Cell Therapy Fails to Alter Gross Motor Scores in Trial

In a randomized, blinded, placebo-controlled crossover trial of 20 children with cerebral palsy, cell therapy failed to alter gross motor function scores compared with placebo, yet diffusion MRI revealed structural white matter changes in 78% of participants, highlighting a major measurement gap in current clinical evaluations.

Structural Imaging vs. Functional Scales in Cell Therapy Trials

The recent crossover trial evaluated 13 children randomized to active cell infusion versus 7 assigned to placebo. Neither bone marrow mononuclear cells nor cord blood produced a statistically significant difference in Gross Motor Function Measure-66 scores.

Out of all participants, 78% exhibited enhanced radial diffusivity within the corticospinal tract, which serves as an indicator of white matter structural health, as detected by diffusion MRI. Increased structural connectivity appeared across motor pathways in all participants regardless of treatment arm, and no cell infusion-related adverse events occurred.

That disconnect between imaging and functional outcomes is a notable finding for the field. The findings imply that existing clinical assessment tools might lack the sensitivity required to spot the structural modifications that cell treatments seem capable of generating.

This challenge has prompted continued work on better biomarkers and longer follow-up in autologous cell trials. For contrast, the Vinmec Research Institute of Stem Cell and Gene Technology in Vietnam ran an open-label, non-randomized trial of autologous bone marrow mononuclear cells administered as two infusions three months apart.

Vinmec reported significant GMFM improvements at 3 and 6 months post-transplantation compared with baseline alongside a reduction in spasticity on the Modified Ashworth Scale. Set against null functional results from blinded placebo-controlled trials, Vinmec’s uncontrolled design illustrates why the field needs more randomized, placebo-controlled data before autologous cell therapy’s functional benefit can be considered established.

Cannabinoids and Movement Modulators in Development

Beyond cellular approaches, developers are advancing targeted pharmacological agents to manage spasticity and dyskinesia. Neurotech International developed NTI164, a proprietary low-THC cannabinoid formulation combining CBDA, CBDP, CBC, CBDB, and CBN.

The company designed NTI164 to ease spasticity with less sedation and cognitive impairment than baclofen, today’s standard pharmacologic option. Approval from the Therapeutic Goods Administration and a human research ethics committee was granted to Neurotech, allowing the launch of a Phase 1/2 trial evaluating NTI164 for spastic cerebral palsy at Monash Medical Centre in Melbourne.

The single-arm, open-label study enrolls up to 14 non-ambulant children across Gross Motor Function Classification System levels II-III with a 12-week treatment course. The Caregiver Priorities and Child Health Index of Life with Disabilities questionnaire serves as the primary outcome measure.

In movement disorders, Neurocrine Biosciences tested valbenazine, a selective vesicular monoamine transporter 2 inhibitor already approved for tardive dyskinesia and Huntington disease chorea. Neurocrine evaluated valbenazine in the KINECT-DCP Phase 3 trial across participants ages 6 to 70 with dyskinetic cerebral palsy over 14 weeks.

In December 2025, Neurocrine reported that valbenazine did not meet its primary or key secondary endpoints versus placebo. The company stated it has not detailed further development plans for valbenazine in dyskinetic cerebral palsy specifically.

Regulatory Submissions for Lower-Limb Spasticity

Merz Therapeutics advanced incobotulinumtoxinA, marketed as Xeomin, following completion of the ELLIE study. Spanning 28 weeks, ELLIE was a multicenter, prospective, randomized, double-blind, placebo-controlled, 2-stage trial involving pediatric patients ages 2 to 17 dealing with lower-limb spasticity tied to cerebral palsy.

Merz reported that the neurotoxin significantly improved lower-limb spasticity with a favorable safety profile, presenting full data at the TOXINS 2026 International Congress on Neurotoxins in January 2026. Building on those findings, Merz submitted an application to the European Medicines Agency in January 2026 seeking to expand Xeomin’s label to cover both upper- and lower-limb spasticity in children and adolescents with cerebral palsy.

Agent	Developer / Sponsor	Trial Phase & Design	Primary Outcome Status
Autologous Cord Blood / BMMNC	Multiple Academic Centers	Phase 2 Crossover, Blinded, Placebo-Controlled	Null on GMFM-66; positive white matter structural imaging
NTI164 (Cannabinoid)	Neurotech International	Phase 1/2, Open-Label, Single-Arm	Active enrollment; early clinical stage
Valbenazine (Ingrezza)	Neurocrine Biosciences	Phase 3, Double-Blind, Placebo-Controlled (KINECT-DCP)	Did not meet primary or key secondary endpoints
IncobotulinumtoxinA (Xeomin)	Merz Therapeutics	Phase 3, Multicenter, Double-Blind (ELLIE)	Met endpoints; under EMA review for pediatric lower-limb label

References

• Centers for Disease Control and Prevention. About cerebral palsy. Accessed October 5, 2026.
• 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.
• 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.
• 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.
• 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.