Study of stem cell-derived Parkinson’s therapy yields positive results after 1 year

Key takeaways:

• PET scans showed evidence of cell survival and engraftment in both cohorts.
• The high-dose group showed greater symptom improvement compared with the low-dose group at 1 year.

Positive data from a phase 1 clinical trial for bemdaneprocel, a stem cell-derived investigational therapeutic for treating Parkinson’s disease, have been announced by its manufacturer.

The data were presented at the International Congress of Parkinson’s Disease and Movement Disorders in Copenhagen.

According to a joint release, the study met its primary objective of demonstrating safety and tolerability in all 12 participants in the study’s low and high dose cohorts, with no serious adverse events reported related up to 1 year. In addition, 18F-DOPA positron emission tomography (PET) imaging scans — which are employed to visualize and assess dopaminergic activity in PD — showed evidence of cell survival and engraftment in both the low and high dose cohorts.

“The need for new therapies to help patients struggling with Parkinson’s disease is clear,” Ahmed Enayetallah, senior VP and head of development at BlueRock Therapeutics, a clinical stage cell therapy company and a subsidiary of Bayer AG, said in the release. “We are excited to be sharing the results and look forward to advancing bemdaneprocel to the next stage of clinical testing.”

According to the release, both cohorts showed improvements in the secondary exploratory clinical endpoints as measured by the MDS-Unified Parkinson’s Disease Rating Scale Part III (MDS-UPDRS Part III) and the Hauser Diary, which assess PD severity in motor symptoms.

In the high dose cohort, MDS-UPDRS Part III measured in the “off” medication state logged a reduction of 13 points compared with baseline compared with a reduction of 7.6 points for the low dose group. For Hauser Diary assessments, those in the high dose cohort showed an improvement of 2.16 hours in the “on” state without troubling dyskinesia compared with baseline, while “off” time decreased by 1.91 hours, compared with 0.72 hours “on” and 0.75 hours “off” for the low dose cohort.

“The data from this Phase I open label study are extremely encouraging,” 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, said in the release. “Meeting the study’s primary objective for safety and tolerability along with initial improvements seen in clinical outcomes represents a great step forward.”

Based on the data, Bayer and BlueRock plan to commence a phase 2 study that is expected to begin enrollment in the first half of 2024.