Funded Studies
Study Rationale:
One promising approach for the treatment of Parkinson’s is to replace the cells that have died in the disease through transplantation of healthy cells into the brain. However, this approach has faced limitations including poor survival of the transplanted cells. We have recently shown that biomaterials (materials specifically engineered to interact with living systems for therapeutic purposes) have the potential to dramatically improve cellular brain repair for Parkinson’s. Specifically, when the brain cells were encapsulated in a growth factor-enriched biomaterial before transplantation into model brain, the survival of the cells was dramatically improved, and this enhanced brain repair and recovery of movement control.
Hypothesis:
We aim to determine if growth factor-enriched biomaterials can improve cellular brain repair for Parkinson’s when using brain cells generated from induced pluripotent stem cells (iPSCs).
Study Design:
We will convert iPSCs into premature dopaminergic neurons. These will be transplanted into Parkinson’s model brain with or without the biomaterial, and with or without growth factor enrichment. We will assess the ability of the cells to repair the brain and restore movement control.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
This approach has the potential to dramatically improve movement control in people with Parkinson’s by repairing and reconstructing the brain circuits that degenerate in the condition. In these patients, the reconstruction and restoration could provide benefits lasting decades.
Next Steps for Development:
Ultimately, if we can show that the biomaterials can improve the brain repair and movement control provided by the iPSC-derived dopamine neurons, then future clinical trials of these cells should move towards including biomaterials in the transplantation approach as this could lead to a dramatic improvement in the outcome for patients.