BlueRock Therapeutics' Bemdaneprocel Shows Promise in Parkinson's Disease Phase I Trial
核心洞察
BlueRock Therapeutics (搜索)' bemdaneprocel demonstrates sustained cell survival and increasing F-DOPA signal after immunosuppression cessation in Parkinson's patients.
High-dose cohort shows significant motor symptom improvement, with a 2.7-hour increase in 'Good ON' state and a 23-point reduction in MDS-UPDRS Part III score.
The Phase I trial indicates potential for addressing non-motor symptoms, with neuropsychological evaluations suggesting improvements in memory and cognitive function.
Bayer AG and its subsidiary BlueRock Therapeutics (搜索) announced positive 18-month data from their Phase I clinical trial of bemdaneprocel, a stem cell-derived therapy for Parkinson's disease (搜索), at the Alzheimer's and Parkinson's Diseases Conference in Lisbon, Portugal. The trial (NCT04802733) assessed the safety, tolerability, and preliminary efficacy of bemdaneprocel in twelve participants with idiopathic Parkinson's disease.
Sustained Cell Survival and Motor Improvements
The data indicated that bemdaneprocel continues to be well-tolerated, with no major safety concerns reported. Transplanted cells survived and engrafted in the brain, with F-DOPA signal increasing even after the cessation of immune suppression therapy at 12 months, as per the study protocol. Participants in the high-dose cohort (2.7 million cells/putamen) demonstrated greater improvements compared to the low-dose cohort (0.9 million cells/putamen).
Using the Hauser Diary, high-dose participants showed a mean increase of 2.7 hours in time spent in the 'Good ON' state compared to baseline after 18 months. Time spent in the 'OFF' state decreased by a mean of 2.7 hours. In contrast, the low-dose cohort showed a mean improvement of 0.2 hours in 'Good ON' state time and a mean decrease of 0.8 hours in 'OFF' state time.
Reduction in Motor Symptoms
Motor symptom severity, measured by the Movement Disorder Society-Sponsored Revision of the Unified Parkinson’s Disease Rating Scale Part III (MDS-UPDRS Part III) in the 'OFF'-medication state, showed a mean reduction of 23 points from baseline in the high-dose cohort at 18 months. The low-dose cohort showed a milder improvement, with a mean decrease of 8.6 points.
Addressing Non-Motor Symptoms
Notably, the higher dose cohort also exhibited trends towards improvements in neuropsychological evaluations, suggesting potential benefits in neuropsychiatric symptoms, cognitive functioning, and frontal-lobe related behaviors. These improvements included measurements indicating enhancements in immediate and delayed memory. According to key opinion leaders interviewed by GlobalData, addressing non-motor symptoms in Parkinson's disease (搜索) represents a significant unmet need, as current treatments are often viewed as inadequate for managing mild cognitive impairment and dementia associated with the disease.
Future Directions
Claire Henchcliffe, MD, chair of the UCI School of Medicine Department of Neurology at the University of California, Irvine and one of the study’s Principal Investigators, stated, "It’s exciting that bemdaneprocel met safety and tolerability criteria at 12 months, and now the 18-month results suggest that these allogeneic cells survive and have potentially positive effects even after discontinuation of immunosuppressants...We should not overinterpret results of a phase I study, but this is a promising step that deserves to be followed up with further studies."
BlueRock Therapeutics (搜索) plans to initiate a Phase II clinical trial to further evaluate bemdaneprocel, with enrollment expected to begin later this year. This trial aims to provide more definitive evidence of the therapy's efficacy and potential to address both motor and non-motor symptoms of Parkinson's disease (搜索).
About Bemdaneprocel
Bemdaneprocel (BRT-DA01) is an investigational allogeneic cell therapy designed to replace dopamine-producing neurons lost in Parkinson's disease (搜索). The therapy involves the stereotactic transplantation of dopaminergic neuronal progenitor stem cells directly into the posterior putamen of individuals with Parkinson's disease. These cells are derived from pluripotent stem cells and have the potential to reform neural networks affected by Parkinson's, thereby restoring motor and non-motor function. Bemdaneprocel is currently under investigation and has not been approved by any health authority for the treatment of any disease or medical condition.