Cerebral Palsy Research Network Blog

CPRN Investigator Receives NIH Funding for CP Prevention Trial

Dr. Yvonne Wu, a pediatric neurologist from the University of California San Francisco, received significant funding ($2.3M over the next 12 months) from the National Institute of Neurological Disorders and Stroke (NINDS) to conduct a multi-center clinical trial to prevent cerebral palsy from a specific type of brain injury — hypoxic ischemic encephalopathy — in full term babies. While this study is not a Cerebral Palsy Research Network (CPRN) study, we are excited by the potential that the study holds to reduce the incidence of CP. Dr. Wu is the site Principal Investigator (PI) for Benioff Children’s Hospital/UCSF for CPRN. Her experience in clinical epidemiology and clinical trials makes her an invaluable asset to CPRN and we look forward to the results of this study. Her Co-PI, Dr. Sandra Juul of the University of Washington, is the lead PI on a similar trial for very low birthweight premature babies (see the PENUT trial for more information on this study).

Hypoxic-ischemic encephalopathy (HIE) refers to brain injury resulting from reduced blood and oxygen flow to a baby’s brain near the time of birth. HIE affects up to 12,000 newborns each year in the U.S. Half of affected infants have a bad outcome including death, cerebral palsy and cognitive impairment despite receiving hypothermia, the only available treatment. Cerebral palsy is the most common long term neurodevelopmental impairment in survivors of HIE. Erythropoietin (Epo) is a cytokine with remarkable neuroprotective and neuroregenerative effects demonstrated in animal models of neonatal brain injury. In a phase II trial, we randomized 50 cooled infants to receive Epo or placebo. Infants treated with hypothermia + Epo had less brain injury on MRI than infants who received hypothermia alone. Epo is commercially available, relatively inexpensive, and safe in neonates. We hypothesize that Epo given to cooled infants with moderate/severe HIE will reduce the combined outcome of death or neurodevelopmental impairment from 49 to 33%. To test this hypothesis, we will perform a randomized, double-blind, placebo-controlled trial of Epo therapy in 500 infants with HIE. We anticipate that Epo will confer improved 2-year neurodevelopmental outcome, will be safe, and will decrease brain injury severity as determined by early biomarkers. If successful, we anticipate that Epo treatment has the potential to reduce the burden of CP caused by HIE from 1368 babies to 760 babies per year in the U.S.