Bayer AG has announced that its clinical stage biopharmaceutical subsidiary, BlueRock Therapeutics, has administered the first DA01, its pluripotent dopaminergic neurons derived from stem cells. BlueRock is attempting to bring back motor function in Parkinson’s patients and reverse the degenerative process by restoring the lost nerve supply to the brain. Patients enrolled on BlueRock’s Phase 1 study (NCT04802733) clinical trial will undergo surgical transplantation of the dopamine-producing cells into a large structure located within the brain called the putamen which is a complex feedback loop that prepares and aids in movement of the limbs. Evaluation of the safety and tolerability of DA01 cell transplantation one year after transplant is the primary objective of this study. Assessment of the evidence of transplanted cell survival and motor effects at one year and at two years after transplant, safety and tolerability evaluations after two years, and an assessment of the viability of transplantation are the secondary aims.
A the same time as this Phase 1 clinical study is progressing, Bayer’s clinical-stage adeno-associated virus gene therapy company Asklepios BioPharmaceutical is in the middle of a Phase 1b clinical study (NCT04167540) also seeking to provide advanced therapies for Parkinson’s disease. This company is using an AAV that delivers human glial cell line-derived neurotrophic factor gene to the neurons in the putamen, resulting in expression and secretion of GDNF protein in brain regions impacted by Parkinson’s disease. The primary objective at this point is to evaluate patients to assess safety and preliminary efficacy.
“For the first time, it might be possible to stop and reverse this degenerative disease and truly help patients with their high unmet medical need“
Head of Bayer’s Cell and Gene Therapy, Wolfram Carius, said: “The potential of BlueRock and AskBio’s clinical candidates to treat Parkinson’s disease could be immense. For the first time, it might be possible to stop and reverse this degenerative disease and truly help patients with their high unmet medical need. The start of clinical trials represents the beginning towards a truly breakthrough treatment option to dramatically improve the lives of patients.”