Objective: This first-in-human Phase 1 study aims to assess the safety, tolerability, clinical efficacy, and functional imaging measures of bemdaneprocel in subjects with PD.

Background: None of the available therapies for PD directly address the loss of DA neurons, one of the principal pathophysiologic processes underlying PD. Bemdaneprocel is an investigational cellular therapy product composed of midbrain dopaminergic neuronal cells derived from human embryonic stem cells that is under development for the treatment of PD.

Method: In this open-label, non-controlled study, 12 subjects have received 1 of 2 different doses of bemdaneprocel to the post-commissural putamen bilaterally, along with a 1-year immunosuppression regimen. Safety and tolerability have been assessed, along with assessments of engraftment and clinical impacts.

Results: At screening, the average age was 66.4 yrs (57-77) and the mean time since diagnosis was 9.1 yrs (5-14). All subjects presented at screening with a Hoehn and Yahr stage of 2 in the on-medication state. The mean MDS-UPDRS part III score was 46.6 (15-73) when assessed in the off-medication state, and patients presented with 4.3 (1.4-6.2) average daily hours of OFF time as assessed by PD diaries. In both cohorts, the safety profile is favorable with the vast majority of reported treatment-emergent AEs being mild to moderate in nature. In the five subjects assigned to the low dose cohort who have completed 1 year of follow-up, 31 AEs were reported. All but 1 incident of a fall were mild to moderate in severity. 16/17 AEs deemed potentially related to the intervention were attributed to the immunosuppression regimen. There were no AEs reported as possibly related to the cell therapy. Patients enrolled in both cohorts to date have demonstrated clinical improvement as measured by the MDS-UPDRS, PD diaries, PDQ-39, and others. F-DOPA PET imaging is also providing evidence of engraftment, survival and functionality of the cells at 1 year.

Conclusion: Results from at least 1 year of follow-up on all patients included in a phase 1 study of bemdaneprocel are reported. An acceptable safety profile, evidence of sustained engraftment, and positive exploratory clinical outcomes in this study will support further investigation of bemdaneprocel for the treatment of PD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/dopaminergic-neuronal-cell-therapy-for-parkinsons-disease-results-from-a-phase-1-study-of-bemdaneprocel/