Discussion in 'General ME/CFS News' started by Ember, Feb 21, 2013.
Patient christine saying "I didn't imagine spending my golden years on the couch". My sentiments exactly!
does Klimas have some treatments/repurposed drugs up her sleeve to start trialing immediately. kinda sounded that way in the meeting?
I had to miss the last four hours of that - looking forward to catching up with it when they post the video recording. I saw it as far as Peter Rowe's presentation and it all looked very positive but I was surprised not to hear questions or presentations from anyone in the drug industry, given that the purpose of the meeting was (I thought) to interest them in our disease. I know they were supposed to be sitting at home watching, but still...
Transcript of FDA Stakeholder Meeting on ME CFS
By Jocelyn | Published April 26, 2013
“This is a MS Word transcript of both days of the FDA stakeholder meeting. I have not attempted to clean it up besides removing the awkward paragraph breaks.”
You're right, we need to do this, it's a genuine opportunity. A lot of very good work has already been done in this area by people at PR, but more work is needed. The problem, I think, is resources because running PR is a huge task. If we as a community want it, we need more people to step up (even though that is really hard, I know...)
Yes, on a quick look (short on time now and for the next few days) there's both an opportunity here and a bit of a hump to get over at first, principally because you can't select ICC/CCC ME as a disease category - take a look at this old thread:
So if everyone piled onto Patients Like Me right now we'd have ME patients mixed in with CFS (who knows what definition) and we could be outnumbered 10:1 and be in the same old mess.
We'd need someone approaching PLM about that category who has some authority behind them (the new NAAME announced at the FDA meeting by Lily Chu? One of the big charities?) and trying to get a new disease category up. And then we need to check that the relevant symptoms are available (PEM/PENE?) and the therapies we're trying (MAF? Kutapressin? Ampligen? etc.).
I'm not familiar with it yet - want to have a look.
It needs some serious thought but also not for us to hang about forever.
Masses of work has been done on this project within PR in the past, but it has been on hold for a long time while we've been working hard on a range of basic organisational and day-to-day issues. We've been making great progress on those issues recently (and we'll be running a series of articles about that work soon), and I'm very hopeful that we'll be resuming progress on the projects soon. But Snowathlete is right, this is going to need volunteers to step forward from the community to get this done, and not just volunteers to work on the project, but volunteers to help out with the day to day work too: we'll be setting out the work that needs doing in one of the forthcoming articles.
That's great that PR is interested in this. Now that Bernard Munos (I thought he was great!) made this dramatic call for us to use Patients Like Me in front of such a big (internet) audience, I think we need some coordination quickly across organisations/advocates to stop someone just 'running off into the forest' with it and taking everyone down the wrong track - as would happen if people just went straight to that site now and started inputting their data, despite there being no appropriate disease category or a complete set of appropriate treatment and outcome options.
Up til now, advocacy has sometimes seemed to appear out of nowhere and it often hasn't been clear whether advocates have consulted with others before making calls to action. I hope this new NAAME group will help people to coordinate.
Is there any possibility of PR just communicating a bit with other advocates quickly now before someone gets off the wrong starting block and takes a load of people with them? Even if the necessary work can't begin yet? Just to get a bit of basic agreement on what needs doing and who might do it or how things might move forward in a sensible way.
I think a lot of us are very keen to volunteer for things but not able to lead. I'm too unreliable in terms of my health day-to-day (well, hour-to-hour) and too sick to take a leadership or organisational role but I can write a bit in odd fragments of the day which is how I manage to write now and again for PR. I'm sure a lot of other people are ready and willing to contribute in a similar way.
I'm very much looking forward to when the board are able to announce their plans and how the rest of us can help (and I know, of course, that the board are also sick and under a lot of pressure).
Just heard about this response from blogger Mary Schweitzer to the FDA meeting (haven't read it yet):
I wrote this comment on Mary's blog, but I thought I'd paste it here too.
As far as I can see, the primary issue here is this:
Clearly, some of the treatments that doctors/researchers have developed for CFS do work. Sometimes they work really impressively. Even the FDA said so, at the meeting a few months ago when they declined to approve Ampligen.
The problem is not that we don't have treatments that work. It's that we don't have treatments that work consistently enough that we can legitimately insist that government agencies in all good conscience MUST approve them.
If treatments only work a fraction of the time, and we can't predict when those times are going to be, then our case for getting people to do what we want is very very weak, no matter how good our rhetoric or lobbying skills are.
So what I would like to see is more stakeholders (researchers, doctors, patients and advocates) focusing attention on what it is that is causing these treatments to work some of the time but not all of the time. If we don't clearly start asking the question and trying to figure out the answer, we're never going to get anywhere -- or at least, so our 30-year history so far would suggest.
As a starting point, here is a new paper by Dr. Ritchie Shoemaker, on a treatment called VIP. (The fact that this is with a population of chronic mold illness patients is irrelevant here -- Dr. Paul Cheney now says that there is no difference clinically between "CIRS" patients from WDB's and his own CFS patients, and the other main CFS doctors have pretty much all acknowledged a mold connection to this disease too.)
One thing that Shoemaker makes clear in various places (though not in this paper itself) is that VIP only works when people are living in a non-moldy environment (e.g. with an ERMI test showing it is in the top 1/3 of all buildings). If people are in a moldy place, VIP doesn't help at all. So for this paper, he only used established patients who already were living in good environments -- those still living in bad buildings were not included.
Is it possible that the reason that -- say -- Ampligen works only some of the time but not all of the time is because it only works when people are living in reasonably decent places?
Could it be that this is also true with other treatments such as Rituximab or Valcyte?
If we don't know the answer for sure one way or the other, might it not be time to start asking?
From a personal experience point of view (e.g. the kind that Ampligen responders wanted the FDA to attend to), I've now tried a lot of treatments in various conditions (good environment vs. bad environment). And I believe very strongly that at least for me, all the things that are supposed to help with CFS actually do help. But at least for me, they only help when I'm in a decent place. A really bad environment trumps everything else.
Based on the increasing number of mold avoiders (dozens and dozens) who are reporting back to me on their own experiences, the same thing appears to be the case for other people with CFS. In a good environment, lots of things help people with CFS. In a bad environment, nothing helps.
Thus, I would like to see CFS researchers and clinicians start to use mold-related measurements (such as the ERMI test or C4a test) as independent variables in their studies.
If by chance we can prove that some treatments actually do work consistently, under the right environmental conditions, then we will be in much better position to go back to the FDA to try to get those treatments approved.
Yes Bernard Munos was inspiring, and tasked us with something pretty complex. We need to control for many other variables and comorbid conditions, it isn't an easy thing for us sick people to know how to do. Also, I was impressed wtih Dr. Vernon's presentation of the CAA, they are really using sophisticated techniques to come up with drug-repurposing solutions for us. I cannot wait until we get a chance to study her slides, could change the course of some of our treatments.
After I saw what the CAA did, I think that they may do a great job of creating a high-powered networking tool for us (with a third-party). If not, whoever does it needs the help of a professional to set it up IMO if the data on the back-end is going to be very meaningful.
Please don't dump on the CAA here as I don't care to debate the org, only to discuss their presentation - thanks!
Having just skimmed through the transcript, so admittedly unable to do it full justice, I still want to express my disgust. Since when are people with cognitive impairments, minimal energy and funds, going to organize and conduct their own basic research? Why should we, alone among all other sick people, be expected to do this? And if we were somehow miraculously able to do all this, then would some drug company stoop to take the final step, and reap all the profits? No doubt of it. I have never seen so many words and so little to show for them. Someone paid for this event and is paying for these people's salaries. I am appalled. No I am not going to do the job of those who are being well paid to do it and are responsible to do it. But it is clear these people will do nothing unless someone else does the hard work for them, while they will take the credit and the profit. After 20 years of this illness, this conference represents a new low in my experience. This is a sucker's pitch, in my estimation.
Nothing personal, but I disagree.
I'm afraid I don't buy that no patients have money when, for example, one sees the amounts some spend on speculative therapies. I previously argued it'd be great if patients gave 1% to research of what they spend trying to get better :
I think if more people with the illness and/or their family members donated or tried to raise money, a lot more could be raised money. A big problem is the small percentage of patients/families that do anything.
A lot of people find when they do fundraise that other people will donate. Fundraising these days can also involve online voting contests, nominating charities when buying with Amazon, etc.
We reap profits of our own if the drugs help our health, giving us a better quality of life and perhaps even helping us financially if they allow us to work.
Maybe some people think they can get better with non-pharmacological approaches. Me personally, I think I'm going to need pharmacological approaches. And I've already been housebound 18 years so prefer progress sooner rather than later (e.g. before I'm dead rather than afterwards).
Good points, Tom. Some people do have money, though a lot do not through not being able to work or work full time and paying for a lot of doctors and treatments, which may not even have helped. As for your next point, that we would be reaping the profit, interestingly this didn't even occur to me as my brain seems to be stuck. Also, what I know about myself is that I only get worse, and I do not believe that anything they come up with, if they ever do, is going to enable me to recover.
I'm not sure I will recover, but I feel there should be a reasonable chance something can help me improve.
I remember Dr. Weir saying he did a small trial (n=10) of alpha-interferon (I think it was) around two decades ago . One person recovered but the statistician said that wasn't significant. This sort of thing both frustrates me and shows to me that developing basic understanding of the condition is important (i.e. if it could be isolated what sort of person responds and who doesn't respond). There are lots of elements to making this happen of course.
We have a systemic problem which the usual simple mechanical research approach can't handle.
It's ok for us to disagree.
I think a lot of patients don't have money even for speculative therapies.
And those that do can spend quite a bit, but then some people do get some health back from that, so it's no surprise that people spend their money there rather than on research efforts, which wont bring results for them for maybe a decade.
Though I do think many people do donate quite a bit to research. When you look at research that does get done in ME/CFS there are almost always charities backing them and they get a lot of their funds from ME/CFS patients, and their families, who donate money.
I so think there is a big problem that the majority of the public are not interested in donating to our disease to anywhere like the same levels as say MS, even though it affects less people. Thats down to perception and it cant just be down to patients to fix that, though many are working very hard there, and have been for years.
I totally agree with us doing the maximum we can for fundraising by the way, I'm heavily involved with fundraising at the moment, as many people are now (more than there used to be I think) and it does speed things up and we are getting better at it, but research costs are astronomical, especially on sizable studies that have the most chance to change things for us.
I think governments have to do a lot more, not just push it back on the sick.
If people like us who have been sick long term ever do improve with some new treatment, I hope we give ourselves a party
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