Continued Evaluation of Safety, Tolerability, and Clinical Outcomes in Participants with Parkinson’s Disease Throughout Three Years After Bemdaneprocel Administration
Harini Sarva1, Claire Henchcliffe2, Andres Lozano3, Alfonso Fasano4, Suneil Kalia5, Kenny Kwok Hei-Yu6, Cameron Brennan6, Melinda Louie-Gao7, Nicole Floro7, Nauman Abid7, Viviane Tabar6
1Weill Cornell Medical Center, 2University of California, Irvine, 3Toronto Western Hosp, 4University of Toronto, Krembil Brain Institute, Edmond J Safra Program in Parkinson’s Disease, Morton and Gloria Shulman Movement Disorders Clinic, 5University of Toronto & Krembil Brain Institute, 6Memorial Sloan Kettering Cancer Center, 7BlueRock Therapeutics, LP.
Objective:
To report safety and clinical outcomes in participants with Parkinson’s disease (PD) 3 years after receiving bemdaneprocel.
Background:
Bemdaneprocel is an investigational cell therapy composed of human embryonic stem cell-derived midbrain dopaminergic neuron progenitor cells. In exPDite (NCT04802733), a Phase 1 open-label 2-year study, predefined safety, tolerability, and feasibility criteria were met at 1 year; trends toward improvement or stability in clinical assessments were observed through 2 years post transplantation.
Design/Methods:
In exPDite, 12 participants received low-dose (n=5; 0.9 million cells/putamen) or high-dose (n=7; 2.7 million cells/putamen) bemdaneprocel injected bilaterally into the postcommissural putamen in a single surgical session. A 1-year immunosuppression regimen began immediately preoperatively. Following study completion, all participants enrolled in a continued-evaluation study for assessment through 5 years post transplantation (NCT05897957).
Results:
Participants (N=12) were a median of 67.0 years of age, 75% male, and 67% White. Median time since PD diagnosis was 9.0 years. Through 3 years post transplantation, 12 participants experienced 112 treatment-emergent adverse events, mostly mild or moderate in severity; none were related to transplanted cells. Eight treatment-emergent serious adverse events were reported, all unrelated to transplanted cells or immunosuppression. There were no deaths or graft-induced dyskinesias. No intracerebral hemorrhages or mass lesions were observed by magnetic resonance imaging. mean (SD) changes from baseline in patient-reported ON times without troublesome dyskinesia were +2.1 hours (3.2), and OFF times were −2.0 hours (3.2), and MDS-UPDRS Part III ON scores (−5.7 points [7.5]) and OFF scores (−17.9 points [8.5]) showed maintained trends toward improvement. PDQ-39 Summary index scores were similar at baseline through 3 years, with variable changes across domains and cohorts. Overall, outcomes in the low-dose cohort were stable.
Conclusions:
These results support ongoing development of bemdaneprocel for the potential treatment of PD.
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