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

V.R.T. said:
Some of us cant afford to wait that long though. If there's an off label drug that might improve my condition with results from a well run phase 2 and there's a decent chance I might be a responder, I'm going to try it. There's a very big risk I don't make it til the results of a subsequent phase 3 study. I'm far from alone in that. And my partner, who is moderate, has to care for me. I can't put that burden on her for any longer than absolutely necessary.

We have people stuck in between life and death in hospitals and dark rooms. People trapped in dangerous living situations. Those people can't wait either.

If the daratumumab pilot responder ratios bore out (obviously its just a sample of ten but for arguments sake) you're talking about 50-60% of pwME responding. And the sooner we have an effective treatment the sooner rank and file doctors start to believe in ME/CFS, which will improve things for everyone.

So if off label prescription is an option we need to prepare for that possibility as best we can, so it is done promptly and safely rather than by Habets-esque cowboys. Maybe that means working to establish an academic centre in the meantime - we've heard that the winds might be changing on that front soon anyway.

Perhaps at first daratumumab might be given by oncologists like F&M? I think the protocol is already laid out in the trial is it not? And they would be most familiar with how to give the drug.
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I think the inclusion criteria of the pilot trial is that the onset should be after inmunological trigger ( infection ).
I am not sure what percentage of pwme follow that criteria.
 
V.R.T. said:
Some of us cant afford to wait that long though. If there's an off label drug that might improve my condition with results from a well run phase 2 and there's a decent chance I might be a responder, I'm going to try it. There's a very big risk I don't make it til the results of a subsequent phase 3 study. I'm far from alone in that. And my partner, who is moderate, has to care for me. I can't put that burden on her for any longer than absolutely necessary.

We have people stuck in between life and death in hospitals and dark rooms. People trapped in dangerous living situations. Those people can't wait either.

If the daratumumab pilot responder ratios bore out (obviously its just a sample of ten but for arguments sake) you're talking about 50-60% of pwME responding. And the sooner we have an effective treatment the sooner rank and file doctors start to believe in ME/CFS, which will improve things for everyone.

So if off label prescription is an option we need to prepare for that possibility as best we can, so it is done promptly and safely rather than by Habets-esque cowboys. Maybe that means working to establish an academic centre in the meantime - we've heard that the winds might be changing on that front soon anyway.

Perhaps at first daratumumab might be given by oncologists like F&M? I think the protocol is already laid out in the trial is it not? And they would be most familiar with how to give the drug.
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I just wanna agree with this. Obviously theoretically I want to be super rigorous S4ME style.

But if theres a successful phase II, and I can afford it. I might try it off label.
I mean I honestly don’t know how long I can last at my current severity. My reality is not sustainable.
 
Sasha said:
Quite possibly! I haven't read stuff properly and am very confused about what stage we're at and what needs to happen next.
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It looks like length of this study is 72 weeks per person, with staggered involvement (not everyone starting or finishing at the same time) and recruitment open from 06.06.2025 until 01.12.2026. So I wouldn’t expect results until later into 2028 at the earliest.
 
EndME said:
The Charite regularly runs trials, @Mebfeb has an ongoing trial, Simmaron is doing some stuff and others are active as well
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Are any of these trials remotely interesting? As far as I can tell the Charité runs two trials on immunoadsorption, one on hyperbaric oxygen and one on methylprednisolone. I'm not sure what @Mebfeb is and Simmaron is looking at rapamycin which, anecdotally, doesn't seem to help.
 
EndME said:
There's currently quite a few ME/CFS clinical trials. The Charite regularly runs trials, @Mebfeb has an ongoing trial, Simmaron is doing some stuff and others are active as well. I don't think one has to worry about things not working out if there is actually positive data. Currently people are running trials whilst being in the dark, I'm pretty sure they'll all happily turn on the lights if they could. Patient recruitment will always be a problem but I'm not sure if there is much one can do about that right now or at least not on the basis of anything that is dependent on Fluge and Mella's work.

I guess "physican led services" is a UK-centric viewpoint (or what would the Charite considered to be)? I thought the problem was that it's hard to get one genuinely interested person invested to run an ME/CFS-clinic and possibly that might take a positive phase 2 trial or other genuinely useful data? At least I don't think that any of that discussion should be dependent on Daratumumab and I don't think one can get anyone smart invested on the basis of the current Daratumumab data.

In short: if you try to get people interested on the basis of the Daratumumab data you'll likely get the wrong kind of people.
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I think we're at cross-purposes! I agree we can't do anything right now based on the Norwegian work but I'm not talking about setting things up for the next trial. I'm talking about preparing for the time when a drug (perhaps dara) has been proved to work, and it needs to be delivered to PwME.
 
hotblack said:
It looks like length of this study is 72 weeks per person, with staggered involvement (not everyone starting or finishing at the same time) and recruitment open from 06.06.2025 until 01.12.2026. So I wouldn’t expect results until later into 2028 at the earliest.
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:arghh:

Although presumably if it's super-successful it would be stopped on ethical grounds (i.e. unethical to continue if you've already demonstrated massive benefit)?
 
Sasha said:
I'm talking about preparing for the time when a drug (perhaps dara) has been proved to work, and it needs to be delivered to PwME.
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It would be great to be able to work towards that, but I'm not sure what we could do. The NHS would be managing it, and I doubt we could easily get involved in the technicalities of rollout and delivery of a drug therapy.

We could certainly advocate for things like support for severely ill people in accessing clinics, but we'd have to find out—presumably through feedback from patients who'd experienced it—what the issues were before we could argue for improvements..
 
hotblack said:
I’m not sure how we would know it’s a success until the trial is complete and unblinding occurs.
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I still know barely anything about this trial (I need to read up!) but conventionally, I think that large RCTs do planned interim analyses to check for outrageous success or harm, either of which might mean early stopping. I don't know if this trial is large enough.
 
Kitty said:
It would be great to be able to work towards that, but I'm not sure what we could do. The NHS would be managing it, and I doubt we could easily get involved in the technicalities of rollout and delivery of a drug therapy.

We could certainly advocate for things like support for severely ill people in accessing clinics, but we'd have to find out—presumably through feedback from patients who'd experienced it—what the issues were before we could argue for improvements..
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I'm really thinking about the day that, for example, a drug is revealed in a trial as successful for ME/CFS. If that happened today, I'd like to know what would be stopping us all from getting the drug tomorrow - so that we can knock those obstacles down. Yes, in the UK, the NHS would be managing it, but it would be doing it from a standing start, with a country full of useless BACME clinics that don't even see PwME after first diagnosis. Maybe the NHS has a ready-made mechanism for roll-out in this situation - but maybe it doesn't. And if our decades of neglect has left us in a position where we need to make sure the NHS is ready to provide something extra to get us to the starting-blocks, now's the time to think about it.

I just don't know what's involved. You may be right that we can do nothing, but I don't want to get to Drug Day and find out that we should have spent years campaiging for something!
 
Sasha said:
I think that large RCTs do planned interim analyses to check for outrageous success or harm, either of which might mean early stopping.
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Would we want early stopping? People in the UK are unlikely to get full access to a treatment unless NICE recommends it, and the best way of doing that is a full scale, completed trial so they can consider the evidence properly.
 
Kitty said:
We could certainly advocate for things like support for severely ill people in accessing clinics, but we'd have to find out—presumably through feedback from patients who'd experienced it—what the issues were before we could argue for improvements..
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Yeah, I think we’ve discussed this before, but getting appropriate basic support in place should be our focus I think. That’s not only badly needed but everything else including trials or treatments depends upon or builds on that.
Sasha said:
I still know barely anything about this trial (I need to read up!) but conventionally, I think that large RCTs do planned interim analyses to check for outrageous success or harm, either of which might mean early stopping. I don't know if this trial is large enough.
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Yeah I’ve heard that can happen too, but what looks like the staggered nature here may make that difficult, I’m not sure. There’s some good documents (and maybe one less good one) on the fundraising page, well worth a read.
 
Kitty said:
Would we want early stopping? People in the UK are unlikely to get full access to a treatment unless NICE recommends it, and the best way of doing that is a full scale, completed trial so they can consider the evidence properly.
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Yes, we would want early stopping if the trial was so successful that it busted through the evidence threshold early. The only reason you do a big trial is to give yourself the statistical power to pick up the predicted small effect size. If the effect is bigger than you thought, you have an ethical obligation to stop early so that you can get the drug out to patients.
 
Sasha said:
Yes, we would want early stopping if the trial was so successful that it busted through the evidence threshold early.
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That's all well and good if you can afford private treatment, but many of us can't—we'll be waiting on the development of NHS services. The best way to get those is as much evidence as possible to convince even the most sceptical minds. We're unlikely to be considered some kind of special case to which the normal rules don't apply.
 
Kitty said:
That's all well and good if you can afford private treatment, but many of us can't—we'll be waiting on the development of NHS services. The best way to get those is as much evidence as possible to convince even the most sceptical minds. We're unlikely to be considered some kind of special case to which the normal rules don't apply.
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But as I understand it, early stopping is normal in trials if they bust through the evidence barrier big-time and early - ethically, they must stop. This is not a special case. It's standard. There would be no reason for the NHS to turn its nose up - it would be business as usual and the results would have the same standing as a trial that trundled on to completion with a smaller effect size.
 
Sasha said:
But as I understand it, early stopping is normal in trials if they bust through the evidence barrier big-time and early - ethically, they must stop. This is not a special case.
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I've only heard of that happening in trials of drugs for life limiting illnesses, and I'd be surprised if it's common even then. As much as we know it can be life limiting, the NHS wouldn't see ME/CFS as that kind of case.

I think the best thing for all of us is properly collected and analysed evidence, with follow up of participants to see if improvements are sustained. If so, that would be a persuasive case even if the drug concerned is quite costly.
 
Kitty said:
The question may have been answered anyway, but it could well be to prevent commercial interests making large profits from treatments developed using philanthropic or charitable funding that was given with the aim of improving sick people's lives.
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I still don’t understand how others would be able to make lots of money on something they can’t patent. Sure, they can compete, but the lack of monopoly would drive the prices and profits way down.

The owner of the patent could make them free to use, or force anyone that uses it to cap the price, but it doesn’t look like that’s the goal here.
hotblack said:
we just don’t know the reasons for Fluge and Mella making this patent claim. It may be useful to, but we don’t.
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Yes, we do know why. Their employer patents inventions with the aim of making a profit from them. Turns out Fluge and Mella doesn’t own the patent, they are just the inventors.
Utsikt

Open Post in thread 'Norway: Plasma cell aimed treatment with daratumumab in ME/CFS - Haukeland University Hospital'

Kitty said:
The reason could be something as boring as a condition of grant,
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I don’t understand how this would work, because why would those that provide the grant care about if something gets patented, unless they own a share of the patent?
Kitty said:
or that their institution requires researchers to do it as a matter of course.
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Turns out that’s what has happened here:
Haukeland University Hospital, through its Technology Transfer Office, Vestlandets Innovasjonsselskap AS, holds a pending patent application related to plasma cell-targeting treatments for ME/CFS, WO2021038097A1: method for...
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