Open Norway: Plasma cell aimed treatment with daratumumab in ME/CFS (ResetME) - Haukeland University Hospital

[BrightCandle]

The response to my email on the issues I found were dismissive. They are going to keep pushing this garbage through and its going to be used to hurt people again. The MEA once again blessing harm for patients while refusing to acknowledge the sheer scale of the problem nor the levels of suffering and death this disease is causing.

 

[ryanc97]

Yes, people that think of donating to a phase 2/3 study as being "you get treatment vs I get treatment" probably won't be donating, but I have hope that there are enough ME/CFS patients that are sensible enough to understand that this is not what is occuring and that understand that the point of such a study is to deliver answers that don't exist without it.

Well, think of it as risk transfer. If you donate you are transferring risk to a patient who is willing to risk treatment and able to do so by participating in the trial, but the patient is getting the treatment FoC. Whereas if you are a patient willing to risk treatment but unable to participate in the study because you are not in Norway...

 

[ryanc97]

The response to my email on the issues I found were dismissive. They are going to keep pushing this garbage through and its going to be used to hurt people again. The MEA once again blessing harm for patients while refusing to acknowledge the sheer scale of the problem nor the levels of suffering and death this disease is causing.

Could it be the people running MEA (not sure how they got there in the first place) are using it as a grift to earn money? I don't know the history of MEA, but unless it was ran by people with actual ME, you would risk having people that are self-serving.

 

[forestglip]

Well, think of it as risk transfer. If you donate you are transferring risk to a patient who is willing to risk treatment and able to do so by participating in the trial, but the patient is getting the treatment FoC. Whereas if you are a patient willing to risk treatment but unable to participate in the study because you are not in Norway...

The risk may or may not be the same. But the benefit is exponentially higher.

If a patient takes it themselves, it might help that one patient and maybe a couple more that are convinced by an anecdote.

If a patient takes it as part of a trial, it could help millions of patients get that treatment. And maybe improve understanding to get better treatments.

 

[EndME]

Well, think of it as risk transfer. If you donate you are transferring risk to a patient who is willing to risk treatment and able to do so by participating in the trial, but the patient is getting the treatment FoC. Whereas if you are a patient willing to risk treatment but unable to participate in the study because you are not in Norway...

The risk are unlikely to be the same and maybe not even the benefits for the individual even if the treatment eventually turns out to be effective.

For a given individual the risk of an off-label attempt is likely much higher than the risk of participation in the trial for the reasons already discussed. The relationship between these different risks need not be linear and straightforward because these things are always contextual. And of course the benefits for the individual also needn’t map from off-label attempt to trial nicely, if contextual information becomes available as part of the trial (for example changes in dosage protocols or other unforeseen events that can occur as part of trials which the off-label attempt person needn't have access to).

 

[Arfmeister]

In my opinion, off-label case studies (n=1 trials) have their merit, as long as they’re properly documented and monitored by a Doctor Who knows ME and how the medicine works - in this case Dara - and what to monitor.

Sadly, not all specialists do know what they’re doing, do meticulous monitoring or write down proper case studies.

But 10 case studies give us a lot of information and in medical history this is often what has led to starting a pilot study.
- also it fills up the gap between today and appr. 3 years when we have the results from Phase 2 Norway.

 

[Utsikt]

In my opinion, off-label case studies (n=1 trials) have their merit, as long as they’re properly documented and monitored by a Doctor Who knows ME and how the medicine works - in this case Dara - and what to monitor.

Sadly, not all specialists do know what they’re doing, do meticulous monitoring or write down proper case studies.

But 10 case studies give us a lot of information and in medical history this is often what has led to starting a pilot study.
- also it fills up the gap between today and appr. 3 years when we have the results from Phase 2 Norway.

We already have a phase 1 study that’s essentially 10 consecutive case studies. There is no need for anyone to do more of them, and F&M are already planning a phase 2.

We know how difficult it is to do proper studies on ME/CFS, and @Jonathan Edwards can testify about how difficult it is to do studies with -mabs in general.

The chance of gaining data or any value with off-label treatments is minuscule.

 

[OrganicChilli]

It’s the Norwegian ME Association with 7k members.

I think a plea in English might be better - the English literacy is decent in Norway. I can help finding all of the email adresses if anyone needs them.

@Utsikt I've emailed them using the contact button on the fundraiser's homepage, but haven't heard back yet. Do you mind sharing the relevant email addresses so I can follow up next week?

 

[Utsikt]

@Utsikt I've emailed them using the contact button on the fundraiser's homepage, but haven't heard back yet. Do you mind sharing the relevant email addresses so I can follow up next week?

Leader of the ME-fund: Bjørn K Getz Wold – bkw@ssb.no

GenSec NMEA: gensek@me-foreningen.no

Communications NMEA: Kommunikasjon@ME-foreningen.no

General contact address NMEA: post@ME-foreningen.no

I would just email all four and ask for confirmation that they’ve received it. All of the emails are from the public NMEA site.

 

[Arfmeister]

The chance of gaining data or any value with off-label treatments is minuscule.

Define minuscule.

Fluge & Mella started their research from a handful of anecdotal case studies (ME remission from cancer)
Consequently they started various offlabel mini-pilots n=3 case studies.

Daratumumab for auto immune diseases started with n=1 off-label case studies.

 

[Utsikt]

Define minuscule.

Fluge & Mella started their research from a handful of anecdotal case studies (ME remission from cancer)
Consequently they started various offlabel mini-pilots n=3 case studies.

Daratumumab for auto immune diseases started with n=1 off-label case studies.

I should have been clearer:
The chance of gaining data of any value from more of the same off-label treatments is minuscule.

You need to start somewhere, but that should ideally be a small pilot trial with as rigorous methodology as possible with an intervention based on a sound rationale. Like JE’s n=5 with ritux for RA or the Dara pilot by F&M.

So «more of the same» is mostly redundant.

None of the people prescribing -mabs for ME/CFS off-label are doing that. They are just making money off of desperate and vulnerable patients.

 

[Arfmeister]

So «more of the same» is mostly redundant.

I think more data = much better. As long as it’s quality data.

- Take into account that the majority of ME drug trials have been quite low quality
- take Abilify : observational study with a lot of defects. Still it’s prescribed widely.

None of the people prescribing -mabs for ME/CFS off-label are doing that.

Agreed. Problematic. Unfortunate.

But it’s too simplistic to say it’s vulture behavior.

E.g. Prof de Meirleir could’ve had a wealth of data on various drugs.
- Unfortunately, he didn’t take time for it
- Probably a combination: of lack of time / too many patients / maybe the data was not good enough (who knows ?)
But in the process of choosing to research ME CFS and treating of label, his academic reputation was ruined.
He could have chosen a different, safer pathway.

BTW I’m not defending him.

*PS I would look the other way and blame BPS crowd for more vulture like behavior.

 

[EndME]

Define minuscule.

Fluge & Mella started their research from a handful of anecdotal case studies (ME remission from cancer)
Consequently they started various offlabel mini-pilots n=3 case studies.

Daratumumab for auto immune diseases started with n=1 off-label case studies.

The situation is quite different though. F&M observations on Rituximab came at a point where they were not setting out making observations about ME/CFS. That tends to give such observations far more meaning due to the role of the observer. If someone sets out to see what they want to see, they are much likely to report so in studies due to various complex reasons. Daratumumab for auto immune disease presumably started because one person on this forum together with his colleagues worked things out and others caught up to that.

There's no need for further Daratumumab case studies in ME/CFS because we're already getting real data soon. Piling up nonsense data till then probably only does harm to the already bad reputation of ME/CFS, but of course nobody serious would do it in the first place which of course means that data by such people will be nonsense to begin with.

 

[Utsikt]

I think more data = much better. As long as it’s quality data.

My assertion is that the data doesn’t have sufficient quality to be of any value.

But it’s too simplistic to say it’s vulture behavior.

It isn’t that hard to do a proper trial. Instead of doing that, they continue prescribing off-label treatments, usually with some reference to how patients are suffering and need treatments now. But they never actually check if their treatments are helping.

PS I would look the other way and blame BPS crowd for more vulture like behavior.

Two wrongs doesn’t make a right. And some of the biobabble people are just as bad as the BPS crowd. Some are even worse, because their treatments are far more expensive and e.g. -mabs can be fatal or lead to severe lifelong issues.

 

[Arfmeister]

we're already getting real data soon

Define ‘soon’ ?
- minimum 3 years if not longer

I’m afraid some severe patients can’t wait that long. Specially the ones that are willing to give up.
- If you’re moderate or mild it’s an acceptable time frame, but I wouldn’t call it soon

 

[Arfmeister]

My assertion is that the data doesn’t have sufficient quality to be of any value.

Absolutely agree.
But “Beggars can’t be Choosers”
- we as ME-patients are at the bottom of the totem pole
- we need to “BEG” for trials and data (vs most of the diseases they have many options to “CHOOSE” for funding trials)
- so do we lower our standards (somewhat) - or do we keep the same standards and except an absolute minimum of data ?

It isn’t that hard to do a proper trial

Actually, very hard if you follow some of the ME scientists.
- even in this thread, we’ve discussed the lack of funding

 

[Utsikt]

Do we lower our standards (a bit) - or do we keep the same standards and except an absolute minimum of data ?

We can’t go below the bare minimum.

Actually, very hard if you follow some of the ME scientists.
- even in this thread, we’ve discussed the lack of funding

Because I have no personal experience, I’m going off of JE’s descriptions.

Besides, part of the reason for the lack of funding is that the underlying rationale doesn’t hold up. There are some double standards here because BPS studies get funding all the time, but many of the bio-ME/CFS studies probably shouldn’t have been done in their current form.

 

[EndME]

Define ‘soon’ ?
- minimum 3 years if not longer

Soon w.r.t. research timelines, not w.r.t to the immense suffering that ME/CFS causes.

 

[Arfmeister]

Because I have no personal experience, I’m going off of JE’s descriptions.

Well, it’s already a very small cake of funding.
- from which a big share is taken (stolen) by BPS researchers + some low(er) quality researchers
- F & M - as good as scientist as they are - are scrambling for cake crumbles

 

[Utsikt]

Well, it’s already a very small cake of funding.
- from which a big share is taken (stolen) by BPS researchers + some low(er) quality researchers
- F & M - as good as scientist as they are - are scrambling for cake crumbles

F&M have so far been able to get millions of GBP/EUR for their studies. They are scrambling on Dara phase 2 for some unknown reason.

And DecodeME got funded. SequenceME will probably get funded. There aren’t many other worthwhile studies out there..