Grant Award Details

Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia
Grant Number: 
TRAN1-12891
Investigator: 
Disease Focus: 
Neurological Disorders
Stroke
Human Stem Cell Use: 
Embryonic Stem Cell
Award Value: 
$5,925,602
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia
Public Abstract: 

Translational Candidate

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.

Project Objective

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
Statement of Benefit to California: 

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.