRI, proven dysautonomia)
So I would say a dysfunctional immune system results in chronic inflammation affecting the brain.
If it’s neurological and the immune system is not involved then what is the mechanism that causes the “inflammation”?
Despite years and years of research and studies no one consistent immunological finding has been confirmed, other than perhaps lower natural killer cell activity, which is quite common to many disorders.
So,in my opinion, the evidence for CFS being an immune disorder is very weak indeed. And the case for ampligen is poor, and a big distraction.
Doesn’t mean to say the immune system is not involved in some way. I think it is.
No consistent finding has been shown in any CFS research. There are lot of distractions but I don’t find this to be one of them.
🙂
ME/CFS is a disorder of subsets – some people respond very well to Ampligen, some respond well to antivirals, others to Rituximab – and others who don’t respond to any of those….. Even if the immune system isn’t it for everyone there’s clearly an immune subset in there.
I don’t care what the mechanism is–Ampligen has moved me from being bedbound 24/7 and in intractable pain to having to rest only about 4 hours a day, with greatly lessened pain. I can have a social life, go to guild meetings, visit my family, travel, do retreats, do my own shopping etc. etc. There is no good reason why Ampligen hasn’t been approved long before now. It doesn’t work for everybody, but it works for a lot of people, and once CFS gets one approved drug that starts getting advertised in magazines, the illness will get the respectability and believability that Fibromyalgia now enjoys because it has FDA-approved drugs.
Thanks for passing that on Laurel and congratulations on your progress 🙂
Laurel, did you get Ampligen in Canada? If not, where did you receive it?
How has it helped you?
I’d love to hear how you are now Laurel and how you obtained Ampligen. I’ve tried in the past to get this into the UK. That was back in the 80s-90s but no success. Thanks
I’m curious…. the MS drug that was disapproved… the manner in which the sentence was written implied a ONE TIME injection. I made my best attempt to get to Charlotte and Dr. Lapp for the Ampligen study but as long as it is not covered by insurance and one must be living in the location of the physician doing the Ampligen IV injections, a million of us will not benefit. We would need Ampligen to be approved with protocol for ALL MD’s to utilize and not simply the handful living in the same town as the small handful of physicians. Of course, this is the problem with many research studies; Dr. Brodericks’ upcoming study in Birmingham requires that one live within 25 miles as do the rest being done. A pity as the numbers of people being tested for efficacy of treatment could be vastly increased if they could enlist some medical universities or other physicians around the USA to participate. And, as per usual, I’m waiting for CFS/ME to be even taught in our medical universities. Grrrrrr. marcie
Cort, if someone (or you?) started a petition on Change.org for the FDA to approve Ampligen, wouldn’t it help? A lot of people look to you and your website for info, so it would get a lot of publicity. Also, if a petition for the NIH to spend more on research for CFS/ME, wouldn’t that be likely to help? Have you thought of starting petitions for those two ideas or would you be willing to post links to petitions or write articles and ask people to sign the petitions if someone else did the ground work and started the petition? Thank you in advance for your input.
I am totally for the approval of Ampligen. We’ve got to put the pressure on them NOW. This is a fact-I became ill when I was 34, and I’m 67 now. What happened to my life? What happened is that there was no treatment I could get, given where I live. Something’s wrong with that picture. I don’t want to have so many others’ lives done in because no treatment has been approved. It’s been a tragedy. I will send this in my letter to my representatives.
It should be our choice whether or not we want to try Ampligen. The FDA has fast tracked all kinds of drugs for AIDS and other illnesses. There is no excuse for their continued denial of Ampligen. We should be allowed to try it. It’s true that it might not work for everyone, but let us try it if we want to.
Ampligen (R) needs FDA approval NOW.
It not only treats at least a subset of ME/CFS patients, it is likely the best Ebola drug available up to 100% cure rate. It binds to the virus rendering it incapable of destroying host immune system RNA. This allows the host immune system to launch an attack that destroys the Ebola virus. And it works on viral mutations, too. Read the results of the just completed Ebola/Ampligen study by US Army scientists: http://stks.co/b1deN
There is also, as you know, a grassroots call for a Congressional hearing to remove the roadblocks to Ampligen(R) FDA approval: http://www.cortjohnson.org/blog/2015/02/05/congressional-hearing-ampligen-roadblocks-fda-called/
I gave up on Ampligen years ago but with all the news about CFS lately I thought I’d check to see if there was anything new. I’m thrilled that there is hope. I will call, write, email anyone who could help move this along.
If they don’t approve it then they have something to hide. The FDA is a joke! Just turn on your tv and you will see one commercial after another from lawyers wanting to sue drug makers for the dangerous drugs the FDA approved over the years.Ampligen is safe.If they don’t approve it to make us healthy then we should make them sick.
I tried to send the letter, having major brain fog, Do you need to cut and paste or can you send directly from this site? I was not able to type my name to the form letter you provided.
Thanks,
Charleah
I have been to hell and back with testing stemming from Ann arbor, OSU, and now Cleveland Clinic where they are looking finally at CFS. There is no way I can put in to words how this has affected my life. I am currently off work due to this. I am the last person walking the face of the planet that thought would be on disability. I have two years of service left to reach my full retirement. I was previously very athletic and very active before this started 4 plus years ago. If there is a drug out there to help at some level it infuriates me to no end the FDA is not approving it. Who are they to make our decisions for us. If they had to suffer with this life altering disease and taking everything else like your a lab rat they would feel differently.
Hope it never gets to market besides 95% of patients diagnosed with CFS/Fibro fatigue/pain disorders have ‘undiagnosed’ types of Ehlers Danlos Syndrome a genetic born mutation of Collagen & its numerous Complications…Also found as well in some M.S. & Autistic patients…Where is the data on Ampligen in types of EDS???
I have had CFS for 40 years. As a young R.N. i caught EBV while working at a V.A. nursing home ward. In those days we rotated shifts every three weeks and this sleep deprivation wore down my immune system. Now after several more chronic viruses I am at my wits end. It is criminal that Ampligen has not been approved in this country.
The American medical system is way behind that of EU nations both in research and treatment.
Money driven Big Pharma and a profit driven healthcare system love to treat high cost elderly hospitalized cardiac patients and ignore less lucrative younger patients with chronic illnesses. I can see insurance companies denying claims for Ampligen even if it were approved due to the varying symptoms and causation of each CFS case . The billing code would always be wrong.
I think the repeated denial of Ampligen goes deeper than the FDA and involves Big Pharma and Insurance companies paying someone off to continue denials. Big Pharma likes band aid drugs, not cures. Why else is stem cell research less funded in the US than the rest of the world? The sad thing is that CFS is a huge cost to the Social Security Disability program and despite this the US still bends to Big Pharma.
I contracted CFS in 1987. Within two years my husband and sister had also fallen ill. I have been hearing wondrous tales of Ampligen’s efficacy since the early 1990s. So WTF FDA? The Australian government, in its wisdom and benevolence, approved the drug for CFS sufferers there and in NZ in March of 2015, I think. What’s their experience so far?
What is your source for the Australia approval? My understanding was that the company had some kind of pre-approval access program, but not formal approval: http://www.thestreet.com/story/13071572/1/hemispherx-enters-a-collaboration-with-emerge-health-for-the-commercialization-of-ampligen-for-chronic-fatigue-syndrome-cfs-in-australia-and-new-zealand.html
Hope is the thing with wings … I guess I was getting way ahead of myself and reading in. I thought Ampligen in Australia was a fait accompli when, upon review, it was only an agreement to market. Sorry for the misunderstanding. I should have known not to cling to optimism.
I was diagnosed with CFS here in Houston by Dr. Patricia Salvato In 2009 who jspecializes in treating AIDS and CFS patients for over 20 years. She ran multiple blood tests and found I had HHV6A levels that were extremely high. Unexplained night sweats,extreme muscle and joint pain, memory loss and excruciating headaches in the back of my head. It dawned on me nine years prior I had Mono at 31 which weakened my immune system allowing the retrovirus to cross blood/brain barrier. Dr. Salvato is not mentioned like Lapp and Peterson because she doesn’t participate in the Ampligen drug cost recovery trial. I was on Ampligen for a year under Dr. Lapp’s care and it was no picnic for the first six months. You get far worse until around six months then you turn the corner and start improving. I graduated from Rescue Swimmer School when I was in the Navy and believed nothing in my lifetime was as physically or mentally exhausting! I was very wrong indeed.
I was bedridden the first six months on Ampligen but could function well enough to take care of myself and drive myself. January 2011 I was 70% better and my employer was holding my truck driving job for me upon return! Unfortunately a month off Ampligen I relapsed and lost everything I gained which crushed me emotionally. With tears flowing I informed my employer of four years I relapsed and couldn’t work. That was when Ampligen was supposed to or so we thought get approved but Beth Unger and her minions made sure it wouldn’t.
There is blame to go around for many; Dr. Carter not selling out to Big Pharma is the political reason why it will never be approved. I learned some things others do not know while receiving my infusions. If Big Pharma had the patent the FDA would have fast tracked Ampligen without hesitation. The FDA will never grant Hemispherix approval because only Big Pharma is allowed to make the big bucks. Dr Unger was instructed to say no and was just following orders so you can’t blame her for it.
I’m a Desert Storm Veteran who like many of my brothers and sisters were given an anti-nerve agent vaccine that later came out when the IOM screwed the Veterans by changing Gulf War Syndrome to something without GULF WAR in the new name. I also like many was exposed to depleted uranium because my job was handling weapons but of course we didn’t know, imagine that. I firmly believe the vaccine and depleted uranium weakened my immune system that caused mononucleosis opening the door for HHV6A to reak havoc on my thyroid that no longer works. My testosterone level dropped to 127 about what an 80 year old man’s level. Not a 31 year old man who was healthy and working for Southwest Airlines loading planes in his fourth year of employment.
In closing I will never know really if I have Gulf War Syndrome or Chronic Fatigue Syndrome going on 47 years of age. There’s a reason the government isn’t funding either and I’ll simply say what Dr. Garth Nicholson said ” Mycoplasma Fermantans Incognitus”. The U.S. ARMY has this patented as a biological weaponised agent that cannot be found in any blood test only a PCR test that no specialist will order for fear of the government. April was my twenty four month of being on SSDI that I paid into for 26 years and waited three years four an ALJ hearing. Upon review the ALJ called my lawyer and said he doesn’t need to report he’s approved. His order was an mandatory full CDR in 24 months and the 25th it was in my mailbox. Two months later I was informed that my SSDI would continue but we will be checking in on you from time to time……that really creeped me out. So I waste away like the rest of you waiting literally for either a treatment or cure. I pray every night for the Lord to take me in my sleep so I’m no longer a burden to my Wife of fifteen years if the government has no intention on helping us with this haneous disease! God Bless all of us who are under a government that would do this to what the IOM estimate of 2.5 million Americans and 80 to 90 % are not diagnosed. I forgot we’re not allowed to give blood or donate organs I think that says it all……
What an incredible and difficult story John. To go from being so physically fit to so debilitated is a fall Shakespearean proportions. I would keep an eye on Dr. Klimas’s GWS work. She is getting funded – surely not as much as she would like – but better than ME/CFS I believe. The breakthroughs for ME/CFS patients could come out of her work with GWS. I will be reporting on it as it comes out.
Good luck and thanks so much for sharing your story.
I am desperately looking for access to Ampligen in Toronto. Will appreciate any advise.