Long-term Cell Restorative Treatment for Parkinsonís Disease
Therapeutic Pipeline Program, 2016
Study Rationale: † † † † † † † † ††
Cell replacement therapies for Parkinsonís disease (PD) propose to replace the lost dopaminergic neurons in the brain. Transplanted fetal dopamine neurons have provided remarkable recovery in some PD patients and can survive and function for decades. More recently, the field has evolved from this innovative but complex fetal dopamine cell transplantation method toward a potential scalable method that depends on stem cell-derived dopamine neurons. Induced pluripotent stem cells (iPSCs) as a cell source allows for the use of the patientís own cells and thus eliminate the need for immunosuppression.
We will test the safety and efficacy of increasing doses of transplanted iPSC-derived midbrain dopamine neurons in a relevant model of PD.
We will generate iPSCs from non-human primates and differentiate toward a midbrain dopamine neuron phenotype. We will perform bilateral transplantation of midbrain dopamine neurons at different doses into the putamen of parkinsonian models, and determine functional recovery and dopamine neuron survival over one to two years.†
Impact on Diagnosis/Treatment of Parkinsonís Disease: † † † † † ††
These are essential pre-clinical studies for advancing iPSC-derived dopamine neuron cell therapy toward clinical testing. These safety and efficacy studies will utilize the therapeutic prepared in the same manner as will be used in humans and use the same functional readouts as will be used clinically in human PD patients.
Next Steps for Development:
Upon successful outcome of these experiments, the next stage of development is a first-in-man clinical trial.†
Professor of Neurology and Neuroscience at Harvard Medical School
Director of the Neuroregeneration Research Institute at McLean Hospital
Location: Boston, Massachusetts, United States
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