“There’s an unmet medical need right here and there has been for a long time” Bob Miller
The first day of the FDA Stakeholders Meeting was like an patient/FDA roundtable session focusing on what symptoms are worst and what treatments worked best. It’s not clear how much ‘actionable’ information the FDA got from this session; they really need a survey or study to get at that, but you couldn’t be in the room without getting a real sense of what chronic fatigue syndrome is like. As always ME/CFS patients provided poignant and often startling testimony about the difficulties this disease presents.
Five patients/advocates started off the meeting. There was an ex-emergency room doctor who was able to improve enough to work full time but still must sacrifice quality time with his family on the weekends to work on recovering from the week. There was Denise with her two formerly athletic young men, who, eight years after becoming ill now require 24/7 care despite trying a long, long list of pharmaceuticals. (Their heart rate at rest is over 110 bpm.) There was Mary Dimmock talking about her now mostly housebound son who not too long ago was backpacking through Asia – by himself. There was the linguist in the seventh year of his two year Master’s program who had trouble speaking and was too cognitively challenged to even consider a career in his field.
“I have a very small life” quotation from CFIDS Association Survey
There was outgoing CFIDS Association CEO, Kim McCleary, reporting that the CAA found that the average illness duration of the people taking their FDA survey was no less than 18 years and one person has had it for 70. The average age of onset is between 19-35, with 36-50 coming in a close second and 18 years or younger a strong third and then past 51 years of age. (Dr. Jason reported that the Chu/Jason FDA survey had really significant results; watch for a report on that tomorrow. )
There was Chris Williams, a former federal employee who’d managed 70 people, who now after early retirement, has to remind her husband that even the mundane tasks of daily life overwhelm her ability to multi-task.
In short, there was chronic fatigue syndrome in all it’s horrible glory…
“My day is structured around this illness”…quotation from CFIDS Association FDA Survey
The FDA wanted to know what crashes were like. One person described suddenly blacking out crossing a busy street in New York City. Bob Miller described a seemingly patternless disorder where one day he can do something that the next day will land him in bed. One person described a two year crash following an attack of pneumonia, and then after pushing too hard shopping, atrial fibrillation that left her in the emergency room. Another person described five days spent in bed for expending 45 minutes too much energy socializing. Another’s limbs started flailing around. One person made it clear that she was really never out of a crash; that she always had symptoms, and for her the question was whether they were severe enough to stop her.
It became clear that ‘crashing’ is a very variable thing; some people crash for long periods and some for short periods; small things can make some people crash while others are more resilient.
When asked what helped, Mary Dimmock described a long list of medications that didn’t help her formerly adventurous son, some that helped a bit (florinef and antifungals) and antibiotics (azithromycin/doxycycline) that worked great – for about six weeks – after which her son was back in bed again.
Another person noted that after coming down with ME/CFS in Angola, she’d been treated in three continents, had recovered using antibiotics only to fall ill again – in the worst of places – the UK, where she found no relief, but later in the US got enough help from antivirals, B-12 and pacing to be able to work almost full-time. She railed against the behavioral mindset that pervades the health service in the UK.
Ampligen – When FDA reps asked for patients experiences on Ampligen one person person who is now starting two practices said she couldn’t have even made to the meeting without Ampligen. Another person described, after being wiped out by a treadmill, dancing out of the doctors office following an Ampligen infusion. Bob Miller listed a long, long string of antivirals and other drug failures that ended when he started Ampligen. He, too was able to come to this meeting because of Ampligen. (Dr. Lapp will later describe his 20 years of successful Ampligen infusions for ME/CFS patients.)
Vistide – another patient spoke of her success with Vistide with Dr. Peterson.
Mary Dimmock warned the FDA not to forget that most people don’t have the money or sufficient insurance coverage to be able to afford many of the treatments reported and one person reported spending $2,500/month on treatments. Another wondered if some of the extensive and expensive treatment regimes some people were on were working at all.
“I have a life of lowering expectations” quotation from the CFIDS Association FDA Survey
One person did well on low-dose naltrexone while another reported getting some benefit from Cymbalta, only to temporarily lose his vision on it, and then get kicked off it anyway once his insurance coverage changed and he was only able to afford generics. Jennie Spotila spoke of trying what sounded like dozens of drugs for pain, some of which worked for periods of time, in what appeared to be an exhausting search for relief. Others pointed out the difficulties of managing the side effects of pain drugs, particularly in a disorder with gastrointestinal issues, which lead one person to say that 22 years of pain drugs was not a good thing at all.
Anita Patton and Bob Miller and others asked the FDA to do whatever it took to facilitate drug development in this disorder. After asking for raise of hands Anita Patton found that some pharma reps were present but not many. Dr. Mikovits laid out what appeared to be a good strategy for drug development in this disorder, one part of which included extensive gene expression and immune studies for the thirty percent in the Fluge/Mella trial that responded to Rituximab.
Dr. Peterson talked about 25 years of ‘therapeutic stagnation’ and noted that a consortium of ME/CFS physicians was ready and willing to do Phase I, II and III clinical trials for ME/CFS. The FDA will be looking for ‘endpoints’ and Dr. Peterson pointed out that ME/CFS physicians are already using biological measures such as VO2 max exercise tests, NK cell functioning, MRI and SPECT scans to inform their therapeutic approaches. Focusing on symptomatic relief will never return a patient to health, he asserted, but he believed immune-based therapies could.
Dr. Lapp reported that in his 20 years of Ampligen use no less than 50% of patients responded significantly and 30% responded very well with many returning to work. Despite the FDA’s huge (and recent) concerns about Ampligen’s safety, a chart review indicated no serious side effects had been reported in his Ampligen patients and he wondered how many FDA approved drugs could match that track record. He also noted that he thought that the clinical trials used the wrong end points and that he was having much more success than the trials indicated.
Dr. Baraniuk, a researcher and physician, took his time to point out some remarkable results from his latest studies. Announcing that not all is doom and gloom, he stated we will soon be able to throw away all the subjective diagnostic criteria because we’re finding objective criteria that will break this disorder up into separate diseases, each of which will end up in Harrison’s Textbook of Medicine before too long.
He then pointed to some remarkable findings from his lab; three different dimensions of exercise induced MRI changes in Gulf War Syndrome patients which he expected to be replicated in ME/CFS. (All the GWS patients met the criteria for CFS). They involved white matter changes in one set of patients and two different types of brain blood flows in response to exercise in other patients.
A report on Dr. Baraniuks latest study is coming up shortly.
Tomorrow is the big day at the FDA Stakeholders Meeting with presentations from ME/CFS physicians, a talk on drug repurposing from Dr. Vernon of the CFIDS Association, the first findings from the CDC multi-center ME/CFS physician study, round-tables, talks on endpoints, etc.
- Check out the agenda here.
- Watch the meeting here; anyone, it turns out – registered or not – can watch.
Tell us how your coronavirus vaccination went and find out how other people with ME/CFS and/or FM fared with their coronavirus vaccination in Health Rising’s Coronavirus Vaccine Side Effects Poll.