Stem Cell Therapy BRT-DA01 Demonstrates Positive Safety, Feasibility in Early-Stage Study of Parkinson Disease
At 1 year, treatment with the investigational cell therapy was well tolerated with no major safety issues and showed evidence of cell survival and engraftment.
Ahmed Enayetallah
Bayer AG and BlueRock Therapeutics recently announced positive phase 1 (NCT04802733) findings assessing its neural stem cell therapy bemdaneprocel, also known as BRT-DA01, in individuals with Parkinson disease (PD). All told, treatment with the therapy was safe and tolerable, with observed feasibility of transplantation and evidence of cell survival and engraftment in the brain after 1 year.1
Detailed trial data on the primary and secondary end points are expected to be presented at the 2023 International Congress of Parkinson’s Disease and Movement Disorders in Copenhagen from August 27-31. Based on the positive findings, the companies are planning a phase 2 study that is expected to begin enrolling patients in the first half of 2024.
"We are on a mission to harness the power of cell therapy with the aim to help people with Parkinson’s disease regain control of their lives by restoring the functions that they have lost to this disease,” Ahmed Enayetallah, senior vice president and head of development at BlueRock, said in a statement.1
"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."
Bemdaneprocel, an investigational agent, is comprised of dopamine producing neurons derived from pluripotent stem cells (PSC). When transplanted, these cells have the potential to reform neural networks that have been destroyed by PD in the hope of restoring motor and non-motor function to patients. In the trial, patients underwent surgical transplantation of the dopamine-producing cells under general anesthesia into the putamen and continued to take medicines that partially suppress their immune system for 1 year.
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The trial, which featured 12 individuals with PD, was intended to assess safety and tolerability of the cell therapy, with secondary objectives that included cell survival, changes in motor function, changes in waking hours in OFF state, and continued safety and tolerability. Eligible patients were between 50 and 78 years of age, had a diagnosis of PD made between 3 to 20 years ago, and were currently taking levodopa.
"At Bayer, we are committed to advancing cell and gene therapy innovations for patients with Parkinson’s disease, a neurodegenerative disorder with debilitating effects on people’s lives for which there is currently no cure and only limited treatment options," Christian Rommel, member of the Executive Committee of Bayer’s Pharmaceuticals Division and head of Research and Development, said in a statement.1 "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."
Aside from PSCs, embryonic and somatic stem cells have been used as potential approaches to treat PD. The first advantage of induced PSC approaches is that lines can be established without the sacrifice of human embryos, removing a large ethical obstacle of human stem cell treatments. Induced PSCs also permit human leukocyte antigen matches in patient-specific treatments, effectively reducing the severity of post-operational immunosuppressants. Histocompatability has additionally shown a reduced immune response of lymphocytes and microglia as well as increased cell survival in induced PSC transplantation of dopaminergic neurons in primate studies.
