My name is Jeannette B. I am relatively new to the advocacy world and I don’t have all the Washington insider lingo down yet. So, instead of “FDA” or “NIH,” I will probably say “the FDA” or “the NIH.” And since I am new here, let me just quickly explain that I personally cannot bring myself to use the demeaning, dismissive and ridiculing name “CFS” or even worse, “chronic fatigue,” for a disease that robs patients of virtually everything that makes life worth living
There are three reasons for the fact that I am able to be here today and that I am able to be play any role at all in Aimee’s life, my almost 3-year old daughter: 1. Ampligen. 2. My husband, Ed, who insisted that I give Ampligen a try when I was ready to give up. 3. Dr. Dan Peterson.
There are two people in this room whom I could not feel more grateful towards and they are Alaine Perry and Dr. Gailen Marshall. Ms. Perry and Dr. Marshall, you voted “yes” on all four questions regarding the recommendation for the approval of Ampligen during the FDA advisory committee meeting on December 20 of last year. The committee opined that the drug is safe. You were part of a minority on the committee that also found that the drug is effective. Many of those on the committee who weren’t convinced of its efficacy—after a very antagonistic FDA presentation that, among many other things, counted symptoms of the disease and even a pre-existing case of cancer, as side effects, that faulted Ampligen for not being a cure (unlike diabetes, blood pressure, thyroid and other meds, I guess) and that denied the sponsor the opportunity for a rebuttal—felt that there is something there that is worth looking into further. Why are some patients responding to Ampligen and others are not, something that, by the way, can be said for many, if not all, drugs? Clearly, what we have here is a sub-group of patients and I am blessed to be a model responder. Why is nobody interested in digging deeper into this potentially game-changing break and finding out why I respond and a friend of mine does not? All this talk about the government’s commitment to making progress for ME: show us that you mean it! We have a huge piece of the puzzle right in front of us and that piece is Ampligen. Let’s run the course with it. It’s not like there are all these other exciting drugs waiting in the wings that Ampligen would take resources away from.
Are we really going to watch a safe drug that is extremely effective for a sub-group of patients go away forever? We are talking about a drug for a disease that has no other treatment and none on the horizon, for a disease that is gravely disabling. Could this possibly make sense to anybody? I mean really! Anybody?
Is the sponsor of Ampligen, Hemispherx, the perfect drug company, whatever that means? Maybe not. But does the drug work? Absolutely, for a maybe significant sub-group. And is it safe? You bet! Even the FDA’s own advisory committee said so. So, for crying out loud, FDA, work with the sponsor to get the efficacy information that you wish to see. And since the sponsor doesn’t have the financial resources for a new trial, let’s get the NIH to help finance that trial that will produce that information. Because if you don’t, future suicides are all but guaranteed!
I attended a very impressive FDA Drug Development Meeting for ME last month and I want to express my sincere gratitude to RADM Kweder and her team for a job very well done. But what we need now are concrete results and some tangible progress. I do feel like we made some ever-so-slight progress at the April meeting. The avalanche of epiphany moments being had by the FDA officials trying to take in the patients’ description of the suffering and the symptoms was oddly shocking and gratifying at the same time.
As an Ampligen patient with astonishing results on the drug, it was quite bittersweet to hear the clinical team leader of the FDA division responsible for not approving Ampligen in February of this year, Dr. Theresa Michele, admit, and stress, that she had not previously been aware of the symptoms of ME as clearly as she was as a result of listening to patients’ testimony at the April meeting. I cannot help but wonder if the decision not to approve Ampligen might have differed had there been a clearer understanding of the seriousness of the disease by the FDA before the decision was made. Given that Ampligen is in grave danger of disappearing as a direct result of the denied FDA approval and the resulting very real risk of the sponsor’s bankruptcy, a predicament the FDA is well aware of, it’s almost intolerable to think that thousands or maybe hundreds of thousands of patients could be helped by an existing safe and effective drug had there just been more understanding of the disease.