Transcript
CIDP Q&A session
Claudia Sommer, MD, Jan Lünemann, MD, MBA, and Jeffrey Allen, MD
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- A question asked by quite a number of people, how to choose among different, currently available and in brackets, and upcoming treatments. - Yeah, I mean this is of course a very important issue and is high need to identify those patients that respond to one specific therapy. So with disease, its very heterogeneous and we need markers, biomarkers, predictive for treatment responses. And we heard about several biomarkers which are under investigation now and unfortunately we don't have a specific biomarker available now. Complement, activated complement could be a biomarker for complement inhibiting therapies if they will be approved. So this is something that we are currently pursuing in depth. But you know, I agree, there's great need for that and we don't have anything in place right now that could be used in clinical practice. - Yeah, I'll just add to that. I think it's important to keep in mind what we know and from an evidence-based standpoint and still start with evidence-based proven effective therapies. Even though it's exciting to see some newer therapies evolving, it's still important to start with evidence-based proven effective therapies, which include IVIG and corticosteroids and rethinking about diagnosis if those aren't effective, before moving on to unproven therapies.
So those still have an important role, you know, where complement inhibitors may fall into that, probably premature to say, but you know, it's exciting to see what we're seeing in the early trials and perhaps they'll find a place earlier in the treatment pathway, but it's too early to say. - May I have have one additional comment on that? This is why I think it's very important then that the phase two, phase three clinical trials have a biomarker component installed because this is the material that we need to identify predictive biomarkers. - Yeah, very important comment, thank you. There's another question supported by quite a number of people in the audience. What are the dangers of upstream blocking of the complement pathway compared to downstream blocking? Do you have any comment about them? - Yeah, this is of course blocking complement, complement is very important in innate immune defence. It has much serves functions beyond innate immunity. So there's always a risk, there's always a risk in blocking complement, we know it from the C5 inhibitors.
There's increased risk for meningococcal, invasive meningococcal infection, probably also gonococcal infection. So the safety profile of therapies that block complement even upstream of C5, this is something that still needs to be better characterized and the way to do it is actually in clinical trials. So they will tell us. - So one question is should we inhibit complement as early as possible in CIDP to avoid axonal damage? - It would make I think, some sense to treat early before the development of a lot of impairment and irreversible nerve injury. But that's another question that I think we'll need more data to really know when it's optimal to intervene both from a long-term disability standpoint, but also a safety standpoint.
- Yeah, and this one looks tricky too to me. Is there a rationale to combine more than one treatment simultaneously? I mean, we've been studying this, not we personally, but the community has been studying this with the currently available treatments like IVIG and corticosteroids simultaneously. The answer is not entirely clear, is it? - No, I think think we're struggling with monotherapies at this point from a targeted mechanism standpoint. So we know the combination of them other than some maybe theoretical benefits, I think we can't speculate on. - Yeah, here's a biomarker question. What is the role of neurofilaments in axonal degeneration in CIDP and how can they be used to monitor disease progression? Well, I think I showed part of the data and the literature is not uniform on this question. You may be aware that some studies have shown that a rise in NFL can predict a deterioration or a relapse in CIDP patients. Others could not replicate that. So it is their data supporting it and data not entirely supporting NFL as a predictor of a relapse, which would be nice to have, of course, if a patient is on a treatment pause because they seem stable and we could see the biomarker rise before the patient then deteriorates and we could treat again. So this is currently under study and I hope, as my colleagues said, that many of the trials will include biomarkers so that we can learn more about this. Can complement activation and macrophage induced demyelination be two parallel pathways in CIDP?
So I know I was showing it as if it was all one big pathway together, but could they be independent, what do you think? - No, absolutely, they could be. So it's a heterogeneous disease also in terms of pathophysiology and pathology, right? So that can happen in parallel. That can also, one of the two pathways can be dominant in one particular patient. So these are all possible scenarios. It will be very important in the future to identify which pathway is the most important one, mediating the disease pathology in order to target this pathway efficiently. - All right, and one more biomarker question. What is the correlation of changes of these potential biomarkers in clinical presentation when patients present with symptoms? It may be too late to use these biomarkers for early detection. Yes, that's maybe two. So as we showed here, many times the biomarkers are more used for following up treatment response and the development of the disease than for diagnosis. And to have a predictive biomarker that even would show the disease before we see clinical signs as people are looking for in Alzheimer's disease and Parkinson's, I think that's something on the horizon, but on the further horizon for CIDP.
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