Advanced Therapy for Parkinson’s Rocks Positive Phase I Data
29 June 2023
Regulatory & Standards
BlueRock Therapeutics has reported promising outcomes from its Phase I clinical trial investigating its neuronal stem cell therapy Bemdaneprocel (BRT-DA01) as a potential first-in-class treatment for Parkinson’s disease.
Parkinson’s disease, characterized by the progressive loss of dopamine-producing neurons in the brain, currently lacks a cure.
BlueRock’s innovative approach utilizes neuronal stem cells to address the underlying cause of the disease and potentially restore neural function.
The investigational Bemdaneprocel is comprised of dopamine producing neurons derived from pluripotent stem cells that are then surgically implanted into the brain of a person with Parkinson’s disease. When transplanted, these cells have the potential to reform neural networks that have been destroyed by Parkinson’s disease in the hope of restoring motor and non-motor function to patients.
The Phase I clinical trial involved a group of 12 patients who received BlueRock’s neuronal stem cell therapy. Preliminary results from the trial demonstrated the safety and potential efficacy of the therapy.
Patients treated with neuronal stem cell therapy exhibited improvements in motor function, including reduced tremors and enhanced mobility. The treatment was well-tolerated, with no significant adverse effects reported during the study.
“The safety profile of bemdaneprocel was encouraging along with early evidence of cell survival and engraftment, marking a very important step in the development of a potential new therapy for patients with this disease. These topline data provide a strong rationale for initiating the next phase study, and we look forward to advancing this clinical program,” commented Ahmed Enayetallah, Senior Vice President and Head of Development, BlueRock Therapeutics.
Detailed Phase I trial data from primary and secondary endpoints will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in Copenhagen, August 27th-31st.
While the Phase I clinical trial signifies a significant milestone, further extensive studies are necessary to confirm the long-term safety and efficacy of BlueRock’s neuronal stem cell therapy. A Phase II clinical study is expected to begin enrolling patients in Q1 of 2024.
“The positive outcome of our first cell therapy clinical trial for Parkinson’s is encouraging not only for the bemdaneprocel development program but also our entire pluripotent stem cell-based platform and warrants further investigation in larger groups of patients” adds Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development.