Cell and Gene Therapies for Parkinson Disease Moving Forward

In an open-label phase 1 clinical study (NCT04802733), researchers successfully administered the first dose of pluripotent stem cell-derived dopaminergic neurons (DA01; Blue Rock Therapeutics, a subsidiery of Bayer Therapeutics, Hanover, NJ) to a participant with Parkinson disease (PD). The cells are surgically transplanted into the putamen with the aim of reinnervation to reverse the neurodegeneration of PD and potentially improve motor function. The objects of the study are to evaluate the safety and tolerability of the procedure and transplant at 1, 2, and 3 years; efficacy for treatment of motor symptoms will also be evaluated at years 2 and 3. This study is currently enrolling with a planned cohort of 10 participants.

In a phase 1b study (NCT04167540), gene therapy is being delivered to the putatmen of people with PD. The gene for human glial cell line-derived neurotrophic factor (GDNF) (AskBio, Triangle Park, NC, a subsidiery of Bayer Therapeutics) is delivered on an adeno-associated viral (AAV) vector. In animal models, this technique has resulted in expression and secretion of GDNF protein, neural regeneration, and improved motor function. This study is fully enrolled with 10 participants

“The potential of BlueRock’s and AskBio’s clinical candidates to treat PD could be immense,” said Wolfram Carius, head of cell and gene therapy, Bayer. “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.”