Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease

Subjects will undergo surgical transplantation of the dopamine-producing cells under general anesthesia into a part of the brain called the putamen. Subjects then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of cell survival (using MRI and PET scans of the brain), and effect on Parkinson's disease symptoms are assessed for 2 years post-transplant.

Inclusion criteria

• Age 50-78 years old (Canada)
• Age 60-78 years old (United States)
• Diagnosis of Parkinson's Disease made between 3 to 20 years ago
• Taking levodopa, but with complications of therapy such as wearing off and/or dyskinesia
• Able to participate in all study visits and evaluations, including brain MRI and PET scan
• Existence of a study partner who may act as potential surrogate over long term for ongoing consent

Exclusion criteria

• Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis or other neurodegenerative diseases such as Alzheimer's disease
• Prior Deep Brain Stimulation , lesion therapy, or gene therapy for PD
• Prior surgical or radiation therapy to the brain or spinal cord
• Any medical condition resulting in high risk of immunosuppressive drugs, including any active infectious disease
• Inability to temporarily stop anti-platelet agents or other anti-coagulant medications without serious risk
• Previous or currently active malignant disease within the past 5 years, except basal cell carcinoma or in situ uterine cervical carcinoma that have been treated
• Severe obesity (>350 lbs) or any condition that prevents use of PET/MRI
• Pregnancy or breastfeeding
• Contraindication to surgery or general anesthesia
• In the opinion of the investigator, any other condition regarded as making subject unsuitable for trial