Phase 1 Data Highlights Sustained Efficacy of Cell Therapy Bemdaneprocel in Parkinson Disease


Over an 18-month period, patients demonstrated significant increases in ON time with bemdaneprocel, even after stopping immunosuppressives 1 year into treatment.

 Claire Henchcliffe, MD, chair of the department of neurology at UCI School of Medicine

Claire Henchcliffe, MD

At the 2024 AD/PD Conference, held in Lisbon, Portugal, BlueRock Therapeutics announced positive 18-month data from a phase 1 study assessing bemdaneprocel, an investigational allogenic stem cell derived cell therapy for Parkinson disease (PD). All told, the agent was safe and well tolerated, with treated patients demonstrating improvements in ON and OFF time over that time.1

The multicenter, open-label, non-randomized, non-controlled study featured 12 patients with PD who received surgical transplantation of 1 of 2 different dose levels of bemdaneprocel cells to the post-commissural putamen bilaterally, along with an immunosuppression regimen for 1 year. Using the Hauser Diary, those in the high-dose cohort demonstrated a mean increase of 2.7 hours in good ON time compared with baseline after 18 months. In addition, this group had a mean reduction of 2.7 hours in time spent in the OFF state.

"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," principal investigator Claire Henchcliffe, MD, chair of the department of neurology at UCI School of Medicine, said in a statement.1 "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."

On the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III, measured in the OFF medication state, results showed a mean reduction of 23 points at 18 months in the high-dose group. Effects were less drastic in the low-dose group, with mean decreases of 8.6 points. Those in the low dose cohort demonstrated a mean improvement of 0.2 hours in ON time compared with baseline and a corresponding mean decrease of 0.8 hours in OFF time.

Between the 2 cohorts, those on high-dose bemdaneprocel (n = 7) received a dose of 2.7 million cells per putamen while those in the low-dose group (n = 5) received a dose of 0.9 million cells per putamen. The original 12-month data of the study, presented at the 2023 International Congress of Parkinson’s Disease and Movement Disorders, demonstrated the efficacy and safety of the treatment. At 1 year, those in the high-dose cohort showed a reduction of 13.0 points in MDS-UPDRS-III scores compared with baseline.

READ MORE: NodThera’s NT-0796 Shows Promise as Parkinson Disease Treatment in Phase 1b/2a Trial

Less pronounced, the low-dose cohort showed a reduction of 7.6 points after 1 year. Imaging analysis using 18F-DOPA PET, a technique used to visualize and assess dopaminergic activity, showed evidence of cell survival and engraftment in both the high and low dose cohorts. At the 1-year follow-up, no serious adverse events (AEs) attributable to bemdaneprocel were observed; however, investigators did note 2 unrelated serious AEs of seizure attributed to the surgical procedure and 1 COVID case. Both of these resolved without sequelae.

"We are on the leading edge in the research for new treatment options for Parkinson’s disease as bemdaneprocel, the most clinically advanced pluripotent stem derived cell therapy candidate to date for this disease, continues to show positive trends,” Christian Rommel, member of the executive committee of Bayer’s Pharmaceuticals Division and head of Research and Development, said in a statement.1 "There are good reasons to be optimistic about these early data, and we are excited to move to phase II later this year."

1. BlueRock Therapeutics phase 1 clinical trial for Parkinson’s disease continues to show positive trends at 18 months. News release. BlueRock Therapeutics. March 6, 2024. Accessed March 14, 2024.
2. Henchcliffe C, Sarva H, Lozano A, et al. Dopaminergic neuronal cell therapy for Parkinson’s disease: results from a phase 1 study of bemdaneprocel. Presented at 2023 MDS Congress; August 27-31; Copenhagen, Denmark. Abstract 65.
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