Phase I Trial of Autologous Induced Pluripotent Stem Cell-derived Dopaminergic Progenitor Cell Transplantation for Parkinson's Disease
Jeffrey S. Schweitzer, MD, PhD
Summary
The goal of this clinical trial is to assess the safety and tolerability of the surgical transplantation of dopaminergic progenitor cells into the brains of participants with Parkinson's disease. The transplanted dopaminergic cells will be derived from the participant's own skin cells.
Description
This Phase I, open-label clinical trial aims to assess the feasibility and safety of autologous midbrain dopaminergic progenitor cell (mDAP) transplantation for the treatment of Parkinson's disease. mDAPs will be produced for each participant from a fibroblast sample and then transplanted bilaterally into the putamen under general anesthesia. The study will assess the safety and tolerability of the cell transplant procedure through clinical assessments and neuroimaging (CT, MRI and 18F-DOPA PET) over a 2-year follow-up period.
Eligibility
- Age range
- 45–80 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Diagnosis of Parkinson's Disease consistent with the Movement Disorders Society 2015 Parkinson's diagnostic criteria. * Age 45 - 80 years * English proficiency sufficient to understand the consent form and participate in a discussion of risks and benefits * At least 5 years since Parkinson's disease motor symptom onset * Modified Hoehn and Yahr stage 3-4 in "off"-medication state * Motor symptoms responsive to levodopa and/or dopamine agonist, defined as taking at least 300 mg/day of levodopa and exhibiting improvement between "off" and "on" MDS-UPDRS of at least 30% * A…
Interventions
- Biologicalautologous dopaminergic cell implantation
Dopaminergic progenitor cells derived from autologous induced pluripotent stem cells will be injected into the brain in two cohorts of Parkinson's patients, one receiving low dose and the other high dose (4 and 8 million cells, respectively)
Location
- Massachusetts General HospitalBoston, Massachusetts