Grant Award Details
Grant Application Details
- Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia
Human induced pluripotent stem cell-derived glial enriched progenitors
Area of Impact
Vascular dementia and white matter stroke, addressing a current bottleneck of poor scale up for existing cell differentiation protocols.
Mechanism of Action
Preliminary in vivo efficacy studies indicate that the MOA is in the promotion of new connections in the brain after white matter stroke, termed axonal sprouting. Axonal sprouting is uniquely present in transplantation of hiPSC-GEPs, and not in transplantation of the precursor stage to hiPSC-GEPs, which is hiPSC-NPCs. Astrocytes promote the formation of new connections in the brain and axonal sprouting, by directly enhancing axonal growth and by promoting the synapses of these growing axons.
Unmet Medical Need
There is no therapy for vascular dementia. The brain responds to this disease, and initiates a reparative response, but is blocked from fully engaging this response. This therapy addresses this condition by delivering a stem cell-derived product that enables recovery in vascular dementia.
Pre-IND meeting with FDA
Major Proposed Activities
- Pharmacology/Toxicology – Confirmatory in vivo pharmacology studies and pilot in vivo tumorigenicity study
- CMC – Cell therapy product generation, formulation and qualification of manufacturing process
- Clin/Reg- Development of clinical trial documents and preparation for pre-IND meeting
This research will develop a therapy for a disease with no treatment, vascular dementia, that is common and devastating in its consequences. The intellectual property for this therapy is held by a State of California public university (UCLA) and commercialization will directly benefit the State of California.