Identifying potential treatments for ME/CFS - recovery trial model

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FMMM1

Senior Member (Voting Rights)
There was an interesting feature on clinical trials drawing on the experience from the UK Recovery Trial re coronavirus --- basically no budget --- just collect data on how people were treated and whether they improved --- within 100 days they had identified an old steroid which worked.
Seems like the sort of thing which might yield results for ME/CFS --- of course an European Reference Network/UK NHS specialist clinics would provide a mechanism to generate the data/possible treatments
https://www.bbc.co.uk/sounds/play/m0014p9j
starts about 19.5 minutes
@Michiel Tack
 
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I don't think that would work for ME/CFS. The Recovery trial was for treating acute Covid in hospitalised patients where they had thousands of patients all at the same time worldwide and could get the numbers quickly, and where they had some idea of what might work from the biology of acute infections. And they could get results quickly because it was a life or death result determined within days or weeks for each patient.

Chronic fluctuating diseases like ME/CFS where so little is known about the biology is completely different. You would have to test thousands of drugs more or less at random and follow up patients for several years. That would be prohibitively expensive and unethical.
 
Arnie Pye said:
Was that betamethasone or dexamethasone? I'm sure I remember seeing mentions of one or both of those.
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Yea it was dexamethasone (from memory). However, as you're aware, I'm really thinking of the analogy to identifying treatments for ME/CFS.
 
Trish said:
Chronic fluctuating diseases like ME/CFS where so little is known about the biology is completely different.
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Agree completely; however, rituximan was identified by Doctors treating people for something different. So collating data from treatments, administered to people with ME/CFS, is a strategy. However, yes - I agree, others have said that it's difficult to see a way into i.e. to treat ME/CFS - which is why they identified GWAS as a strategy.

@Simon M perhaps the data collected from those donating samples (and controls) could include any drugs or other treatments which helped?
@Michiel Tack

Trish said:
You would have to test thousands of drugs more or less at random and follow up patients for several years. That would be prohibitively expensive and unethical.
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Yes, I agree, I'm think of serendipitous findings - like rituximab.
 
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FMMM1 said:
Yes, I agree, I'm think of serendipitous findings - like rituximab.
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So not really like the recovery trial model, but straightforward standard trial methodology that takes years to go through the stages of getting approval, getting funding and recruiting patients. Yes, Rituximab was serendipidous, and was worth testing, but turned out not to be effective once fully tested. It could take centuries if we wait for serendipidy to turn up a drug that works for ME/CFS, with hundreds of failed trials along the way.
There are other drugs being tried for ME/CFS that seem to get some enthusiastic support from patients and their doctors, like Ampligen, Low Dose Naltrexone, Low Dose Aripiprazole. Each will need to be tested in proper clinical trials that run for at least 2 years follow up. That all costs money and requires clinicians with the will to do them.

My point was that whatever is tested for ME/CFS has to be done properly over years with significant funding. There are no short cuts like there is for acute pandemic infections when there is a captive patient set, funding and apporoval easy to obtain, knowledge about infections, and rapid outcome measure of life or death.
 
Trish said:
So not really like the recovery trial model, but straightforward standard trial methodology that takes years to go through the stages of getting approval, getting funding and recruiting patients.
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I think we're thinking of slightly different things here.
I'm part of another group which has had a limited discussion with European Federation of Pharmaceutical Industries and Associations*. One of the issues is identifying potential treatments - others on Science 4 ME have pointed out that it's difficult to see a way in - to understand/treat ME/CFS. So this is just some brain storming to see if it could generate some ideas/candidate drugs/treatments.

*"European Federation of Pharmaceutical Industries and Associations is a Brussels-based trade association and lobbying organisation, founded in 1978 and representing the research-based pharmaceutical industry operating in Europe."
@Michiel Tack

Trish said:
Yes, Rituximab was serendipidous, and was worth testing, but turned out not to be effective once fully tested.
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I would agree i.e. my understanding is that rituximab didn't work. My thoughts were more about identifying options e.g. if there was a way to collate potential treatments --- rituximab was a cancer drug used to treat someone with ME/CFS for cancer ---

Trish said:
It could take centuries if we wait for serendipidy to turn up a drug that works for ME/CFS, with hundreds of failed trials along the way.
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Yea but in a sense there may have been countless mini-trials. Like the person who had cancer and was treated with rituximab and appeared to improve. The other thing to bear in mind is that the GWAS study might help to provide some evidence of potential treatments - triangulation. So this is about identifying options now --- yes they have to be tested.

Trish said:
There are other drugs being tried for ME/CFS that seem to get some enthusiastic support from patients and their doctors, like Ampligen, Low Dose Naltrexone, Low Dose Aripiprazole. Each will need to be tested in proper clinical trials that run for at least 2 years follow up. That all costs money and requires clinicians with the will to do them.
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Yea finding interested clinicians is a challenge; however, this is all interwoven e.g. one of the issues is the difficulty in seeing a way in (treat/investigate cause of ME/CFS) and the GWAS study might help to provide that.

Trish said:
My point was that whatever is tested for ME/CFS has to be done properly over years with significant funding. There are no short cuts like there is for acute pandemic infections when there is a captive patient set, funding and apporoval easy to obtain, knowledge about infections, and rapid outcome measure of life or death.
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As above, this is about identifying candidates (drugs/other treatments) but yes, even if an off-label drug was identified then it would take time/money to do the trials - think rituximab.

Hope some of this clarifies.

@Simon M - references to GWAS.
 
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