Emerging Therapies and Unmet Needs in MS

EP: 1.Disease-Modifying Therapies (DMTs) in Multiple Sclerosis (MS)

EP: 2.Approaching Treatment for MS Management

EP: 3.Risk of MS Disease Progression and Complementary Diagnostic Tools

EP: 4.DMT Adherence and Routes of Administration in MS

EP: 5.Efficacy and Safety of Oral Therapies in MS

EP: 6.Use of Cladribine in MS Treatment

EP: 7.Sequencing of Therapies in MS

EP: 8.Role of Oral BTKi in MS Management

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EP: 9.Emerging Therapies and Unmet Needs in MS

Ahmed Zayed Obeidat, MD, PhD: What other emerging therapies are in the pipeline that you’re excited about? I know you’re also very interested in stem cells.

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: Yes. Cell-based therapies are coming anew. Unfortunately, there’s a lot of medical tourism, and individuals think they’re getting stem cells. That gives it a bad name because who knows what they’re really getting. If they get something done in some country, and you can’t even know what they put in, and it didn’t work, then it draws away from the excitement. Then maybe some form of stem cell therapy would be beneficial.

There have been some studies. So far, they’re safe. We’ve done a major study in mesenchymal stem cells. They’re safe to give; patients didn’t get sick on them. You can give them intravenously and intrathecally. There are some suggestions that they’re doing something. At this point, knowing that they’re safe to give helps bolster interest in research going further, and there are a lot of companies developing stem cells that are willing to go in.

At ACTRIMS [Americas Committee for Treatment and Research in Multiple Sclerosis] Forum, the theme was viral. One of the interesting aspects might be the Epstein-Barr virus [EBV], which may have an important role in MS [multiple sclerosis]. It may be driving some of the progression we see in the later patients. This ability to generate cells has led to a potential therapy that may be targeting the EBV-transformed cells. If that therapy pans out, that’s going to be a game changer.

Ahmed Zayed Obeidat, MD, PhD: Yes, it is.

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: Phase 2 is just completing. Maybe there will be a phase 3 study soon.

Ahmed Zayed Obeidat, MD, PhD: That’s the hope: to move to a phase 3 study, and hopefully the treatment analysis will show good results. When you think about unmet needs in relapsing multiple sclerosis, [let’s have] a few final words on that. What are the unmet needs in our management of relapsing multiple sclerosis or our care for patients with relapsing MS? What’s missing? We talk about all these therapies, but what else is missing?

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: A lot, unfortunately. The guidance of how to know if it’s working. If I’m your patient, I’ll say, “Dr Ahmed, I’ve been taking this stuff for the last 2 years. Is it doing anything?”

Ahmed Zayed Obeidat, MD, PhD: My answer would be, “From what we can tell or what we can see, yes, it looks like it’s working.”

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: Why? What are you measuring that tells you it’s working?

Ahmed Zayed Obeidat, MD, PhD: Your MRI has been stable.

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: You haven’t seen the activity?

Ahmed Zayed Obeidat, MD, PhD: Yes.

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: Maybe that would have happened anyway.

Ahmed Zayed Obeidat, MD, PhD: That’s right. Maybe I can’t see it.

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: Maybe I didn’t need to take the drug at all. How do you know it’s working?

Ahmed Zayed Obeidat, MD, PhD: This is where biomarkers are going to be very important.

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: Something like neurofilament light chain, which goes down, may help.

Ahmed Zayed Obeidat, MD, PhD: That’s right. Then you’ll be able to show that this is going on.

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: You can say, “Look, this went down. It’s working.” You can’t say that you didn’t have an attack, you didn’t progress, you didn’t have any MRI lesion, because that could have been a natural history without the drug. We know that.

Ahmed Zayed Obeidat, MD, PhD: You want to show a positive effect.

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: A positive biomarker—something that works. That’s 1 of the things that’s an unmet need. The other 1—we touched on it a little earlier—is when can you stop the drug? Do I need to take this for the rest of my life? If not, when is it safe to stop?

Ahmed Zayed Obeidat, MD, PhD: How can we know that it continues to be safe to stop?

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: In particular, with some of the cell depletion therapies like the B-cell depletion, I’m convinced you don’t have to take this long term. There may be an opportunity to treat for 3 or 4 years and then stop. Studies are going to tell us whether we need to continue the barrage against the B cell.

Ahmed Zayed Obeidat, MD, PhD: In the last minute or so, what are some take-home messages to the field, and to patients, and to health care professionals taking care of patients with MS?

Mark S. Freedman, HBSc, MSc, MD, CSPQ, FAAN, FRCPC: I’ve been through years where we had no therapies. Now we have 20-odd therapies. There’s something that can be done for this disease. We can diagnose it early, and we have ways of treating it that are more effective than what we had 10 years ago. We have readouts that help us know when it may be possible to change into a more effective therapy, and we have ways of mitigating against any ill effects from the drugs. There’s no reason that we can’t control this disease in 80% to 90% of patients. That’s extremely hopeful.

Ahmed Zayed Obeidat, MD, PhD: Thank you very much, Dr Freedman. This has been a great discussion. We learned a lot from you and your experience over many years of taking care of patients with MS. Thank you to everyone for watching this NeurologyLive® Peers & Perspectives®. If you enjoyed the content, please subscribe to our e-newsletter to receive upcoming Peers & Perspectives® and other great content right in your in-box.

Transcript edited for clarity