FDA Workshop on Drug Development for CFS and ME

Great article by Mary! Thanks, Sasha.

Mary closes, “The larger question, of course - the elephant in the room - has yet to be addressed by anyone in the government: WHERE IS THE SENSE OF URGENCY? What is it going to take to hear that word spoken by someone in a position to actually change things?”

Dr. Klimas gave her opinion during the workshop: “The best way for drug development to leap forward right now, I'm going to be bold and crazy and say right out front, we need philanthropic support....”

I don't think Dr Munos was suggesting we do our own research, just that we provide our data. I think that he has done us a big favour by highlighting an area that could pay off big if patients did the bit that they can do and others do the rest.

I don't think it's beyond a working party of the more functional patients to agree on what we want from Patients Like Me (an ICC/CCC ME disease category; the full range of our symptoms, including PENE; the full range of our therapies, including MAF and Ampligen), to influence PLM to implement those changes, and at that point as many patients as possible can go to that site and input their data.

As I understand it, it's at that point that industry can go in and mine the data. No-one has to conduct a research study for them - although if that were needed, our charities could commission it very cheaply because there'd only be the data analysis to do, not the data collection which is the big cost.

Does anybody knows what anti-inflamatory they were talking about??? I know they are testing one on Dr Elander but I heard the reference again in the meeting but there is no name in transcript.

Yes.

I don't think it has to be either/or - I was only suggesting 1% of what people spend on treatments. That 1% wouldn't even have to come from the treatment pot as it were. People can still spend money but invest a small amount (1%) of money in bringing the field forward, giving a better idea what might work/not work (rather than treatments often being a shot in the dark), increasing the chances treatments will be covered by public health systems or insurance companies, increasing the chances drug companies will invest money in trials, etc.

A decade may or may not be a long time. Possibly if people have been fed the idea that lots of people get better in a few years, a decade may seem like a long time. The reality I think is that if people have been ill and diagnosed a couple of years, and are not young people/children, there's a very good chance they'll still be ill in ten years.

Outside the UK and the US and perhaps a few other countries, I'm not sure this is the case. Lots of countries don't have research funds for the illness.

And even in the best countries, it may only be 1% of people estimated to be affected in any one year.

From seeing what happens in Ireland, a lot of people seem to get to a level and forget about the ME cause in lots of forms.

My experience is that a lot of the public will donate a little, and people who know people affected will often donate more.

I'm not sure if many outside the UK and a few in other countries have been working that hard to raise money for research.

Great.

Well, who is going to come up with money? Outside the US, the amount spent on most individual illnesses by government research bodies isn't "astronomical". I think some people can think that, because in many countries, it's the government's responsibility to pay for healthcare, it's also their responsibility to pay for all the research for illnesses. But this is not realistic: governments have rather limited pots of money for research to be spread over maybe 100-200 (?) common conditions and thousands of less common conditions.

A fair amount for governments (outside the US) to put in wouldn't be an "astronomical" amount.

The "Let's do it for ME" fundraising for Invest in ME in the last couple of years is something I'd like to see repeated a lot more around the world. Lots of people raising two and three figure amounts. Such small scale fundraising doesn't happen enough in my mind.

Many ME/CFS patients struggle to survive and have suffered cutbacks in pay since the introduction of government austerity since 2008. Waiting a decade or 2 decades for some cure may sound attractive to some, but the destruction of lives in the process is not acceptable. Some may be comfortable enough to sit it out for a decade or two but most cannot. Certainly, patients giving some funds to research is important but there are limitations to what this can achieve in these times of austerity.

Existing research papers have been forgotten about in the rush to keep researching and funding research. The new drive for translational research - that is turning research into diagnostic and treatment outcomes is a great new development. They have given us biomarkers which we can work on ( www.cfs-ireland.com/structure.htm#7 )
. And despite all the negativity, despair, and doom and gloom, many ME/CFS patients have recovered from proper medical treatments. The move towards new types of clinics providing biological based diagnosis and treatments needs to be continued and more fully supported. It is the only alternative to Wessely's psychiatric clinics or dying slowly of medical neglect waiting for miracle cures.

I never said it was attractive to anyone.

I said I didn't find an excuse that people won't donate or try to raise some money for research because it wouldn't have an immediate effect, convincing. A lot of important things involve delayed gratification as it were.

Huge numbers more weren't donating and/or raising money for research during non-austerity times, as it were. And austerity also affects the amounts of money governments are likely to be willing to spend also.

Compared to the CFSAC meetings, I felt that this FDA meeting was so much more respectful to our patients. It seemed to me that there was a genuine interest in assembling information as a start in order to see what the needs are.

I did not expect miracles in this one initial meeting. I loved the fact that there were question and answer opportunities for patients, advocates, panel and staff.

Unlike the CFSAC which almost seems robotic and even harsh with the patient representatives, I felt that the FDA here bent backwards to accommodate us.

We need a movement towards progress. It will never be "perfect". We can keep on harping on all the problems..ie. the name, the definition, which biomarkers to use etc.

It is true that we have been ignored for so long. It is true that there has been minimal funding from the government and from private fundraising. It is true that few real large scale studies have been done.

But, we are where we are. The choice is how to go forward. Every step forward is in the right direction. Steps need to be taken to amass data. The data that is already out there needs to be merged into one central location and then refined. We need more advocates and activists for raising awareness and for fundraising. Every little step taken helps.

I think that someone speaking at the conference mentioned the fact that the current specialists in the field need to be working together. They need to combine all their knowledge and experience for the benefit of the patients. I'm not sure how that could be achieved but, if it can, that will be a huge step.

I think that because so many of us have been suffering a very long time and have been mistreated by the medical establishment, we have become very cynical and that cynicism will not serve us right.

It is true I'm sure that all this will take time and especially for us to see real results. Some of you have mentioned that it will be too late for us. I still have hope that we will be proven wrong but, nevertheless, we can still leave a legacy for the next generation of sufferers.

Our work will not be in vain.

In Ireland





Delayed gratification is not an issue for most ME/CFS patients as most cannot work and they live in poverty and misery. There is no gratification in misery. Austerity has meant cutbacks in income for disabled people who are already on low income.
As regards "attractive", well ME/CFS patients differ substantially in personal circumstances. Some can afford to wait a decade or two, while others cannot. The existence of an Irish ME/CFS clinic using the best diagnostic and treatment technologies and translational research could serve as a beacon of hope for the world. And lead the field. Many presently ill people could be given at least an opportunity to recover and resume their studies in University or training institutes and resume their careers.
Of course there is opposition to us, obviously Wessely and company and surprisingly some ME patients are against us, and they claim we cannot do this. Meanwhile ME patients exist without hope, medical support and die of heart attacks, cancers, suicides.

I imagine it would take a good while for the clinic you have envisaged to get approved/funded*, built and get up and running particularly, as you keep point out, this is a time of austerity.

Also, research progress doesn't have to take a decade or two.

*Not an insignificant sum:

WELL, AT LEAST THE FOOD WAS GOOD

By Mindy Kitei
Sunday, April 28, 2013

I just wanted to add my appreciation t the patients who with great sacrifice attended the meeting to represent us. They are the true heroes.

They might have had something like this in mind:

Thank you Ember. I did not realize that the Open Institute had so many experts on it's list. I did notice that Paul Cheney and Derek Enlander were missing from the list.
I took a quick look at their website: http://openmedicineinstitute.org/research-initiatives/mecfs-merit/
Their list of projects are pretty impressive!

Yes, it's an impressive list. It's unclear how many if any will happen due to a lack of money.

Well if you never start, then you will never have a clinic. You have to start somewhere. Anything worthwhile ever achieved began small and took time but it had to begin. And of course they had their critics and naysayers.

How much does it cost to keep people sick ? It costs billions per year and this is included on web site. it costs more to keep people sick than help them recover, but this is never discussed or analysed in any depth. Even supposedly intelligent people neglect to mention this, analyse it.

Its strange how there is plenty of money to bail out bankers and help them keep their high salaries and bonuses, but when it comes to peoples' health, and particularly the very ill, it is (falsely) claimed to be too expensive. Tom it cost 80 billion euros to bail out corrupt banks and bankers and the sheep (the people) dare not say this is too expensive or disagree with it.

The webcasts are available now on the FDA site: http://www.tvworldwide.com/events/fda/130425/

And that's my attitude to raising money for research.

CFS Patient Advocate's response to the meeting on his blog (haven't read it yet but he's always good value):

http://cfspatientadvocate.blogspot.co.uk/2013/04/fish-or-cut-bait_29.html

Chris Cairns highlights:

CFS Patient Advocate
MONDAY, APRIL 29, 2013
Fish or Cut Bait

Regarding the April 25-26th FDA meeting, this much can be said. There is the real world - and then there is the world of the government. These two worlds do not intersect at any point.
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By the way, the FDA should fish or cut bait. After this two-day FDA conference on April 25th and 26th, the FDA can no longer plead ignorance about the true nature of this illness. They have an opportunity now to "send a signal". They should start with a significant move to get Ampligen approved for this patient population. What has happened with Ampligen is a first-rate travesty.

The FDA workshop closed with the awkward statement, “I want to thank you again for your inquisitiveness, commitment, your willingness to ask questions, your willingness to challenge each other and wish you a safe travels and a wonderful rejuvenating weekend.” (Ouch!)

We all knew that it would take the patient representatives more than a weekend to recover and that the experience wouldn't be wonderful. Jennie Spotila has now posted a video of herself: “Crash Day 3 After FDA Meeting:”