Closed 2022 Pilot study in Norway - Daratumumab in ME/CFS

We can’t gamble with dangerous drugs just because there is a chance that it might work.

Who should take responsibility if it goes wrong and people get worse or die?

Who should cover the resources for proven treatments that other sick people are deprived of because we used a limited healthcare budget on experimental and unproven treatments?

Everyone wants treatments as soon as possible. But there are many very good reasons that we do trials first.
maybe see it more as a cost benefit analysis. When does the probability and weight of risk outweigh that of reward. I've been in periods where I was thinking if I stay this bad for another 3 months I don't want yo live anymore. Imagine in a case like that I think it would be ethically dubious to withold a drug with stunning results in a Double Blinded Phase 2 RCT. Of course, bureaucratic and institutional rules mean that sort of sceenario is basically impossible unless you're rich and paying for a drug at a private clinic.

The most loogical thing of course (Imagining the data from this and the phase 2 RCT hold up) is to have an accelerated phase 3 trial with emergency drug approval for ME once the results come out if they are positive.

But of course, we are still far from any of this. in such a small sample, the HLA differences could be randomness. We haven't even had a Controlled and Blinded trial yet. etc
 
You have not really addressed any of the points I highlighted, so I don’t really see any reason to go back and forth saying that we disagree with each other. There are other threads dedicated to the considerations with regards to trialing drugs, so we should try to avoid derailing this thread any further.

But you did not address any of my points. You acted like I was saying i want random untrialled drugs administered because i think a drug should be fast tracked for approval.

Who should take responsibility if it goes wrong and people get worse or die?

Was rolling out the covid vaccines fast a bad thing? People had bad side effects then. Sometimes a situation demands accelated approvals.

Who should cover the resources for proven treatments that other sick people are deprived of because we used a limited healthcare budget on experimental and unproven treatments?
This doesnt really deserve a response because its a ludicrous zero sum game fallacy. Again you keep talking about experimental and unproven treatments and not accelerated approval of a drug tested by a group widely agreed to be among the best ME researchers
 
I really am puzzled by the response here sometimes.

I keep being told there's all this hope then in the same breath that treatments are a decade away and any hint of accelerated approval is tantamount to administering drugs to patients with no evidence.
 
Was rolling out the covid vaccines fast a bad thing? People had bad side effects then. Sometimes a situation demands accelated approvals.
A vaccine like that is far safer than Dara, and covid had far higher fatality rates than ME/CFS will ever get close to.
This doesnt really deserve a response because its a ludicrous zero sum game fallacy.
We do not have unlimited resources, so you have to always consider the alternative cost. That’s basic economics.
Again you keep talking about experimental and unproven treatments and not accelerated approval of a drug tested by a group widely agreed to be among the best ME researchers
It does not matter who did the testing if the data is unsufficient - something it very well might be after a phase 2 study.
 
A good number of people went into remission in the early rituximab trials but a formal controlled trial showed no effect. Life isn't that simple.

Didn't they cut the dose in half in the phase 3 that showed no effect? Or was that Ampligen?

But yes, unfortunately life isn't that simple because otherwise when your doctor tells you your symptoms are being caused by depression and you need to exercise you wouldn't end up with a severe form of one of the worst illnesses in the world.

Life is fiendishly complex but there is no excuse for making people as sick as me wait so unbearably long to try something that has a good chance of helping us.
 
Okay apologies all for crashing out the other day.

On reflection I think as @Yann04 says the most sensible/hopeful course of action is an accelerated phase 3 if phase 2 goes well.

Apologies @Utsikt I took our disagreement a bit too personally. It's a difficult time for me at the minute.
No worries - and apologies back for being brash. We’re in terrible circumstances and I completely understand your desire for things to move as quickly as possible.
 
Now published

Background: Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) entails low quality of life for patients and massive societal costs. There is an urgent need for elucidation of disease mechanisms and for rational treatment. Our working hypothesis is that ME/CFS in a subgroup of patients is associated with functional autoantibodies emerging after an infection, and that plasma-cell depletion with transient reductions in serum immunoglobulins will have a beneficial effect on patients’ symptoms.

Objective: To evaluate feasibility and toxicity of plasma-cell targeting treatment using the subcutaneous anti-CD38 antibody daratumumab (Darzalex®) in moderate to severe ME/CFS, and to assess the clinical course through 12–24 months follow-up after daratumumab intervention.

Methods: We performed a prospective, open-label pilot trial (EudraCT 2022–000281-18). Ten female patients were enrolled. Following 12 weeks run-in, six patients received four daratumumab injections. The next four patients received four, followed by three additional injections from week 14.

Results: All planned treatments were administered, and there were no serious adverse events. Four patients had no significant clinical changes. Six patients experienced marked improvement. For all 10 patients, mean SF-36 Physical Function (SF-36 PF) increased from 25.9 to 55.0 at 8–9 months (p = 0.002). DePaul Questionnaire-Short Form (DSQ-SF) symptom scores decreased from 72.3 to 43.1 (p = 0.002). In six responders, mean SF-36 PF increased from 32.2 to 78.3, and DSQ-SF score decreased from 71.1 to 24.3. Five of these six patients had major and sustained improvement with a mean SF-36 PF of 88 (range 80–95) toward end of follow-up. Mean steps per 24 h was 3,359 (range 1,493–6,277) at baseline. At 8–9 months, the mean number of steps was 5,862, and 7,392 in the six responders. All five patients with sustained improvement reached a mean step count above 10,000/24 h for some weeks, and above 15,000 on individual days. Relative reduction of serum IgG levels was 54% in six patients with clinical improvement, and 40% among four with no benefit. Low baseline NK-cell count in blood was significantly associated with lack of clinical response.

Conclusion: Subcutaneous daratumumab in 10 ME/CFS patients was well tolerated. In six patients, treatment was associated with clinical improvement and concurrent transient reduction of serum IgG levels, indicating important pathomechanistic roles for long-lived plasma cells and functional autoantibodies. No definite conclusions should be drawn before a randomized study has been performed.

 
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