The Phase I clinical trial for bemdaneprocel (BRT-DA01), conducted by BlueRock Therapeutics, has shown promising results in treating Parkinson's disease. The study, which included 12 participants divided into low and high dose cohorts, met its primary objective by demonstrating the therapy's safety and tolerability over one year. No serious adverse events related to bemdaneprocel were reported, although two unrelated serious adverse events occurred.
Participants in the high dose cohort showed greater improvement in exploratory clinical endpoints, as assessed by the MDS-Unified Parkinson’s Disease Rating Scale Part III (MDS-UPDRS Part III) and the Hauser Diary. These tools measure the severity of motor symptoms in Parkinson's disease. Specifically, the high dose cohort experienced a median improvement of 2.16 hours in the "ON" state without troubling dyskinesia and a median decrease of 1.91 hours in the "OFF" state time after one year.
18F-DOPA PET imaging scans provided evidence of cell survival and engraftment in both cohorts, indicating the therapy's potential to reform neural networks affected by Parkinson's disease. The study's positive outcomes have paved the way for a Phase II trial, expected to begin in the first half of 2024, aiming to further evaluate bemdaneprocel's efficacy and safety.
Bemdaneprocel is an investigational cell therapy designed to replace dopamine-producing neurons lost in Parkinson's disease. Derived from human embryonic stem cells, these dopaminergic neuron precursors are implanted into the brain, offering hope for restoring motor and non-motor functions to patients.
The study was conducted across multiple centers and sites, with participants receiving one of two dose levels of bemdaneprocel cells. The feasibility of transplantation and the therapy's motor effects were assessed, with all evaluations set to continue over two years. The research represents a significant step forward in developing new treatment options for Parkinson's disease, a condition affecting over 10 million people worldwide with no current cure.
