Cord Blood Registry is conducting a Phase 2 clinical trial at University of Texas Health Science Center to evaluate the safety and efficacy of delivering stem cells intravenously to cerebral palsy patients. The goal is to recruit 30 children with cerebral palsy, aged two to ten years, and administer either autologous cord blood stem cells (collected at the time of birth) or autologous bone marrow stem cells (collected from a bone marrow harvest). Five patients from either group will receive a placebo at baseline but will be given the option to receive stem cell treatment after one year. Follow-ups at 6, 12, and 24 months will be conducted at the University of Texas Health Medical School and Children’s Memorial Hermann Hospital.
During follow-ups, patients will be evaluated for treatment safety through pulmonary and hepatic function, neurological status, and hemorrhagic or ischemic lesions; they will be evaluated for treatment efficacy through MRI analysis of white matter tracts correlated with motor function studies. The study was initiated in November 2013 and is projected to end in November 2015. As of January 21, enrollment for the bone marrow harvest group was met.
This trial, like all trials, began in the laboratory. “There is preclinical data indicating that the ongoing neuroinflammatory response is a driver of further injury in cerebral palsy so the hope is to reduce this neuroinflammation,” stated Dr. Charles Cox, principal investigator of the study and a professor of Pediatric Surgery at UTHMS. The inflammation seen in cerebral palsy is caused by abnormal brain development and damage to the motor nervous system. It is thought that infusing a patient’s own stem cells may trigger repair mechanisms to alleviate the damage. “Our goal is to break the cycle of inflammation and injury,” said Dr. Cox in material from Cord Blood Registry. This goal is good news for the 1 in every 323 children in the United States who has been diagnosed with cerebral palsy, as the prevalence of the disease has increased over time.