Invest in ME 2016 research conference summary

Sasha

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Invest in ME research conference 2016

Personal summary and reflections from Dr Charles Shepherd

Full report - which is quite detailed at over 6,000 words - available on the MEA website:

http://www.meassociation.org.uk/201...y-for-sharing-ideas-on-research-20-june-2016/
Thanks so much - looking forward to reading it!

BTW, I don't know if you'd consider putting up a PDF of the text of the report? A lot of us struggle with reading long things online and I always like to print off things like this to read them. (If anyone wants to do that straight away it's easy to cut and paste the text into a Word doc - possibly cutting and pasting into Notepad first, to strip out the formatting.)
 

AndyPR

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Marky90

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Thanks CS, especially liked the information from UCL.

One thing though: Why should it be hard to make a protocol for a rtx-study, in light of Haukeland already having one?
 

Marky90

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Also I don`t see the ethical concerns, there are way more adverse effects reported from CBT/GET-trials, and they don`t seem to have a problem supporting those anyway. With rtx, the worsenings in the study have been transient, and other adverse effects (e.g pneumonia) have been treatable.
 
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Thanks CS, especially liked the information from UCL.

One thing though: Why should it be hard to make a protocol for a rtx-study, in light of Haukeland already having one?
Maybe I should comment here. Dr Cambridge has never been directly involved in the setting up of a trial, only in background research. Speakers at the IiME meeting were often asked questions that had nothing to do with their talks at all. There was a question to Dr Cambridge about setting up trials and she did her best to give her thoughts on the difficulties but only because she felt she ought to produce some sort of answer. She was not speaking for those involved in trial planning.

In relation to Marky90's question we need to distinguish a treatment protocol from a trial design. Haukeland have devised a treatment protocol. It would be sensible to use the same protocol in other studies until we know more, but that has nothing to do with designing a trial that will answer a question. The size of trial that there is a budget for in the UK would not provide statistically powerful evidence just for efficacy over and above the Norwegian phase 3 study. It was always assumed that a UK trial would address a different scientific question - perhaps about markers of response or dose ranging - which could usefully be assessed in a relatively small trial.

Dr Cambridge is right to point out that it is essential to have a dedicated principle investigator with a team that understands both ME/CFS and rituximab. Trials run by subcontractors are not a good idea. At present it is not clear that there is a suitable PI in the UK to take a trial forward.

Although Fane Mensah has been working hard at detailing B cell subsets in ME/CFS we do not yet have a replicated marker that could be used look for correlation with response. That means that it is not clear what scientific question a UK trial would answer. I appreciate the enthusiasm for a UK trial but we cannot treat people with a drug with potential harmful effects without justifying that with a clear scientific objective. Drs Fluge and Mella would not wish that. Discussions continue but my own view is that setting up a trial in the UK should wait until either we have new findings that provide a clear direction or until we have a positive outcome from Norway phase 3, which would justify a small dose ranging study. Above all we need some handle on a B cell abnormality that we can follow so that we know what is going on.

As people will realise, I have been through this mill once before (more than once in fact) and it is far from simple. It is not a question of if things go wrong but when, and how best to pick up pieces when they do, to ensure the best outcome. That does not mean getting cold feet, it just means being patient about when is the right time to move forward. Pharmaceutical support can be helpful but only if the science has been well sorted out in advance. Company trials can distort design in ways that are not in the patients' interests. The Norwegians have done everything by the book and all credit to them for getting this far. I do not think we should be muddying the waters with underpowered or badly controlled studies done in haste.

An important part of exploring use of rituximab in other countries is to get a feel for clinical response outside a trial setting, which is a legitimate objective at this stage. Quite a large number of people with ME/CFS have now been treated in the USA, Germany and Norway at least and it would be useful to have published reports from these centres. Unfortunately at present we only have hearsay.
 

Marky90

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Thanks for your thoughtful and insightful reply JE, i agree with the points you made.
You might be right about it being better to wait for the phase 3 trial to be published. An argument against that might be that it will take longer to replicate, or not, their findings in another country. However it also makes sense to wait for the phase 3 trial to show if the findings can be replicated within a country in different hospitals.
 

Kati

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Thanks for your thoughtful and insightful reply JE, i agree with the points you made.
You might be right about it being better to wait for the phase 3 trial to be published. An argument against that might be that it will take longer to replicate, or not, their findings in another country. However it also makes sense to wait for the phase 3 trial to show if the findings can be replicated within a country in different hospitals.
I also want to thank @Jonathan Edwards for his thoughts. I am not from the UK, but in Canada which is still at a very dark ages when it comes to ME and where there seems to be a picking up of the psychiatry influence these days.

It is so very hard to wait and be patient, when the quality of life drops and when the so called authorities are making it even hrder to access treatment.

Patients need hope and transparancy as of what exactly is happening. We need to know 'something' s happening.
 

Bob

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For future reference, an easy way to convert a webpage to a pdf document, to either save it or print it, is to use one of the websites below. They can produce a very neat copy. I sometimes experiment with each of them each time I want to save/print a webpage, in order to get the best formatted copy, because the results can sometimes be a bit random.

They're exceptionally easy to use - you just copy and paste the url of the website you want to convert to PDF...

http://webpagetopdf.com/
http://www.printfriendly.com/
http://www.printwhatyoulike.com/
http://pdfmyurl.com/
 
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Maybe I should comment here. Dr Cambridge has never been directly involved in the setting up of a trial, only in background research. Speakers at the IiME meeting were often asked questions that had nothing to do with their talks at all. There was a question to Dr Cambridge about setting up trials and she did her best to give her thoughts on the difficulties but only because she felt she ought to produce some sort of answer. She was not speaking for those involved in trial planning.

In relation to Marky90's question we need to distinguish a treatment protocol from a trial design. Haukeland have devised a treatment protocol. It would be sensible to use the same protocol in other studies until we know more, but that has nothing to do with designing a trial that will answer a question. The size of trial that there is a budget for in the UK would not provide statistically powerful evidence just for efficacy over and above the Norwegian phase 3 study. It was always assumed that a UK trial would address a different scientific question - perhaps about markers of response or dose ranging - which could usefully be assessed in a relatively small trial.

Dr Cambridge is right to point out that it is essential to have a dedicated principle investigator with a team that understands both ME/CFS and rituximab. Trials run by subcontractors are not a good idea. At present it is not clear that there is a suitable PI in the UK to take a trial forward.

Although Fane Mensah has been working hard at detailing B cell subsets in ME/CFS we do not yet have a replicated marker that could be used look for correlation with response. That means that it is not clear what scientific question a UK trial would answer. I appreciate the enthusiasm for a UK trial but we cannot treat people with a drug with potential harmful effects without justifying that with a clear scientific objective. Drs Fluge and Mella would not wish that. Discussions continue but my own view is that setting up a trial in the UK should wait until either we have new findings that provide a clear direction or until we have a positive outcome from Norway phase 3, which would justify a small dose ranging study. Above all we need some handle on a B cell abnormality that we can follow so that we know what is going on.

As people will realise, I have been through this mill once before (more than once in fact) and it is far from simple. It is not a question of if things go wrong but when, and how best to pick up pieces when they do, to ensure the best outcome. That does not mean getting cold feet, it just means being patient about when is the right time to move forward. Pharmaceutical support can be helpful but only if the science has been well sorted out in advance. Company trials can distort design in ways that are not in the patients' interests. The Norwegians have done everything by the book and all credit to them for getting this far. I do not think we should be muddying the waters with underpowered or badly controlled studies done in haste.

An important part of exploring use of rituximab in other countries is to get a feel for clinical response outside a trial setting, which is a legitimate objective at this stage. Quite a large number of people with ME/CFS have now been treated in the USA, Germany and Norway at least and it would be useful to have published reports from these centres. Unfortunately at present we only have hearsay.
Thanks Jonathan for providing this additional and very helpful information about what is happening with regard to proposals to set up a clinical trial of rituximab here in the UK.