Most drug trials with a known biological target fail. Without one, the odds are almost zero. This is not a viable way to do anything unless a large number of already-tested drugs are tested, and even then it's a terrible methodology since it's unlikely that any treatment would be effective for most. There would be too much noise. Without basic research this will go nowhere. Even RECOVER's $500M budget for trials doesn't inspire me one bit of confidence.
I doubt that it was the primary outcome scale that was the major problem here, causing the people who own the BC-007 intellectual property to pack up and go home. If patients were reporting miraculous recoveries in everything except fatigue, I'm sure they would tweak their experimental design and proceed on to Phase 3. Also, I'd have to go back and look, but I'd be surprised if the researchers didn't also have a range of secondary outcomes. Presumably they were not looking great either.
Also, if there was a positive effect, I think this would have still be recorded by that measure - as I think it was used already in their first studies and now It's just the placebo controlled phase that probably showed that previously reported improvements (by the same scale) couldn't be due to the supposed mechanism of the drug? I hope they will publish their data soon so people can look at this in detail and no need to speculate. Primary Outcome Measure: Mean change from baseline in score on FACIT-FS scale at Day 30. Secondary Outcome Measures: To compare GPCR AAB neutralizing effect of BC 007 [different doses, MSE ] with that of placebo.
From what I've heard from the people that are part of the trial in Erlangen, is that they are seemingly being told that the neutralizing effect of BC007 is high in comparison to placebo. If that was true it would suggest that the drug works as intended, without yielding any efficacy. I'm not sure whether we'll get a quick publication. When results are bad these things tend to often get backtracked. After all Erlagen hasn't even published more than the one case report till now even though they had mentioned that they had intended to publish multiple.
I hope they publish something because a negative result could rule out a mechanism. On an unrelated note, I now wonder what the people who raised money to conduct a trial of BC-007 in ME/CFS are going to do with their funds.
Yes, even if the proposed mechanism related only to the proportion of pwLC that showed what some researchers established as (potentially) pathological levels of GPCR AAB. (I forgot what the proportion actually was) Perhaps still related -- using the funds for a publication of the LC study results and maybe an additional sensible review on the evidence on the potential role of GPCR AAB in ME/CFS?
It seems a shame people are criticising the primary outcome measure and study design after the fact. But I guess some people had invested their hopes in this? That would be interesting. Publication would really help, in terms of potential for understanding mechanisms but also to help those disappointed with the results. Going quiet seems particularly unhelpful for them.
This is a common thing. I see almost as much people saying papers chose the wrong outcome measures for what should have been a positive effect, than the opposite. And as most of us on this forum can attest to, understating findings seems far less common than overstating. People want to believe things work. So they tend to be more likely to criticise a study showing no positive effect than one showing positive effect.
Did they get an opportunity to comment on them beforehand? I don't know much about this study, tbh. It's not very common for patients to be consulted on the outcome measures before a trial design is finalised, but if they did in this case, it is a shame they didn't raise concerns earlier. Some of the comments are a useful reminder that if we get to the stage of testing drugs for ME/CFS, we need to plan for the possibility of people seeing worthwhile improvements in individual symptom domains (sleep, pain, etc) even if the overall effect is limited.
Hoping that they make the data public so that others can analyze it. Is this all that they report: not even an effect size or table of the main findings?
Soundis like a problen with our medixal system if health companies have no incentive to publish negative data even if it is useful for figuring out things about a condition.
It sounds like a private limited company, though, rather than part of the public health system. If there's no prospect of future profit, their investors are likely to simply stop the funding. They would have to pay a salary to get the data prepared and a paper drafted, and there's no incentive for them to do it if they're sure the drug is a dead end for long Covid.
But doesn’t the public health system end up atleast partially funding a lot of these trials done by private companies? If that’s the case, I don’t see why they couldn’t require a public release of anonymised data or even a publishing of negative results.
The problem is then that probably becomes a disincentive for private companies to run trials. Or at least they will say it is and/or threaten to run trials elsewhere and so governments won’t push for it. I don't think there’s a reliable way of forcing companies to do this. Other than taxing them and investing in R&D where governments can stipulate this requirement.
Honestly I doubt companies will shy away from doing clinical trials on drugs they have patented, just because the funding they receive from governments is conditional on publishing results. I do think they’d put a lot of pressure on government officials through their lobbyists though. At the end of the day, their goal is bringing a profit to shareholders, and having to publish a few studies isn’t going to change whether they conduct trials on drugs they patent, it just means they’ll have to ever so slightly spend a tiny bit more.
Interesting claims from Bettina Hohberger from the German Fatigatio Facebook page (FB translation): #LongCovidCongress: Dr. Bettina Hohberger from the FAU Erlangen-Nürnberg takes on the 3. Long Covid Congress on November 25, 2024 in Berlin preliminary presentation on the drug #BC007: This afternoon, presenting its own research data and announced results different than Berlin Cures recently. You can detect significant differences in the #Placebo- and the BC007 group, for example in the special MRI, according to Dr. Hohberger. This makes the substance seem to have an effect - unlike the brief representation of Berlin Cures on their website. We hope that Dr. Hohberger's results in her upcoming presentation at the Long Covid Congress, which will not be broadcast live, will be presented later in the summary and therefore clear to us.
I suspect if the drug would have efficacy she would quote data from the outcome measures not some speculative correlation?
If none of the outcome measures show a meaningful change, crumple it up and can it. It doesn't work and scientists need to spend their effort on other treatments for long Covid. That being said, I'm a bit sad seeing it fail after all the dramatic testimonials of it curing long Covid. A couple years ago, I had a decent amount of hope that BC-007 or another drug would show some effect. And it failed. Hopefully another drug will show promise. Fortunately, there are more being studied.
