Allogeneic hESC-Derived Neural Stem Cells in Acute Neonatal Hypoxic-Ischemic Brain Injury

Funding Type: 
Early Translational III
Grant Number: 
TR3-05688
Investigator: 
ICOC Funds Committed: 
$0
oldStatus: 
Closed
Public Abstract: 
Hypoxic ischemic (HI) brain injury occurs when the brain does not receive enough oxygen during the birthing process. Based on our proof on concept study showing that transplantation of neural stem cells into a model of HI brain injury improves sensorimotor skills, we propose to generate human neural stem cell banks that will qualify for the FDA mandated quality controls and complex testing processes. We will perform studies that will confirm the long-term safety and efficacy of the transplanted cells in HI brain injury model. In the United States, neonatal HI injury is sustained by 1-8 per 1,000 births. In severe cases, the mortality rate is 25-50% with most deaths occurring in the first week of life. The large majority of survivors develop serious complications, such as cerebral palsy. These statistics, costs for care and rehabilitation and lack of therapy indicate that HI brain injury is a current significant unmet medical need in California and US - one that is not represented in CIRM’s current Translation Portfolio. Our proposal involves milestone-driven deliverables to develop a neural stem cell product that may improve the quality of life for these children who demonstrate a deep inner strength when tackling their daily challenges.
Statement of Benefit to California: 
Based on our proof on concept study in which we demonstrated that the transplantation of neural stem cells into a model of hypoxic ischemic (HI) brain injury improves sensorimotor skills, we propose milestone-driven deliverables that will allow us to develop a neural stem cell product for the treatment of children with HI brain injury in California, US and the world. In the United States, neonatal HI brain injury is sustained by 1-8 per 1,000 births. In severe cases, the mortality rate is 25-50% with most deaths occurring in the first week of life. The long-term complications depend on the severity of the lesion, with the large majority of survivors developing serious complications, such as cerebral palsy. These statistics, costs for care and rehabilitation and lack of therapy indicate that HI brain injury is a current significant unmet medical need in California and US - one that is not represented in the CIRM’s current Translation Portfolio. We believe that we have a cellular product that may improve the quality of life for children with HI injury who demonstrate a deep inner strength when tackling their daily challenges. Furthermore, the technology used to create the stem cell banks will have been developed in California and provide a significant beneficial impact on the economy in terms of employment and commercialization of the cellular product.

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