A Clinical Trial of Transplanted Neurons Derived From Induced Pluripotent Stem Cells
In past years, researchers have attempted various forms of cell therapy for Parkinson's disease, with the goal of replacing the population of dopamine generating neurons that are lost to disease processes. Fetal sources of neurons have been used, but since the discovery of cellular reprogramming, the trajectory has been towards the production of neurons from induced pluripotent stem cells. As noted here, this has reached the stage of human clinical trials.
Bayer AG and BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned independently operated subsidiary of Bayer AG, today announced positive top-line results from a Phase I clinical trial of investigational drug, bemdaneprocel (BRT-DA01), a potential first-in-class cell therapy for Parkinson's disease. The trial showed that bemdaneprocel was well-tolerated in all 12 patients in the study to date, with no major safety events. In addition, an assessment of the study's secondary endpoints demonstrated feasibility of transplantation and evidence of cell survival and engraftment in the brain through one year. Based on these results, planning is underway for a Phase II study that is expected to begin enrolling patients in H1 (first half) 2024.
Bemdaneprocel (BRT-DA01), an investigational therapy comprised of dopamine producing neurons derived from pluripotent stem cells, is surgically implanted into the brain of a person with Parkinson's disease. When transplanted, these cells have the potential to reform neural networks that have been destroyed by Parkinson's disease in the hope of restoring motor and non-motor function to patients. The primary objective of the Phase I trial is to assess the safety and tolerability of bemdaneprocel (BRT-DA01) transplantation at one-year post-transplant. The secondary objectives of the trial are to assess the evidence of transplanted cell survival and motor effects at one- and two-years post-transplant, to evaluate continued safety and tolerability at two years, and to assess feasibility of transplantation.
Very interesting. I am not holding my breath, but if it works it would be a sing that transplanted neurons can integrate. That can open the door to other major brain treatments first for stroke and wound victims. And eventually for AD. Of course at some point it would raise questions whether a repaired AD brain holds the same person as the pre-disease one.