From Cord Blood to Cannabinoids: 7 Agents to Watch in Cerebral Palsy

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 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

Ahead of World Cerebral Palsy Day on October 6, here's 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

1. S-Quatre (Kidswell Bio Group) — GCT-103 (SQ-SHED)

GCT-103, also referred to as SQ-SHED, is a cell therapy developed by S-Quatre Corporation, a Kidswell Bio Group company based in Tokyo, using stem cells from human exfoliated deciduous teeth (SHED), harvested from naturally shed baby teeth.³ In a preclinical model of chronic-phase CP-like injury, researchers from Nagoya University reported that intravenously administered SHED migrated to the injured brain and secreted hepatocyte growth factor, which activated the PI3K-Akt pathway and promoted proliferation of endogenous neural stem cells; treated animals showed improved forelimb use, better motor coordination, and improved performance on learning and memory testing even when treatment began after deficits were already established.^4,5

On the clinical side, Kidswell Bio and London-based Treehill Partners announced in February 2026 that they were launching Kidswell USA, a new US-based entity, to accelerate development of SQ-SHED for American children with CP, with Thermo Fisher Scientific selected as the preferred development services partner. The companies described the program as already clinical-stage, with “strong initial signals of clinical efficacy in CP,” though they did not disclose trial-level data in the announcement.³ With an estimated 8,000 to 12,000 new CP cases in the US each year and no disease-modifying options available, the partners framed the effort as building a faster, more capital-efficient path toward US approval.

2. CHA University — Allogeneic Umbilical Cord Blood Plus Erythropoietin

Researchers at CHA University's Rehabilitation and Regeneration Research Center in Seongnam, South Korea, have tested a combination approach that pairs donor (allogeneic) umbilical cord blood (UCB) cells with erythropoietin (EPO), reasoning that the 2 could work synergistically: UCB cells for their immunomodulatory and paracrine effects, and EPO for its neurotrophic and anti-apoptotic properties.⁶

In a 2×2 factorial, randomized, placebo-controlled trial of 88 children with CP, the group that received both UCB and EPO showed significantly greater improvement in gross motor performance measure (GMPM) scores at 12 months than children who received placebo for both (a change of 6.85 points versus 2.34 points; P = .018), outperforming either treatment given alone, with no treatment-related harm reported. The authors concluded that the combination potentiated motor recovery beyond what either cell therapy or EPO achieved individually.

That synergy is the key finding to build on, and it raises the obvious next question for the field: whether larger, dose-optimized trials of combination regenerative approaches can replicate and extend this effect, a path CHA University's group has continued to pursue in follow-on CP research since this trial published.

3. UTHealth Houston (McGovern Medical School) — Autologous Bone Marrow Cells vs Autologous Cord Blood

A different academic program, based at McGovern Medical School at UTHealth Houston, has focused on autologous (self-donated) cell sources instead of donor cells, directly comparing autologous bone marrow-derived mononuclear cells (BMMNC) against autologous umbilical cord blood in the same randomized trial.⁷

In a randomized, blinded, placebo-controlled crossover trial of 20 children with CP (13 randomized to active cell infusion, 7 to placebo), neither BMMNC nor cord blood produced a significant difference in Gross Motor Function Measure-66 scores compared with placebo. However, diffusion MRI showed improved corticospinal tract radial diffusivity, a marker of white matter structural integrity, in 78% of participants, with increased structural connectivity in motor pathways across all participants regardless of treatment arm; no cell infusion-related adverse events occurred.

That disconnect between imaging and functional outcomes is itself a notable finding for the field: it suggests current clinical scales may not be sensitive enough to detect the kind of structural change cell therapy appears capable of producing, and it's prompted continued work on better biomarkers and longer follow-up in autologous cell trials for CP.

4. Vinmec Research Institute of Stem Cell and Gene Technology — Autologous Bone Marrow Mononuclear Cells

Vinmec's Research Institute of Stem Cell and Gene Technology, part of the Vietnamese Vinmec healthcare system, has run one of the larger published cell-therapy programs in CP using autologous BMMNC, administered as 2 infusions 3 months apart, without a placebo comparison.⁸

In an open-label, non-randomized trial, gross motor function (measured by GMFM) improved significantly at 3 and 6 months post-transplantation compared with baseline (P < .001), with a similarly significant reduction in spasticity on the Modified Ashworth Scale; safety was favorable, with no serious complications, though some children experienced transient fever or vomiting around the infusions.

Set against UTHealth's blinded, placebo-controlled null result on functional outcomes above, Vinmec's uncontrolled design is the clearest illustration of why this field needs more randomized, placebo-controlled data before autologous cell therapy's functional benefit (as opposed to its apparent safety) can be considered established; that gap is the main thing to watch as larger, controlled follow-up trials of this approach emerge.

5. Neurotech International — NTI164

NTI164 is a proprietary, low-THC (<0.3%) cannabinoid formulation combining CBDA, CBDP, CBC, CBDB, and CBN, developed by the Australian biotech Neurotech International, which has also studied the compound in autism spectrum disorder and Rett syndrome.⁹ The company's stated rationale for spastic CP is that NTI164 might ease spasticity with less sedation and cognitive impairment than baclofen, today's standard pharmacologic option, though a specific mechanism of action in CP has not been detailed publicly.

Neurotech received human research ethics committee and Therapeutic Goods Administration clearance to begin a Phase 1/2 trial of NTI164 in spastic CP, planned as a single-arm, open-label study enrolling up to 14 non-ambulant children (Gross Motor Function Classification System levels II-III) at Monash Medical Centre in Melbourne, with a 12-week treatment course and the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire as the primary outcome measure.^9,10

As of the company's most recent public disclosures, the CP program remained in its early clinical stages, with no efficacy data yet reported for this indication. The trial's enrollment start and first readout are the milestones to watch next.

6. Neurocrine Biosciences — Valbenazine (Ingrezza)

Valbenazine is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor already FDA-approved for tardive dyskinesia and chorea associated with Huntington disease; Neurocrine Biosciences tested it in dyskinetic CP on the rationale that dampening excess dopamine release via VMAT2 inhibition could reduce the chorea and other involuntary movements that define that CP subtype.¹¹

In KINECT-DCP, a randomized, double-blind, placebo-controlled Phase 3 trial in participants ages 6 to 70 with dyskinetic CP and choreiform movements, treated over 14 weeks with an optional open-label extension, Neurocrine reported in December 2025 that valbenazine did not meet its primary or key secondary endpoints versus placebo, despite the study being, by the company's account, the largest double-blind, placebo-controlled trial ever completed in dyskinetic CP; the safety profile was consistent with valbenazine's established profile.

Neurocrine said it plans to present the full dataset at an upcoming scientific meeting but has not detailed further development plans for valbenazine in dyskinetic CP specifically.

7. Merz Therapeutics — IncobotulinumtoxinA (Xeomin)

IncobotulinumtoxinA, marketed as Xeomin, is a botulinum toxin type A that blocks acetylcholine release at the neuromuscular junction to reduce muscle overactivity; it's already FDA-approved for pediatric upper-limb spasticity in the US, and Merz Therapeutics has now generated Phase 3 data specifically for lower-limb spasticity in CP.¹²

That evidence comes from ELLIE, a prospective, randomized, placebo-controlled, double-blind, 2-stage, multicenter trial running 28 weeks in children and adolescents ages 2 to 17 with CP-related lower-limb spasticity, which Merz says significantly improved lower-limb spasticity with a favorable safety and tolerability profile; full data were presented at the TOXINS 2026 International Congress on Neurotoxins in January 2026.^12,13

Built on that trial, Merz submitted an application to the European Medicines Agency in January 2026 seeking to expand Xeomin's EU/EEA label to cover both upper- and lower-limb spasticity in children and adolescents with CP. The EMA's decision on that application, and whether Merz pursues a parallel lower-limb submission with the FDA, are the next milestones to track.

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.