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(Supplement) Cellular Brain Repair for Parkinson’s Disease: Preclinical Therapeutic Validation of Biomaterial-supported Cell Transplants Coupled with Immunosuppression

This grant builds upon the research from a prior grant: Biomaterial-enhanced Cellular Brain Repair for Parkinson's Disease

Study Rationale: Although transplantation of stem cell-derived brain cells is a promising therapeutic approach for Parkinson’s disease (PD), only a small fraction of the cells that are transplanted actually mature into the brain cells that are required to repair the Parkinsonian brain. We recently found that this efficiency can be enhanced 15-fold if we use a biomaterial to support the cells in the brain after transplantation. However, we have not yet found a way for this biomaterial-boosted brain repair to work on conjunction with the type of immunosuppressive drug therapy that most individuals would receive after transplantation therapy.

Hypothesis: In this project, we want to find ways for the biomaterial-enhanced cell transplantation to improve brain repair and restore movement control to Parkinsonian rats that are receiving immunosuppressive drug therapy.   

Study Design: To do this, we will first compare how the brains of Parkinsonian rats react to the biomaterial-aided cell transplant — both with and without immunosuppression. Based on these results, we will then investigate different approaches to immunosuppression to find one that permits the biomaterial to improve the maturation of the stem cell-derived brain cells. Finally, we will fully test the ability of biomaterial-enhanced cell transplantation to improve brain repair and restore movement control to immunosuppressed Parkinsonian rats.

Impact on Diagnosis/Treatment of Parkinson’s disease: If implantation in biomaterial dramatically improves the repair provided by stem cell-derived brain cells in immunosuppressed rats, the same approach should also enhance the therapeutic potential of these cells in people with PD, improving their movement control, reducing their need for drugs (with their associated side effects) and enhancing their quality of life.

Next Steps for Development: To progress this to clinical testing, we would need to engage with relevant stakeholders to initiate the process for regulatory approval and clinical development of this therapeutic approach.


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