Neurology Minute

Dr. Bradley Ong discusses the use of eptinezumab in combination with patient education is an effective treatment for reducing disease burden in patients living with chronic migraine complicated by medication overuse. 
Show citation: 
Jensen RH, Lundqvist C, Schytz HW, et al. Eptinezumab With Patient Education for Chronic Migraine and Medication-Overuse Headache: The Randomized, Placebo-Controlled RESOLUTION Trial. Neurology. 2026;106(8):e214863. doi:10.1212/WNL.0000000000214863

Dr. Dan Ackerman and Dr. Reza Bavarsad Shahripour discuss the diagnostic performance of 4 major modalities: TCD, TTE, TEE, and cardiac CT in patients with embolic stroke of undetermined source. 
Sho citation: 
Read the related article in Neurology® Clinical Practice.

Dr. Shuvro Roy and Dr. Amanda Piquet discuss a brief overview of stiff person syndrome, as well as the trial and the trial results. 
Read more about this abstract on the AAN website.  
Show transcript: 
Dr. Shuvro Roy:
Hi, this is Shuvro Roy from the University of Washington and welcome to today's Neurology Minute. I just wrapped a longer conversation with Amanda Piquet from the University of Colorado Anschutz School of Medicine. We were just talking about the recent Phase 2 trial evaluating Miv-cel Kyverna Therapeutics' anti-CD19 CAR T-cell therapy in patients with Stiff Person Syndrome. Amanda, would you mind taking us through a brief overview of SPS as well as the trial and their trial results?
Dr. Amanda Piquet:
So Stiff Person Syndrome, or SPS, is a rare disabling autoimmune neurologic disease with a major unmet need. About 80% of patients ultimately lose their mobility and we currently have no FDA approved therapies. Existing treatments like IVIG, rituximab, and plasmapheresis are all used off label, often requiring chronic dosing and frequently failing to stop progression. KYSA-8 is a registrational Phase 2 study of 26 patients with refractory SPS. Patients experienced rapid, statistically significant and clinically meaningful improvement across all primary and secondary endpoints. Primary endpoint was the timed 25-foot walk. And this improved by a median of 46% at 16 weeks. Of patients requiring walking aids at baseline, about two thirds no longer needed them by week 16 to complete that 25-foot walk. Some patients who had struggled to walk were even able to run again after treatment.
Another key finding was that all patients discontinued chronic immune therapies and remained off treatment as of the last follow-up. From a safety standpoint, miv-cel was generally well tolerated, with no high grade CRS or ICANS observed. In my opinion, these outcomes are unlike anything we've seen previously with Stiff Person Syndrome and may represent a paradigm shift, not only for SPS, but potentially for other antibody-mediated neurologic diseases more broadly.
Dr. Shuvro Roy:
Just curious, are there any upcoming implications for the application of this treatment for patients, you think, in the coming year or so?
Dr. Amanda Piquet:
Kyverna, the company who developed miv-cel, has initiated a rolling BLA with the FDA for potential approval and this would be, if approved, the first CAR-T therapy for SPS. So we're anxiously awaiting the outcome of that process.
Dr. Shuvro Roy:
Fantastic. Amanda, thank you so much for your time. And if you are intrigued and want to know more details behind the findings in the study as well as a conversation around CAR-T therapy for autoimmune neurologic disease as a whole, I encourage you to check out the Neurology Podcast feed for our full conversation there. Thanks for tuning in.

Dr. Shuvro Roy and Dr. Amanda Piquet discuss a brief overview of stiff person syndrome, as well as the trial and the trial results. 
Read more about this abstract on the AAN website.  
Show transcript: 
Dr. Shuvro Roy:
Hi, this is Shuvro Roy from the University of Washington and welcome to today's Neurology Minute. I just wrapped a longer conversation with Amanda Piquet from the University of Colorado Anschutz School of Medicine. We were just talking about the recent Phase 2 trial evaluating Miv-cel Kyverna Therapeutics' anti-CD19 CAR T-cell therapy in patients with Stiff Person Syndrome. Amanda, would you mind taking us through a brief overview of SPS as well as the trial and their trial results?
Dr. Amanda Piquet:
So Stiff Person Syndrome, or SPS, is a rare disabling autoimmune neurologic disease with a major unmet need. About 80% of patients ultimately lose their mobility and we currently have no FDA approved therapies. Existing treatments like IVIG, rituximab, and plasmapheresis are all used off label, often requiring chronic dosing and frequently failing to stop progression. KYSA-8 is a registrational Phase 2 study of 26 patients with refractory SPS. Patients experienced rapid, statistically significant and clinically meaningful improvement across all primary and secondary endpoints. Primary endpoint was the timed 25-foot walk. And this improved by a median of 46% at 16 weeks. Of patients requiring walking aids at baseline, about two thirds no longer needed them by week 16 to complete that 25-foot walk. Some patients who had struggled to walk were even able to run again after treatment.
Another key finding was that all patients discontinued chronic immune therapies and remained off treatment as of the last follow-up. From a safety standpoint, miv-cel was generally well tolerated, with no high grade CRS or ICANS observed. In my opinion, these outcomes are unlike anything we've seen previously with Stiff Person Syndrome and may represent a paradigm shift, not only for SPS, but potentially for other antibody-mediated neurologic diseases more broadly.
Dr. Shuvro Roy:
Just curious, are there any upcoming implications for the application of this treatment for patients, you think, in the coming year or so?
Dr. Amanda Piquet:
Kyverna, the company who developed miv-cel, has initiated a rolling BLA with the FDA for potential approval and this would be, if approved, the first CAR-T therapy for SPS. So we're anxiously awaiting the outcome of that process.
Dr. Shuvro Roy:
Fantastic. Amanda, thank you so much for your time. And if you are intrigued and want to know more details behind the findings in the study as well as a conversation around CAR-T therapy for autoimmune neurologic disease as a whole, I encourage you to check out the Neurology Podcast feed for our full conversation there. Thanks for tuning in.

In the fourth episode of this series, Dr. Stacey Clardy discusses care team essentials and working within multidisciplinary teams. 
Show transcript: 
Dr. Stacey Clardy:
This is the Neurology Minute. I'm Stacey Clardy from the Salt Lake City VA and the University of Utah. This is our 4th episode in our four-part series on Rett syndrome. Today we're going to discuss care team essentials and working within multidisciplinary teams.
Rett syndrome requires coordinated, ongoing, multidisciplinary care across the lifespan. So core team members will often include neurology, genetics, developmental pediatrics, gastroenterology, pulmonology, cardiology, orthopedics, and a range of rehabilitation specialists.
Speech language pathology especially plays a central role, particularly through augmentive and alternative communication strategies, given the characteristic profound expressive language limitations in Rett syndrome. Care coordination obviously is essential, and neurologists will usually serve as the central point of integration, helping families navigate the complexities of care systems internationally and anticipating who might need to be brought in at certain times, given evolving needs.
And caregiver input is especially critical in Rett syndrome patients because the patients have limited verbal communication. So it's these caregivers who are going to be able to provide key insights into daily neurologic status, behavior, and subtle clinical changes that clinicians may well not be able to detect in periodic short office visits. Another essential component is transition planning, right? 
As Rett syndrome patients age, structured transition from pediatric to adult care systems is necessary, essential to maintain continuity and avoid fragmentation. And as in any rare disease, many families find that participation in specialty clinics, and registries, and clinical trials, when available, can provide access to evolving therapies and contribute to ongoing advances in the field.
That's it for our Rett syndrome series. Be sure to listen to the three prior Neurology Minute episodes on Rett syndrome for a full overview. I'm Stacey Clardy for the Minute.