Bayer is advancing its efforts in cell and gene therapy for Parkinson's disease, with two of its subsidiaries initiating clinical trials for novel treatments. BlueRock Therapeutics has dosed the first patient in a Phase 1 trial of its cell-based therapy, DA01, while Asklepios BioPharmaceutical (AskBio) has begun recruiting for a Phase 1b trial of its gene therapy. These trials represent a significant step towards potentially halting or reversing the progression of Parkinson's disease by regenerating dopamine-producing neurons in the brain.
BlueRock's Cell-Based Therapy: DA01
BlueRock's DA01 involves the surgical transplantation of pluripotent stem cell-derived dopaminergic neurons into the putamen, a brain region affected by neuron loss in Parkinson's disease. The Phase 1 trial will enroll 10 patients who will receive immunosuppressant drugs to prevent rejection of the transplanted cells. The study will monitor the safety, tolerability, survival of the cells, and impact on Parkinson's symptoms over two years, with final results expected in November 2023. The first patient was treated at Memorial Sloan Kettering Cancer Center in June, marking the first trial in the US to study pluripotent stem cell-derived dopaminergic neurons in Parkinson's patients.
The FDA has granted fast-track designation to DA01, which could expedite its development and review. This designation allows for more frequent interactions with the FDA and a potentially shorter six-month review period.
AskBio's Gene Therapy Approach
AskBio's gene therapy utilizes an adeno-associated virus (AAV) vector to deliver a gene for human glial cell line-derived neurotrophic factor (GDNF) into neurons within the putamen. GDNF is a growth factor that has demonstrated the ability to stimulate the regeneration of midbrain neurons in animal studies. The Phase 1b trial will involve 12 patients with early- and late-stage Parkinson's disease, each receiving a single dose of the gene therapy. Participants will be followed for 18 months to assess the therapy's effects on dopaminergic neurons, motor, and non-motor symptoms. Safety will be monitored for five years, with initial data expected by the end of 2022.
The Need for Novel Parkinson's Treatments
Parkinson's disease is the most common neurodegenerative movement disorder, affecting over 10 million people worldwide. Current treatments primarily manage symptoms but do not address the underlying loss of dopamine-producing neurons. Bayer's two-pronged approach, through BlueRock and AskBio, aims to provide disease-modifying therapies that could significantly improve the lives of Parkinson's patients.
Wolfram Carius, head of cell and gene therapy at Bayer, stated that the potential of these clinical candidates to treat Parkinson's disease could be immense, offering the possibility to stop and reverse this degenerative disease and address the high unmet medical need.
Other Players in the Field
Other companies are also exploring cell and gene therapies for Parkinson's disease. Voyager Therapeutics plans to initiate trials of its candidate later this year. Prevail Therapeutics is developing a gene therapy for Parkinson's caused by mutations in the GBA1 gene, and Aspen Neuroscience is working on a personalized stem cell therapy using a patient's own cells.
