BlueRock Therapeutics, in collaboration with Bayer AG, recently announced the successful results of their Phase I clinical trial for bemdaneprocel (BRT-DA01), an innovative cellular therapy derived from stem cells, aimed at treating Parkinson’s disease.
The findings were presented at the International Congress of Parkinson’s Disease and Movement Disorders in Copenhagen, Denmark.
Key Highlights:
- Safety and Tolerability: The primary objective of the study was to assess the safety and tolerability of bemdaneprocel. All 12 participants, spanning both low and high dose groups, showed no significant safety concerns for up to a year. Two serious adverse events were reported, but they were not linked to the therapy.
- Positive Clinical Outcomes: Preliminary results indicate improvements in clinical outcomes. Using tools like the MDS-Unified Parkinson’s Disease Rating Scale Part III and the Hauser Diary, participants in the high dose group displayed more significant improvements in Parkinson’s disease severity concerning motor symptoms.
- Cell Survival and Engraftment: 18F-DOPA PET imaging scans, a technique to evaluate dopaminergic activity in Parkinson’s disease, provided evidence of cell survival and engraftment in both dose groups.
- Next Steps: Given the promising results, preparations are in progress for a Phase II study, which is anticipated to commence patient enrollment in the first half of 2024.
Dr. Claire Henchcliffe, one of the study’s Principal Investigators, expressed optimism about the findings, emphasizing the significance of achieving the primary safety and tolerability objectives.
She also highlighted the potential of these preliminary results to lead to substantial benefits for Parkinson’s disease patients in future controlled clinical trials.
About Bemdaneprocel and the Phase I Trial: Bemdaneprocel aims to replace the dopamine-producing neurons lost in Parkinson’s disease. These precursors of dopaminergic neurons are sourced from human embryonic stem cells.
Through a surgical procedure, these neuron precursors are implanted into a Parkinson’s patient’s brain. Once transplanted, they have the potential to reform neural networks severely impacted by Parkinson’s, thereby restoring both motor and non-motor functions.
The Phase I study was a multi-center, open-label, non-randomized trial. Participants received surgical transplantation of bemdaneprocel cells and underwent a one-year immunosuppression regimen. The study assessed safety, tolerability, cell survival, and motor effects.
Conclusion: The positive results from this Phase I trial signify a significant advancement in the quest for innovative treatments for Parkinson’s disease. With millions affected globally by this neurodegenerative disorder, the potential impact of successful therapies like bemdaneprocel cannot be overstated.