Mechanism of Stem Cell Therapy Bemdaneprocel for Parkinson Disease: Amit Rakhit, MD

WATCH TIME: 3 minutes

"Our 24-month data from the phase 1 study of bemdaneprocel shows a favorable safety profile and promising exploratory efficacy trends. We've seen improvements in on-time by about 1.8 hours, with a corresponding reduction in off-time, and we’re hopeful this therapy could offer a transformative treatment option for patients with Parkinson disease."

To date, Parkinson disease (PD) has been typically managed through a variety of symptomatic approaches, including medicines that increase the level of dopamine, with levodopa as the main therapy. There are several investigational agents in development currently, such as bemdaneprocel, a cell therapy designed to replace the dopamine producing neurons lost in PD. This treatment, designed by BlueRock Therapeutics, has received regenerative medicine advanced therapy (RMAT) designation from the FDA, which enables expedited development review and development planning guidance for a potential future approval.

BlueRock is currently testing bemdaneprocel in a phase 1 study, dubbed exPDite, an open-label, non-randomized, non-controlled trial of 12 individuals with the disease. Newly presented 24-month data at the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS), held September 27-October 1, in Philadelphia, Pennsylvania, showed that the bemdaneprocel is safe, with no adverse events related to the study treatment. In the high-dose cohort, patients showed a mean reduction of 21.9 points on MDS-Unified Parkinson’s Disease Rating (UPDRS)- Part III compared with baseline. Meanwhile, the low dose cohort showed a mean decrease of 8.3 points.

On MDS-UPDRS Part II, those treated with high doses of bemdaneprocel demonstrated a mean reduction of 3.4 points relative to baseline, while the lower dose cohort had a mean increase of 2.0 points. According to Amit Rakhit, MD, chief medical officer and chief development officer at BlueRock, the therapy is unlike traditional treatments, in that it involves the use of stem cells that are differentiated into neuronal precursor cells.

During the meeting, Rakhit sat down with NeurologyLive® to discuss the mechanism of action of bemdaneprocel and its early safety success seen in exPDite. He described that the cells of the agent are injected into specific brain areas with the goal of restoring lost dopaminergic neurons and neural networks. In addition, Rakhit discussed plans for a future phase 2 trial, stating the need for larger cohorts of patients and how it may incorporate similar aspects of design from the phase 1 study.

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