Mary M. Schweitzer, Ph.D.
Thank you for the opportunity to speak to you today.
I am speaking to you today as a patient with CFS/M.E. who has been taking the experimental drug Ampligen since February 4, 1999. The out-of-pocket cost to my family will be $40,000 for this year alone. But I am grateful I have had this opportunity.
This time last fall, I was tested for the defective 37kDa RnaseL that Dr. Suhadolnik discovered in 1995. I was positive for the 37kDa, and my count was 12 (which was high). At the same time, Dr. Ablashi, the co-discoverer of HHV-6, tested my blood for the virus. He found that my leukocytes were riddled with HHV-6a DNA.
On July 12, 1999, 22 weeks into Ampligen treatment, my blood was tested again for the 37kDa and for HHV-6a. The 37kDa was gone, and so was the HHV-6a. Dr. Ablashi recently presented evidence about my case at an international conference on herpes viruses.
Six years ago, Dr. Ablashi and Dr. Paul Levine were able to eradicate HHV-6 in vitro using Ampligen. Today I stand before as the first patient for whom it has been possible to demonstrate that the same thing can happen in a real person. Why has the government shown no interest in this? [Several government representatives rose to protest this statement, but six months later no one has contacted me, and I must therefore conclude that they still have no interest in a drug that shows strong evidence of being the only way to destroy HHV-6.]
Those of you who remember me from past CFSCC meetings (I have presented evidence at every CFSCC meeting since the first one in April 1997) remember a person who used a wheelchair, or walked in a halting manner with an irregular gait. You remember a person who could barely speak to you without collapsing afterwards. You remember a person who would struggle to remember the rest of a sentence. You remember a person who was deteriorating, and was frightened by that deterioration (though I may have tried not to show it here).
I started walking again in March. We took the wheelchair back in April. I began driving again in May. I read an entire novel in August! And I danced at my son's wedding in September.
I cannot describe for you the exquisite joy of walking again, really walking. I thought I would never be able to do it again.
I believe that I am first-hand evidence of several things: there is a clue to the disease CFS in the 37kDa RnaseL marker. There is a clue to the disease CFS in the presence of the disease HHV-6a. The reason so many patients seem to "start out" with mono is that the mono itself may be a symptom of having the disease: the Epstein-Barr virus had also reactivated last fall, but is once again gone. And Ampligen is an effective drug.
I am also here to represent patients on Ampligen who have formed a new organization called the Association of CFS/M.E. Ampligen Patients. We are careful to do nothing to prejudice current trials in place, but we have a number of concerns that we would like to see addressed here.
CFS/M.E. patients have suffered long enough from public and government apathy towards this disease. We ask the CFSCC to require a report, in writing, from the FDA, explaining what impediments remain to the fast-track approval of the drug Ampligen, the only drug currently in FDA trials specifically for CFS/M.E. [and the only drug that has shown an ability to permanently destroy HHV-6].
- Why have we never been given a satisfactory answer to why Ampligen is not being fast-tracked?
- Why does the FDA website contain only one outdated memo on Ampligen that portrays the drug in a very bad light? The memo, from 1991, states that Hemispherx's request for a Phase III study is denied until they can present further evidence that the drug is not toxic. Nowhere on that website can you learn that Hemispherx did meet that requirement, that the FDA gave permission for cost recovery studies to start in June 1997 (that is the 511 study I am in); and that the FDA gave permission for double-blind (Phase III, Ampligen 516) studies to start last year. [I was informed by Dr. Cavaille-Coll that there is a second memo online, from 1993, but as that memo is as outdated as the 1991 memo, it only served to make me even more perplexed as to why the memo from the summer of 1997 approving the cost-recovery studies, and the memo from the winter of 1997-98 approving the Phase III trials, have not yet been put on the website.]
We ask that this committee request the FDA to update its website immediately to reflect the current state of Ampligen trials.
- The gag requirements for the Ampligen 516 patients are too stringent, and stem from a misplaced emphasis on the vernacular (as opposed to scientific) definition of a "placebo effect." Patients in the Ampligen 516 study are forbidden to discuss their symptoms in the fear that it will promote a "placebo response." Those of us in the 511 are concerned that that has left us unable to help them when they encounter problems.
CFS/M.E. is an enormously complex illness. The ten month period during which the study is to be blinded risks another type of danger - patients will develop CFS/M.E. symptoms having nothing to do with Ampligen and not be properly treated for them. Many of the patients in the study, for example, have never been tested for dysautonomia, Dr. Rowe. Of course, that's well within the CDC constraints of a "CFS" study - but a patient who experiences problems because of dysautonomia has no real way to find that out. This is but one example. Why must these patients be put through this? It is the only way they can get Ampligen; they cannot afford $40,000 a year. The company can't afford the level of compassionate care that would be required to meet the needs of so many indigent patients. Surely there is a better solution.
- Patients in the Ampligen 516 are receiving IV infusions twice a week, without knowing whether they are getting Ampligen or a simple saline solution. Their doctors do not know who is getting Ampligen and who is getting IV saline either. While we appreciate the logic behind double blind studies, we suggest that there are other ways that are scientifically sound to test the efficacy of this drug. IV infusions are an invasive procedure. Ampligen patients are known to have weak veins. The trial period for the first group has lasted ten months. During that period, some patients have been forced to endure the addition of permanent artificial ports to receive their infusions. Is it right to require this of someone who is very ill, and is only receiving a saline solution? We believe that it is unethical for the FDA to insist on double-blind studies when patients are severely ill, and when the protocol requires an invasive procedure such as IV infusions.
- The secrecy required for 511 and 516 trials is not serving the needs of the public. Ampligen needs to be shifted to the next level of experimental drugs, where it can be used openly in trials conducted at universities and major research centers. We are way past the time for a double-blind study as to whether or not it has any effects! What we need are studies to show how long a patient needs to stay on the drug. Studies to show what other drugs work well in concert with Ampligen. Studies to tell who should try the drug, and who should not. And, most of all, studies guided by the philosophy of outcomes-based research.
- We believe that Ampligen would have been approved a long time ago had not the U.S. federal government refused to face the severity of the illness CFS/M.E., and the dangers this disease poses to the nation as a whole. The same attitude that led to the NIH insisting that only 4-10,000 patients in the U.S. had CFS/M.E., a year and a half after the CDC revised its own estimates to 400,000 patients, has kept this drug from being available for CFS/M.E. patients. The same attitude that led to funds being diverted from CFS to other uses within the CDC, has kept this drug from being available to CFS/M.E. patients. As long as so many within the U.S. federal government believed this was a disease caused by "modern day stress," that it affected only a few upper middle class white women, and that patients got well in two years anyway - all misguided assumptions that should have been corrected many years ago - it was too easy for the FDA to assume any benefits from Ampligen must have been psychosomatic (i.e., "placebo effect"). As long as the public was denied the truth about this disease - and continues to be denied the truth about this disease - there is no one to ask "What happened to Ampligen?"
As an example of information that has been available for years, but hidden from the public, let me quote from a court decision ordering an injunction to prevent Hemispherx from withdrawing Ampligen from 15 patients after the closure of the 1991 double-blind trials:Reitz et al v. Hem Pharmaceutical Corp, December 1991If Ampligen approval is not speeded up, the patients in the current double-blind face the same potential fate as the patients who successfully argued for the injunction in 1991.
U.S. District Court, District of Nevada
"... The first amendment to the protocol reduced the study from 12 months to 6 months, provided that only significant responders would receive Ampligen in the open-0label phase, provided that such patients would have to pay for the cost of infusing the Ampligen, and provided that those patients not deemed significant responders would be discontinued until establishment of efficacy. ... we have concluded that efficacy has been established, entitling even those not designated as specific responders to receive Amligen on an open-label phase.... Guthrie ... is entitled to ... receive the drug for free and will receive the drug for 12 months.
"... We conclude for each plaintiff that without the preliminary injunction, on their contract claims, each plaintiff will suffer irreparable harm that outweighs any harm to defendants in granting the preliminary injunction.... Plaintiffs' potential harm should we not grant the injunction is quite high... the length of time for providing Amligen is six months, not twelve months. This second six months without Ampligen is serious potential harm to such plaintiffs.
"Uncontradicted evidence shows that if patients on Ampligen are removed from Ampligen and then placed back on Ampligen at a later time, the Ampligen drastically loses its effect. Consequently, all plaintiffs face irreparable harm without the injunction ..."
If Ampligen approval is not speeded up, patients who go into the 511 and run out of funds face the same dire fate as argued by the court: "The second six months without Ampligen is serious potential harm to such plaintiffs."
If short-seller Manuel Asensio succeeds in his mission to bankrupt Ampligen (and thereby make a fortune for himself), all Ampligen patients face the risks so described. This is a real threat until the drug is approved.
If the FDA turns its back on us again, as it did before, we all stand to face the same "irreparable harm" as the earlier patients.
This ruling occurred EIGHT YEARS AGO. Why isn't Ampligen approved today?
- In the fall of 1997 Congress held hearings for a law to speed up the process of drug approval. The husband of CFS/M.E. patient Sara Milliner testified that she needed access to Ampligen now because her condition with CFS/M.E. was deteriorating. As a result of the testimony of Mr. Milliner and others, Congress passed the FDA Modernization Act of 1997. Two years ago. This new law established provisions for fast tracking drugs if they met certain conditions.
At the CFSCC meeting in April of this year, Dr. Marc Cavaille-Coll stated that Ampligen meets the four requirements for fast-tracking. Dr. Cavaille-Coll agreed that:
- CFS is a serious, life-altering illness.
- There are no other drugs available to substitute for Ampligen. (Indeed, this is the first experimental drug to ever go through the FDA approval process for CFS.)
- Ampligen has demonstrated promise as an effective treatment for CFS.
- There is no evidence that Ampligen is seriously toxic.
I asked, why then hasn't Ampligen been fast-tracked?
Dr. Cavaille-Coll answered, "Ampligen can not be given 'Fast-Track' Designation until the Phase III studies have been completed." He also stated that Ampligen could not be given "Fast-Track" designation until a New Drug Application had been filed by Hemispherx Biopharma, the makers of Ampligen.
According to FDAMA, however, "Fast-Track" designation can be given at any point during the investigation of a drug. Indeed, AIDS drugs have been fast-tracked before the Phase II has been completed.
We ask again, Why hasn't Ampligen been fast-tracked?
[at the February meeting of the CFSCC, Dr. Cavaille-Coll stated mysteriously that the reason lies within Hemispherx' behavior. This is what he also told his superiors in the DHHS. That seems to satisfy them, but it does not satisfy us. If something is seriously wrong with the drug, why aren't those of us taking it being told? Conversely, if nothing is seriously wrong with the drug, why isn't it being fast-tracked? There is no excuse for waffling in this situation.]
Thank you again for your time and attention.
Mary M. Schweitzer, Ph.D.
The Association of CFS/M.E. Ampligen Recipients [this organization has not existed since 2000.]