The FDA denied the drug application for Ampligen, asking the manufacturer, Hemispherx, to complete new studies
Hemispherx released a statement on Monday indicating the FDA had denied their application of Ampligen to treat Chronic Fatigue Syndrome (ME/CFS) because of a lack of ‘substantial’ proof of efficacy and (rather ominously) because they did not have enough data to assess safety concerns.
The FDA’s denial of the application has not been made public and may never be made public (Hemispherx’s decision) but Hemispherx’s report and its actions, including a formal appeal of the decision, suggest the FDA’s requirements may be beyond their capabilities. Those requirements include having Hemispherx conduct ‘at least’ one clinical trial, complete various nonclinical studies and perform several data analyses. Hemispherx was not able to comply with similar FDA requirements in 2009 and it’s not clear why they would be able to comply now.
The FDA had other choices; they could have conditionally approved the drug with tight restrictions. They could have required a smaller trial within Hemispherx’s budget and, after that was completed, conditionally approved the drug (with more studies to come). The problem for Ampligen, after all, appears to be largely financial; Hemispherx doesn’t appear to have the money for the large studies the FDA wants and with their stock in the dumps, and investor lawsuits underway it’s hard to imagine they’ll be able to get it now.
Unless something changes the fight to get Ampligen approved appears to be over – for good.
The goal on the patient end was simply to find a way to get the money to do the studies this drug needs to get FDA approval but FDA doesn’t appear to be interested in finding ways to make this situation work…
They sure talk a good game, though.
Out of Touch Agency…
Things seemed far different a couple of months ago when, for once, things seemed to be trending in this disorder’s direction. After a 2012 law (FDASIA) specifically directed the agency to put a premium on finding ways to get drugs to people with serious disorders with few treatment options, it seemed the times were made to order for Ampligen and ME/CFS.
Citing what they called an ‘incredible’ response from the chronic fatigue syndrome community (over 750 testimonials) the FDA rep said they got it….this community is in dire need of treatment options and they were working for them.
The FDA’s public statements that they considered ME/CFS to be a serious and life-threatening condition also seemed to be setting the community up for a win. Woodcock could have been speaking directly about ME/CFS and Ampligen when she said
A promising but not yet fully evaluated treatment may sometimes represent the best choice for individuals with serious or life-threatening diseases who lack a satisfactory therapy.
Let’s see……Ampligen is definitely promising…..according to the FDA it’s not fully evaluated….. chronic fatigue syndrome is a serious disorder…it doesn’t have satisfactory therapies…check, check, check, check…..but no, Dr. Woodcock must have been referring to other disorders…
Indeed, as Dr. Woodcock indicated, the FDA has shown the ability to bend many times in the past. Citing a lack of other treatment options, it had recently gone so far as to approve a drug for lupus that didn’t even meet its final endpoints.
The FDA wants much more safety data for Ampligen but it does not need huge patient databases to make a decision. In a 2011 Congressional Hearing, Dr. Woodcock noted the FDA approved a drug for a disorder (N-acetyglutamate synthase deficiency) that affects less than 10 patients in the US on the basis of a ‘case series of 23 patients.
The FDA relies on doctors’ opinions to assist it in making its decisions but not with ME/CFS. At the FDA-sponsored Chronic Fatigue Syndrome Advocacy meeting Dr. Kweder stated that doctors’ statements are given weight in cases where other data is not available yet the FDA panel was advised not to strongly consider doctors’ reports at the FDA Advisory hearing.
The FDA also routinely requires post-marketing studies to address safety concerns not met by study data – but not with ME/CFS. In 2011 Dr. Woodcock noted that in the past 4 years the FDA had required drug companies to carry out approximately 375 ‘post-marketing studies’ which addressed safety questions that had not been adequately addressed prior to approval yet that was not an option for Ampligen even though its own Advisory Committee voted 8-5 that the drug was safe enough for marketing.
If the drug is safe enough and patients are made aware of questions regarding efficacy, then surely they’re capable of determining whether they want to try the drug. Should they not be able to make that decision?
What is the greater evil? Allowing very ill patients the opportunity to try a drug that needs more study but that doctors vouch for and studies suggest is safe or denying everyone the opportunity to even try the drug for fear that something bad is going to crop up?
The Ampligen application had problems; the age of the studies, the shifts some studies took in midstream, the questions about data, and the FDA’s harsh response suggested that this drug application had more problems than usual. But it also had its successes; it did meet its endpoints and the drug appeared to be safe and physicians and patients strongly vouched for it.
Magnifying everything was the likelihood that the request for more studies doesn’t just put a promising treatment off into the future; it very likely kills it forever.
Bob Miller knows what that means….
“It means I’ll slide back and it’s not just me. Where we’re going is back to our beds and being chained to our houses.”
For everyone chained to their houses or their wheelchairs or whatever, it means an opportunity you’ll never have…
Agency With Its Head Stuck in the Sand
The FDA recognizes that ‘orphan’ disorders; ie disorders that affect small numbers of patients – don’t get the funds necessary to produce large studies so it relaxes its requirements for them. The goal, after all, is to help people get the drugs they need…not to follow some standardized protocol for testing.
CFS is not an orphan illness – too many people have it – but it shares some important features. The fact that it has, except for Hemispherx been orphaned by drug companies leaves ME/CFS and patients with rare diseases dwelling in a treatment ‘ghetto’ that’s devoid of FDA-approved drugs. The only differences between the two are that there are a lot more ill ME/CFS patient and that people with rare diseases get help and people with ME/CFS get penalized for having a disorder that’s different.
If the FDA’s role is to truly to bring safe and effective drugs to ill patients it must take into account factors that impede that. That includes recognizing the difficulties disorders like ME/CFS have in attracting major drug company support and the difficulties small drug companies like Hemispherx have in participating in an unbelievably expensive drug approval process.
After the FDA Advisory Meeting, I told the same FDA rep who’d announced that the FDA had ‘gotten it’ regarding chronic fatigue syndrome that she’d just participated in forever killing Ampligen for ME/CFS patients. I asked her if Hemispherx had taken 20 years to get to this point and had been unable to produce the study the FDA wanted in 2009, why did she think it would be able to produce another study in 2012.
This was a difficult question for her for at least three reasons; she’d publicly recognized how much the community needed new options, the dramatic patient testimony still lingered and many of the panel members agreed the drug did appear to be effective for at least some patients. Had she just supported removing the one drug possibility from a community that had none?
She basically rejected the question stating that economic feasibility was not their concern and they couldn’t factor that into their calculations. But why not? Economic feasibility is factored into the equation for people with orphan illnesses. In fact, direct federal support is sometimes given to small orphan disorders to produce drugs.
But chronic fatigue syndrome, ironically, is too big to meet orphan disorder status and so the FDA expects products for it to jump through the same hoops that drugs for cancer or heart disease do.
But chronic fatigue syndrome is not cancer or diabetes and or asthma; instead of several hundred million dollars a year in research, it gets six. It’s a relatively young disorder to the research community, it’s probably larded with subsets and it’s going to be a while before it’s ‘clean enough’ for drug companies or large numbers of researchers to be interested in it. It’s probably not going to get the big studies the FDA wants.
Until disorders like ME/CFS get acceptable levels of support they’re going to need some help, and until they get it, the patients are going to feel the pain that the lack of flexibility, understanding, and ultimately commitment at the federal level produces.
It appeared for a while that the FDA was the one federal agency that put the patients first; their mandate after all is to get drugs out to ill people, but they didn’t put patients’ needs first in this case; in the end they, like the NIH and the CDC wanted ME/CFS to be cancer or heart disease and when it wasn’t they turned it aside.
In the meantime, the patients bear the weight of having an illness that is different.
This disease really needs a champion -someone in power in the federal arena who is courageous enough and committed enough to take some chances – and we just don’t have that in the FDA or the NIH or the CDC.
Hemispherx To Appeal Verdict
Hemispherx formally appeals the FDA’s verdict
Hemispherx is not going quietly. In their press release, Hemispherx hit the FDA hard, essentially accusing them (in polite terms) of ignoring a Congressional mandate (FDASIA Act) to bring stakeholders (sponsors, patients, researchers, and clinicians) together to ‘overcome challenges and find solutions.
In the end, Hemispherx believes the FDA simply made the wrong decision and they think they have the data to prove it. Several Hemispherx reps reported great frustration at the lack of opportunity they felt they were given to respond to issues the FDA raised at the FDA Advisory Meeting. Hemispherx’s request for an ‘end of review conference’ prior to their submitting a formal appeal of the FDA’s decision should remedy that.
The conference is designed to address the issues the FDA raised in verdict (‘Complete Response Letter’) as well as highlight clinical evidence from physicians and patients and other data indicating Ampligen’s safety and efficacy. Not surprisingly, they cited Dr. Klimas recently published letter of support.
Hemispherx is taking the risky option of further angering the FDA with their appeal. If they had a better option they would probably take it but with their stock plummeting and expensive new studies in front of them they may have no better options.
- Check out the FDA/Ampligen Resource Center for complete coverage of the FDA decision
- Check Bob Miller’s Hunger Strike, Ampligen and the FDAs decision on Bioworld
- Check out a new Reno news story on Bob Millers strike
Hard-hitting article. Thanks, Cort.
Thanks Merry…
I think assigning all the blame on the FDA is overlooking a lot of the misconduct and poor business decisions make by Hemispherx. Perhaps they need to consider working with someone like WPI and giving up the hope of becoming a mega pharmaceutical company on the back of CFS. If they really care about our community they need to look for financial help and do the necessary work.
I got what you say but I think its more about finding a way to make this drug work. Does Hemispherx have the financial capability to carry out the studies the FDA wants? I don’t actually know but my guess, given that this is their ‘blockbuster drug’, that could make them all rich and they still haven’t done the studies that they don’t the financial wherewithal to do that….
It would make sense for them to partner with other companies and it would certainly make sense for them to partner with investors (and I’m sure they have). My guess is that they’ve tried to do that and have not been wholly successful since they’ve been unable to come with the funds comply with the FDA’s requirements.
If that’s true then where are we? We’re at the point where we have a drug that certainly works in some patients which is about to disappear. If the FDA is committed to finding ways to get drugs to communities in need then Ampligen would be a great place to demonstrate that. I know this isn’t any easy problem but
I just don’t see any evidence that the FDA is trying to do that.
They may be quite correct in their decision to require more studies – in fact I’m sure they are – but if that decision leads to Ampligen disappearing then somethings wrong because of doing that actually removes a beneficial product.
There’s got to be a way for the FDA to foster drug development in small pharmaceutical companies that don’t have the funds to do the big studies or in companies that simply messed up! Hemispherx messed up! There’s no doubt about that..Now lets find a way to get a good drug to a community that needs it.
I quite agree, Cort. “..Now lets find a way to get a good drug to a community that needs it.”
Definitely. This is NOT over until there IS a drug. Sooo, Plan ‘B’ it is.
( We already have a name..Plan ‘B’. )
And I have no doubt there will eventually be a drug. Or a treatment of some efficacy.
Sometimes I wish I were not too ill to review, to study the history of AIDS research , the political and money drivers, the pattern that led to breakthrough. And too, I often wonder how much AIDS’ own chain of expenses, massive as it was/is..actually deters efforts towards breakthroughs for CFIDS. I feel sometimes that there is perhaps some correlation. But..maybe I am grasping at straws here. Pretty desperate, our plight. What if the cause is in the air, the water, our food…ubiquitous? Ugh.
The FDA is not the only out of touch Gov’t agency (that’s why I lean libertarian, both parties suck), I try to do some activism, but am not going to expend to much effort on continually trying to get our gov’t to fund and recognize the severity of our illness. The XMRV saga brought much attention, and more research via private institutions is happening now, I’m not going to hold my breath and wait for the gov’t run by clowns!
GG
Great review, Cort. We need people emailing and calling the FDA every day to keep the pressure on. Here is a list:
Patients should email:
kathleen.sebelius@hhs.gov, howard.koh@hhs.gov, jarrettpublic@who.eop.gov, margaret.hamburg@fda.hhs.gov, janet.woodcock@fda.hhs.gov, sandra.kweder@fda.hhs.gov, 511bobmiller42@gmail.com
Steven G Chilinski, MD
kathleen.sebelius@ hhs.gov; howard.koh@ hhs.gov; jarrettpublic@ who.eop.gov;
margaret.hamburg@ fda.hhs.gov; janet.woodcock@ fda.hhs.gov; sandra.kweder@ fda.hhs.gov;
511bobmiller42@ gmail.com
( some parties are not able/don’t know how to access the protected addy’s apparently..so I posted these with a SPACE after the @ sign. Do NOT forget to REMOVE the space when you send your emails )
Thanks Deborah, I was at a loss myself…..I had never come across this before.
Great article, Cort. Fascinating analysis of what’s going on.
Like anybody, the FDA should be judged by what they do, not what they say. They say they ‘get it’ and yet they deny us Ampligen. Ergo, they don’t ‘get it’.
Yes…we’ve heard so many time that federal officials are sooo concerned and sooo committed but it all ends up the same- little or no action taken and things go on the same.
In this case its a bit worse since we’re actually losing something – an important drug – as a result of federal action. Hemispherx definitely has alot to make up for AND allowing this or any other drug that shows promise, and everybody agrees that this shows promise, to disappear – without making an effort to forestall that – runs very counter to what the FDA purports to be about.
Nobody is saying that the task is easy but in extraordinary circumstances you do extraordinary things and I think ME/CFS – a large disorder with alot of suffering and no treatments – presents a extraordinary circumstance that calls for the FDA to everything in their power to assist – to go beyond where they’ve gone before – even if it involves assisting a company they don’t much like.
Hi Cort,
Very passionate post! And I happend to agree that something needs to be done as one of my highschool friends Mom suffers from ME and there needs to be a treatment option! I had done a lot of reasearch myself prior to the Advisory Committee to gain knowledge on how this will go with the FDA. I found and looked at a similar disorder Lupus (which you mentioned) that recently had a drug approved. It was Benlysta, IV administered, estimated same population, unknown bio-markers and unmet medical need as well as botched trial data. I thought for sure the FDA would decision would go the other way based on this approval. Please feel free to use this in your arsenal! The last one is the FDA briefing Docs for Benlysta. After reading this, one has to wonder why Ampligen was not given a fair “trial”.
This is long but I will include the website and some bullet points from the reports, but it really makes a point on how the FDA is a hypocrite.
http://healthlifeandstuff.com/2010/04/benlysta-limited-efficacy-for-lupus/
The current treatments for lupus are limited and not so great. They are commonly used off-label and can cause side effects over use. For several decades, due to the complex nature of the condition, there has been no new treatment for lupus.
One of the first major studies into Benlysta showed no superiority over placebo. Instead of dropping the project, the company looked at the study and changed its design to improve how their treatment works. They developed an individual responder index to measure improvement, removed participants who were non-seropositive and changed some of the time related measures.
The next two Phase III trials had better results. One, the BLISS-52, had 865 participants and 51.4% response rate on 1mg/kg dose and a much better 57.6% response to a 10mg/kg dose. And the BLISS-76 of 819 patients had 40.6% response to the lower and 43.2% response to the higher dose.
The bad news? In both trials, the response rate was not that much better than placebo – 46.3% responded to placebo in the first, and 33.8% in the second study. That’s about a 10% difference in efficacy from placebo to treatment.
From WEB MD about Benlysta:
An FDA advisory panel last November voted 13-2 in favor of approval. But the panel noted that Benlysta is no wonder drug. Overall, it offered a modest benefit. Only 30% of patients who took the drug in clinical trials saw a benefit.
That’s what happened in clinical trials of Benlysta. On average, patients taking the drug were able to reduce the doses of prednisone they were taking.
In those studies, most of the benefit of Benlysta seemed to come from improvement of skin rash and ulcers in the nose and mouth.
In clinical trials, it wasn’t clear whether Benlysta helped patients whose lupus affected the kidneys, brain, and blood vessels.
In clinical trials, there were more deaths and serious infections among lupus patients taking Benlysta than among those taking placebo plus standard therapy.
The most common side effects in clinical trials were nausea, diarrhea, and fever. Patients also commonly experienced infusion reactions, so treatment with an antihistamine may be needed before each infusion.
From the FDA briefing doc prior to the Advisory Committee meeding for Benlysta (can be found on FDA.GOV)
Efficacy Conclusions
Although Studies 1056 and 1057 demonstrated a statistically significant increase in belimumab-treated patients achieving a response, defined as a 4-point reduction in the SELENA-SLEDAI, no worsening in the physician global assessment, and no new 1A/2B BILAG domain scores, there are a number of findings in these studies that also raise questions regarding the efficacy of belimumab:
o Lack of a consistent dose-response effect—in some analyses, 1 mg/kg appears to provide a greater treatment effect than 10 mg/kg and in other analyses, 10 mg/kgappears to be more effective, e.g., SRI Subcomponents (Table 10), SLE Flares
o Lack of statistical significance for Responder Index results at Week 76 (Table 17)
o Lack of statistical significance in increasing the proportion of patients able to reduce prednisone by at least 25% to less than 7.5 mg/day (Table 18)
o Lack of consistency between studies—e.g. reduction in BILAG flares (Table 22), 10 mg/kg increased time to BILAG flare in Study 1057 but not Study 1056; change in PGA (Table 10)—1 mg/kg better in Study 1056 and 10 mg/kg better in Study 1057; effect in the Native American subgroup (Table 15)—favorable in Study 1057, unfavorable in Study 1056.
o Lack of efficacy for the African heritage subgroup of both studies (Table 15)
On the other hand, post-hoc exploratory analyses of the effect of treatment on various organ system manifestations overall appear to be suggestive of a treatment benefit with belimumab (Tables 25 to 28). Some inconsistencies were again noted, but as the numbers of patients with particular organ system involvement was small in most cases, it is difficult to draw definitive conclusions.
The evidence for the efficacy of belimumab in the treatment of SLE is far from consistent and unquestionable. There could be a number of reasons for this, but discussion would be primarily speculative. Given the data as they are, a more relevant question is whether the degree of efficacy demonstrated is worth the apparent risks of treatment, and whether they are worth the anticipated risk of treatment if other immunosuppressives are required to treat serious SLE manifestations.
Safety Review
There were 14 deaths reported in the controlled period of the IV SLE trials, with one additional death occurring in a patient 15 weeks post treatment discontinuation, for a total of 15 deaths, as follows: 3 patients died of cardiovascular (including stroke) events, 5 patients died of infectious etiologies, 2 patients committed suicide, 2 patients died of unknown causes, 2 patients died of SLE-related complications, and 1 patient died of a malignancy
Based on 14 deaths observed in the controlled period of the IV SLE clinical trials, and one death occurring 15 weeks after patient withdrawal, the death incidence rate per 100 subject-years was almost twice as high for belimumab as for placebo treated subjects
Because of its mechanism of action, belimumab would also be anticipated to increase the risk of infections, including serious infection. In fact, as shown in Table 40 below, infections were the most common system-organ-class (SOC) reported, and the exposureadjusted-incidence of serious infection was higher in the combined belimumab groups compared to placebo (5.2 vs. 6.0 infections per 100 patient-years for placebo and belimumab groups, respectively).
Page
Neuropsychiatric manifestations are a not uncommon complication of SLE, although the actual incidence of neuropsychiatric involvement appears to range widely depending on the population studied and the specific manifestation in question. Some sort of cognitive dysfunction is reported in the majority of patients (from 55% to 80%). Headache (24 to 72%) and mood disorders (14 to 57%) are also commonly reported. Depression and anxiety are common in SLE patients and have been reported to occur in 24 to 57% of SLE patients. Frank psychosis is relatively uncommon (up to 8% of patients).7 Not unexpectedly, these adverse events have been reported in the belimumab SLE clinical development program; however once again, there was a numerical imbalance against belimumab, with more belimumab-treated patients reporting neurologic and psychiatric adverse events, SAEs, and suicides. Ascertaining the role of belimumab in this imbalance is again difficult, as patients were exposed to placebo-treatment for shorter durations and there was unequal randomization. However a promoting or permissive role of belimumab cannot be ruled out.
Because of the overlap in symptoms with infusion reactions, hypersensitivity reactions, and anaphylaxis, it is difficult to ensure that adverse events were adequately captured and classified.
Most patients ( 92%) experienced an adverse event during the controlled IV SLE trials.
Safety Conclusions
Treatment with belimumab appeared to be associated with an increase in death, serious adverse events, infections and serious infections, and neurologic and psychiatric adverse events/serious adverse events, including 3 suicides in belimumab-treated patients through the data cut-offs of the BLA submission. This imbalance holds true even when the incidence of these adverse events is adjusted for exposure. In some cases, the magnitude of the difference between the belimumab and placebo treatment group in the trials was relatively large, such as the almost 2-fold increase in exposure-adjusted incidence of mortality for belimumab-treated patients.
Conclusion
In two randomized, controlled trials, belimumab demonstrated a statistically significant difference in the proportion of responders as defined by the primary endpoint of SRI in the treatment of autoantibody positive SLE. However, the results in Study 1056 are less robust, and for both studies data from other endpoints and subgroup analyses were not consistently supportive. In light of this somewhat marginal efficacy, the relative safety profile of the product must be weighed. Increased risk of serious infection is almost a given with biologic immunosuppressives, so the risk of infection with belimumab is expected
Clearly there is a need for effective therapies in SLE. However whether belimumab’s benefits sufficiently outweigh its risks is the crux of the issue. Given that flares and steroid reduction may not be impacted, is a reduction of 4 points in the SELENASLEDAI (the main component driving Study 1056’s efficacy result) clinically meaningful? If belimumab only has a modest effect for some patients and manifestations, is a possible increased risk of death, infection, or neuropsychiatric adverse effects worth the potential benefit?
Additionally, patients with serious SLE manifestations, such as renal and CNS lupus, were excluded from the studies. If belimumab is not effective for serious SLE manifestations, then it is likely that more potent immunosuppressives, including possibly other biologics, will be needed; the safety of combining of combining those immunosuppressives with belimumab will not have been adequately evaluated.
Thanks John for all that information,
Ampligen is certainly not a miracle drug (except for occassionally when it is; aka Anita and some others), and some people are effected moderately by it and some people show no response at all; it is able to move one of the most difficult to achieve factors in CFS – endurance – across a broad range of patients which is saying something; there are no hints of death due to Ampligen and death rates appear to be doubled with Benylstra and…Benlystra appears to cause more neurological problems than placebo…… This is an amazing statement: “he magnitude of the difference between the belimumab and placebo treatment group in the trials was relatively large, such as the almost 2-fold increase in exposure-adjusted incidence of mortality for belimumab-treated patients”…..ultimately it sounds like its quite a bit more effective and safer than Benlystra :).
Hemispherx clearly dug themselves a hole with their study changes and lapses in data and ME/CFS patients do have other treatment options but there are no other FDA approved drugs and many of the patients who did respond had tried everything….
Very interesting…thanks.
Hope your friends mom does better
Its good of you to look into this 🙂
All I will say is this: CFS/ME is like dying a thousand deaths with no end in sight. Ampligen being approved was my light at the end of the tunnel. I refuse to leave my 4 year old son and my husband for 6 months and deplete my son’s college fund. Relocating to another state hundreds of miles away, should not be something we have to consider. Eight months ago, I was a managing attorney . Today, I am housebound and have been since July. I was very sick from 2009 until present, but I pushed until my body totally crashed in July. I seem to worsen daily. I just want to be a mother to my son.
What a heartbreaking experience…..and yet repeated by so many. It’s really not acceptable to have to leave ones family and spend all ones savings and go live somewhere in order to try and get better. We should be able to do better than that here…and most people can; most people with a disorder can probably find a treatment fairly close to them..
I wish I had an answer…
Cort, you said: “If that’s true then where are we? We’re at the point where we have a drug that certainly works in some patients which is about to disappear.”
Why is it about to disappear? That seems to be a presumption, and I doubt Hemispherx would agree with you.
While I do think that it should be approved on a restricted basis, Hemispherx should be held to account for their trying to evade the 2009 request for new trials by reshuffling data (and deleting the patients who did poorly) to make their case look better. That’s galling to me, and I can understand the why the FDA wasn’t pleased.
Also, one of the most famous original Ampligen patients was Nancy Kaiser. Here is what she had to say about the drug (from the NCF website):
“The drug did help me initially and those participating in the original study were all so thrilled to be involved in a possible treatment for this disease. However, in time it started causing more and more problems. When I was taken off the drug, it was very apparent the drug is not the answer. When anyone asks me about Ampligen my answer is very simple and straight forward. In my opinion, I would never advise anyone to take the drug nor interact with Hemispherx. In one of their patents (patent # 5,958,718) statements were made about my improvement and background which were not true. I reported this to the FDA and there was never a return acknowledgment from the FDA. Also, there has never been a long term assessment regarding my health by Hemispherx as to the safety of this drug and I was on it for five year.– yet they keep talking about the drugs long term safety record of Ampligen. ”
Kaiser’s experience was also noted on cfs-news.org:
“On Ampligen, Kaiser began her remarkable recovery, becoming the poster girl for the drug. There would be stories about her in Newsweek and Reader’s Digest. But those stories didn’t report what Kaiser, now 58, says she really thought about Carter and HEM. “Because I believe the drug saved my life, I put up with Dr. Carter’s demands,” she says. “Dr. Carter told me repeatedly that if I didn’t write more letters to the FDA and Congress, I would lose the drug. It shouldn’t be up to sick people to get a drug approved. It should be up to the drug company to do good studies and present data.”
And finally from ncf.org, in a tribute to her after she died:
“Although there are many reports saying she was forced off the drug, it was Nancy’s decision to stop the infusions because she found the drug not only was not helping but was, in fact, worsening her condition.
In spite of the predictions of the manufacturers of Ampligen, Nancy did not die when withdrawn from the drug but actually improved! A decade later, she urged the company and Dr. Peterson to do a longterm study of those who had first been on the drug who were still alive. She did not have success with that idea. Yet, she recently was urged to go back on the still experimental drug by the same physician who had diagnosed her years ago! She adamently declined the offer. For years, she had offered to have her phone number given out to CFIDS/ME patients who were considering a trial with the drug and she informed them that many who had been on Ampligen were now dead and many of the factual reports and test results given out about those patients by the manufacturers were not accurate. ”
http://www.ncf-net.org/forum/fall-vol12-3-2.htm
Yes, there are patients who have improved dramatically, but there is also the other side of the story.
Thanks for telling the other side of the story :).
Excellent synopsis on the approval process for Ampligen or lack of approval. What is the time line for Hemispherex to appeal?
I am not suffering from the disease. But I did go through the treatment for HEP C. 2008. Side effects were awful..not everyone is cured by the process…
It was my choice to go through treatment. I was given the scenarios of what might happen. I was told about the side effect(but until you go through the treatment you really don’t know how bad they are) BUT IT WAS MY CHOICE and I am glad I did for I am cured.
This drug cocktail to had trials…it just feels morally wrong not to have something for these for these patients.
Here is a Reply posted very recently on another CFIDS related website by me (a Google search will give that website, as well as another recent article by “Med Page” that is much more factual and well-researched than the one here by Cort Johnson):
John Chavez 02. Feb, 2013 at 11:02 am #
I WAS ONE OF MANY AFFLICTED AND NEGATIVELY IMPACTED BY AMPLIGEN DURING THE FDA PHASE III TRIALS.
I was one of 25 subjects in San Diego CA during the 1999-2000 FDA Phase III drug trials for Ampligen. The trial was “overseen” by Dr. Anthony Mercandeti of San Diego; Dr. Mercandeti was seldom present, visible or available during the drug trial. There were very few drug trials for Ampligen.
Of the 25, at least 4-5 were immediately, negatively and severely impacted during the drug administration portion – I was one of those. I had an immediate major relapse of a well-documented 20 history of CFIDS which had been mostly in remission over many years. While it was not known who received a placebo and who received Ampligen, those of us who had a severe and immediate negative reaction knews. We, the afflicted, withdrew from the drug trial.
The process was a farce, a sham, and poorly supervised. The bi-weekly reports were trivial and mostly relied on the “Karnofsky” scores which were superficial and almost irrelevant. The medical staff not only did not want to hear of any negative impacts, they showed the afflicted as “dropouts” with no notations of negative symptoms or impacts. They refused to record our complaints and to show us our drug trial records (so much for HIPPA and FOIA). The FDA did receive complaints directly from those afflicted, but quickly shelved or disregarded those reports.
I now find it interesting that the FDA moves forward, albeit over 10 years later, despite known information that Ampligen has some serious negative side effects and is in fact counter-productive for many.
How is it that those of us WHO WERE THERE AND AFFLICTED do not show up in the review and approval process?? We KNOW FIRST HAND; everyone else gets second and third hand laundered and redacted info.
Actually, I see the rejection as good for patients. If you look at the FDA analysis you’ll see that there is pretty much no difference at all between Ampligen and placebo. It looks like they got their positive results by basically cherry-picking a particular part of the results. Also Ampligen has a lot more adverse affects than placebo. Again they deliberately hid this fact in their study by saying that 95% of PEOPLE had adverse effects (rather than saying that the actual NUMBER of adverse effects was much higher in the Ampligen group!)
So really, the drug seems expensive, dangerous and worthless. It would be detrimental to patients for the FDA to approve it just because there are no other drugs. It also seems counter-productive for CFS patients to be ignoring the science and clamouring for it. It just makes us look like a bunch of loons.
There are other ways you can help yourself with CFS that are a lot more useful than Ampligen.
This situation with the Ampligen is NOT over yet! I refuse to believe anyone of ethics would deny a chance to the sickest of patients. It is not about money, anymore, it is about making guided decisions within an agency ‘for the greater good’. Why is everyone assuming that it is futile because of inconsistent conclusions held by the FDA? Hemispherx can rebound financially, as well as participate in fast tracking compassionate care, while doing trials/studies of specific relation to FDA’s non-approval.Let the FDA fund the studies. I had a wonderful, life changing response to the medication, and was a different person when given a large part of my life back. We cannot give up hope, and must believe in our government doing something of merit for these patients. I think they must visit the homes of those most severely affected, and see how life-robbing M.E. is. Of course people are going to die…this is a very serious medical condition that is NOT being properly recognized as a killer. While we hate to see people lose their lives, it is this very fact that SHOULD make people sit up and take notice! Why not take this approach, and hit hard with the truth that the public doesn’t even know…they still think it is about being tired! Death rates will go up as hope flounders. I still think this is insufficient information concerning the FDA decision….We need the Freedom of Information Act brought in, and a series of explanations from certain officials. This decision resembles and continues to downplay accuracy of medical facts, lack of consistent definitions and a lack of a diagnostic codes. This responsibility is within our government to acknowledge what has happened to patients, how discrimination started and has continued, and education of these illnesses and subsets could effectuate change. Give the FDA a chance to revise and recommit to saving lives!