Encouraging Results for Investigational Cellular Therapy for Parkinson Disease

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Results of a phase 1 study (NCT04802733) presented at the 2023 International Congress of Parkinson’s Disease and Movement Disorders revealed positive safety and tolerability outcomes through year 1 for bemdaneprocel (BRT-DA0l; BlueRock Therapeutics, Cambridge, MA) in patients with Parkinson disease (PD). In addition, encouraging secondary outcomes from this study were reported which showed improvements in motor symptoms associated with PD. Bemdaneprocel is an investigational cell therapy implanted into the brain with the goal of replacing dopamine-producing neurons.

The open-label, noncontrolled study included 12 subjects who received bemdaneprocel transplantation in the brain at either 0.9 million cells per putamen (cohort A; n=5) or 2.7 million cells per putamen (cohort B; n=7). Both cohorts met the primary outcome of safety and tolerability measured at 1 year by incidence of serious adverse events (SAE) or abnormal tissue overgrowth related to cell transplantation. The 2 cohorts also met secondary outcomes, with transplanted cells showing evidence of survival and engraftment according to 18F-DOPA PET imaging as well as clinical improvement of 7.6 points and 13.0 points on the Movement Disorder Society–Unified Parkinson’s Disease Rating Scale Part III (MDS-UPDRS III) compared to baseline in cohort A and B, respectively. Additionally, participants in cohort A demonstrated an improvement of 0.72 hours in the “on” state and a decrease of 0.75 hours in the “off” state, whereas cohort B showed an improvement of 2.16 hours in the “on” state and a decrease of 1.91 hours in the “off” state according to an analysis of Hauser Diary records.

“The data from this phase 1 open label study are extremely encouraging,” said Claire Henchcliffe, MD, Chair of the UCI School of Medicine Department of Neurology at the University of California, Irvine and one of the study’s Principal Investigators. “While this is a small open label study, meeting the study’s primary objective for safety and tolerability along with initial improvements seen in clinical outcomes represents a great step forward.”

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