Project description
Advancing stem cell therapy for preterm babies born with brain injuries
Neonatal encephalopathy is a term encompassing any pathology of the central nervous system caused by brain injury in new-born babies. Encephalopathy of prematurity refers to those pathologies specifically associated with preterm birth. A remarkable spectrum of developmental events occurs during the last half of pregnancy. Not surprisingly, brain abnormalities in survivors of preterm birth display a complicated combination of developmental and non-developmental disturbances that have chronic impact on babies’ survival and quality of life. For example, about one fourth of all cases of cerebral palsy are associated with preterm birth. The ambitious five-year EU-funded PREMSTEM project brings together 15 partners from Europe and Australia to speed the development of stem cell therapy for preterm infants. Success will be life-changing for millions of babies and their families.
Objective
This project will provide new knowledge regarding the best regimen, timing, dose, and route of administration of human mesenchymal stem cells (H-MSC) for regenerating the brain damage in infants born preterm; born before 37 of a typical 40 weeks of gestation. Over 15 million babies are born preterm every year it is a leading cause of death and permanent disability, often due to brain damage. The H-MSC that we will comprehensively screen are FDA-approved and in the process of being produced in a GMP grade, making them suitable for future clinical trials in human preterm born infants. These H-MSC will be evaluated in a large number of rodent models mimicking the key insults and brain damage observed in human preterm infants who will develop later motor, cognitive, and behavioral deficits. We will also apply theseH-MSC in two sheep models of injury relevant to brain damage associated with preterm birth. These large animal trials are a key step before initiating clinical trials in this high risk human population. Additional Workpackages will address the mechanisms underlying the regenerative effects of these H-MSC. This project addresses the call text by value adding to current knowledge of stem cell types and applying it to a large patient group with an unmet clinical need – infants with encephalopathy of the preterm born infant. Steps on the innovation chain targeted – preclinical research, proof of concept and to a lesser degree characterization of regenerative mechanisms.
Programme(s)
Funding Scheme
RIA - Research and Innovation actionCoordinator
75654 Paris
France