7 Cerebral Palsy Agents to Watch, Gluten-Free Diet Improves Cognition and Quality of Life in PD, Real-World Data on Treatment Transitions to Nipocalimab in gMG

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

First, cerebral palsy (CP) is the most common motor disability of childhood, a group of permanent movement disorders caused by a disturbance in the developing brain before, during, or shortly after birth. It isn't just one disease—presentations range from spastic to dyskinetic and ataxic forms, and roughly 85% to 90% of cases are congenital with no single identifiable cause—and there is still no treatment that addresses the underlying brain injury itself.¹

To recognize World Cerebral Palsy Day on October 6, neurologylive has complied a snapshot of 7 investigational agents and therapies currently being studied in CP, spanning regenerative cell therapies, a cannabinoid formulation, and new approaches to the dyskinesia and spasticity that define the condition, along with the data behind each and what to watch for next.²

Cell-based approaches for CP include GCT-103, or SQ-SHED, which uses stem cells derived from baby teeth and has shown motor and cognitive improvements in preclinical models. Researchers are also studying umbilical cord blood combined with erythropoietin, which improved gross motor performance more than either treatment alone, as well as autologous bone marrow and cord blood therapies, which have produced mixed results in clinical studies.^3-6

Other emerging approaches include NTI164, a low-THC cannabanoid formulation being studied for spastic CP, and valbenazine, a VMAT2 inhibitor that recently failed to meet its primary and key secondary endpoints in a Phase 3 trial of dyskinetic CP. Meanwhile, more established treatment strategies continue to advance, with Phase 3 data supporting Xeomin for lower-limb spasticity in children and adolescents with CP. Together, these programs highlight a pipeline ranging from investigational regenerative therapies to refinements of existing symptomatic treatments, although additional controlled studies will be needed to establish their long-term clinical benefit.^7-13

In other new, fall conference season is officially underway, and NeurologyLive is bringing you the latest research and clinical developments from across neurology. According to late-breaking findings presented at the 2026 International Congress of Parkinson’s Disease and Movement Disorders (MDS), held October 4-8, in Seoul, South Korea, a 12-month gluten-free diet (GFD) was associated with improvements in cognitive performance and quality of life among patients with Parkinson disease (PD). Researchers also reported that the dietary intervention did not significantly affect motor function.¹⁴

Led by Hana Brozova, MD, head physician of the Extrapyramidal Disorders Center at Charles University and General University Hospital in Prague, the prospective, randomized, rater-blinded study included 57 patients with PD who were assigned to either a GFD (n = 30) or a control group without dietary intervention (n = 27). Investigators evaluated motor function, cognition, quality of life, laboratory measures, and serum metabolomic profiles at baseline, 1.5 months, and 12 months.¹⁴

Switching gears, a retrospective survey study presented at the 2026 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Annual Meeting and Myasthenia Gravis Foundation of America Scientific Session in Orlando offered a real-world look at what is driving treatment switches to nipocalimab in generalized myasthenia gravis. The analysis drew on electronic survey responses from 450 neurology health care providers, collecting de-identified data on 37 unique patients who transitioned to nipocalimab from prior advanced therapies including intravenous immunoglobulin, complement inhibitors, FcRn inhibitors, and anti-CD19 agents.

Overall, fluctuating symptom control was the most commonly cited reason for discontinuing a prior therapy, identified by 57% of providers, while preference for a consistent treatment schedule was the top reason for initiating nipocalimab, cited by 68%.

In this Q&A, lead author Christen Kutz, PhD, PA-C, discussed what the finding around fluctuating symptom control reveals about patients' day-to-day experience on therapy and how treatment schedule factors into clinical conversations compared with efficacy data. Kutz, an experienced neurology physician assistant and clinical researcher specializing in multiple sclerosis and neuroimmunological disorders, commented on what the steroid- and immunosuppressant-sparing findings suggest for long-term management, and how clinicians should interpret the 73% improvement rate given the retrospective nature of the data.

To read the full pieces and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. About cerebral palsy. Centers for Disease Control and Prevention. Accessed October 5, 2026. https://www.cdc.gov/cerebral-palsy/about/index.html
2. About World CP Day. World Cerebral Palsy Day. Accessed October 5, 2026. https://worldcpday.org/about/
3. Kidswell Bio and Treehill Partners launch US-based Newco to fast-track development of cell therapy for cerebral palsy. News release. Kidswell Bio Corporation. February 16, 2026. https://www.kidswellbio.com/Portals/0/resources/pdf/en/Topics/2026/20260216_01EN_8fgSyej9x.pdf
4. Kanzawa T, Onoda A, Okamoto A, et al. Novel stem cell therapy for cerebral palsy using stem cells from human exfoliated deciduous teeth. Stem Cell Res Ther. 2026;17:28. doi:10.1186/s13287-025-04828-y
5. Novel regenerative therapy for cerebral palsy using stem cells from baby teeth. News release. Nagoya University Graduate School of Medicine. January 23, 2026. https://www.med.nagoya-u.ac.jp/medical_E/research/pdf/Ste_260126en.pdf
6. Kim M, Lee J, Sung SI, et al. Potentiation of cord blood cell therapy with erythropoietin for children with CP: a 2×2 factorial randomized placebo-controlled trial. Stem Cell Res Ther. 2020;11:509. doi:10.1186/s13287-020-02020-y
7. Cox CS Jr, Juranek J, Kosmach S, et al. Autologous cellular therapy for cerebral palsy: a randomized, crossover trial. Brain Commun. 2022;4(3):fcac131. doi:10.1093/braincomms/fcac131
8. Nguyen LT, Nguyen AT, Vu CD, Ngo DV, Bui AV. Outcomes of autologous bone marrow mononuclear cells for cerebral palsy: an open label uncontrolled clinical trial. BMC Pediatr. 2017;17:104. doi:10.1186/s12887-017-0859-z
9. Biopharmaceutical trials. Neurotech International Limited. Accessed October 5, 2026. https://neurotechinternational.com/biopharmaceutical-trials/
10. Neurotech International Limited. 2024 AGM chairman's address and investor presentation. ASX announcement (ASX:NTI). November 20, 2024. https://announcements.asx.com.au/asxpdf/20241120/pdf/06blcplhbl4bd9.pdf
11. Neurocrine Biosciences provides update on Phase 3 study of valbenazine in dyskinetic cerebral palsy. News release. Neurocrine Biosciences. December 22, 2025. https://www.prnewswire.com/news-releases/neurocrine-biosciences-provides-update-on-phase-3-study-of-valbenazine-in-dyskinetic-cerebral-palsy-302648241.html
12. Merz Therapeutics submits application to the European Medicines Agency for new indication of XEOMIN in pediatric spasticity. News release. Merz Therapeutics. January 25, 2026. https://merztherapeutics.com/merz-therapeutics-submits-application-to-the-european-medicines-agency-for-new-indication-of-xeomin-in-pediatric-spasticity/
13. Banach M, Marciniak M, Smith M, et al. Efficacy and safety of incobotulinumtoxinA for lower-limb spasticity in children and adolescents with cerebral palsy: results of the ELLIE study. Toxicon. 2026;271(S1):7.
14. Brozova H, Polakova K, Gentileova T, et al. Effects of a Long-Term Gluten-Free Diet on Motor Function, Cognition, Quality of Life, Biochemical Markers, and Metabolic Profile in Patients with Parkinson's Disease. Late-Breaking Abstract LBA 14. International Congress of Parkinson's Disease and Movement Disorders; October 4-8, 2026; Seoul, South Korea.