Stem cell derived Parkinson’s therapy shows promise

Degeneration of dopaminergic neuron in Parkinson's disease

A stem cell derived investigational therapy for treating Parkinson’s disease has shown promise in early clinical trials.

BlueRock Therapeutics, a subsidiary of Bayer, has announced details of the positive data from a Phase I clinical trial for bemdaneprocel (BRT-DA01).

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

The study met the 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 (SAEs) reported related to bemdaneprocel through one year.

In addition, 18F-DOPA PET imaging scans demonstrated evidence of cell survival and engraftment in both low and high dose cohorts. 18F-DOPA PET imaging is a neuroradiological technique used to visualise and assess dopaminergic activity in Parkinson’s disease.

Secondary exploratory clinical endpoints improved in both cohorts, with participants in the high dose cohort showing greater improvement, as assessed by the MDS-Unified Parkinson’s Disease Rating Scale Part III (MDS-UPDRS Part III) and the Hauser Diary, which are tools used to assess Parkinson’s disease severity in motor symptoms.

“The data from this Phase I open label study are extremely encouraging,” said 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. “While this is a small open-label study, meeting the study’s primary objective for safety and tolerability along with initial improvements seen in clinical outcomes represents a great step forward. The hope now is that these trends continue and translate into meaningful benefit for people with Parkinson’s disease in controlled clinical trials.”

Based on these results, planning is underway for a Phase II study that is expected to begin enrolling patients in H1 (first half) 2024.

Edited by Diana Spencer, Senior Digital Content Editor, Drug Discovery World

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