Positive 24-Month Data of Cell Therapy Bemdaneprocel in Parkinson Disease: Amit Rakhit, MD

WATCH TIME: 4 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."

Parkinson disease (PD), the second most common neurodegenerative disorder, has been long considered to be among the most promising target diseases for cell replacement therapy due to the specific loss of dopaminergic neurons in the substantia nigra. Many of the currently available treatments, all symptomatic, have limited effects on patients’ daily lives, and don’t directly target the underlying issues of the disease. One therapy in development, bemdaneprocel (BRT-DA01), is designed to replace the dopamine producing neurons that are lost in PD.

At the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS), held September 27-October 1, in Philadelphia, Pennsylvania, investigators presented new 24-month data from the phase 1 exPDite trial assessing bemdaneprocel in patients with PD. This multi-site, open-label, non-randomized trial featured 12 patients with PD who received surgical transplantation of 1 of 2 different dose levels of bemdaneprocel to the post-commissural putamen bilaterally, and administration of a 1-year immunosuppression regimen. Cohort A (n = 5) received a dose of 0.9 million cells per putamen, while Cohort B (n = 7) received 2.7 million cells per putamen.

The dopaminergic neuron precursors of bemdaneprocel are derived from pluripotent stem cells that are human embryotic stem cells. In a surgical procedure, these neuron precursors are implanted into the brain of a person with PD. To date, bemdaneprocel has received a Regenerative Medicine Advanced Therapy designation from the FDA, which allows for quicker development review and development planning guidance.

Amit Rakhit, MD, MBA, chief medical officer and chief development officer at BlueRock Therapeutics, sat down with NeurologyLive® during the meeting to discuss the newly presented 24-month data. He spoke about the design of the trial, the safety of the cell therapy, and some of the encouraging efficacy trends observed. Furthermore, he provided commentary on the challenges clinicians face when testing new mechanisms of action, as well as the importance of identifying the right patient who may be eligible for cell therapy treatment.

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