Cord Blood Registry (CBR) from AMAG Pharmaceuticals have announced research recently published in the journal Stem Cells Translational Medicine found that children with cerebral palsy who received an infusion of their own neo-natal cord blood (collected & stored at birth) saw improved motor function and brain connectivity one year after treatment, when receiving a high cell dose (>20 million total nucleated cells per kilogram). The prospective, randomized, double-blind, placebo-controlled phase 2 clinical study examining the efficacy of autologous cord blood in treating children with cerebral palsy was led by researchers at Duke University.

“The study results are compelling for further study of the use of autologous blood cord infusions in children with cerebral palsy,” said Joanne Kurtzberg, M.D., the study’s principal investigator and director of the Carolinas Cord Blood Bank and The Duke Pediatric Blood and Marrow Transplant Program. Dr. Kurtzberg is also chief scientific officer of the Robertson Clinical and Translational Cell Therapy Program at Duke University.

Following a phase 1 study which showed the use of autologous cord blood to be safe in children with neurologic disorders, the aim of the phase 2 trial was to determine efficacy using autologous cord blood for treatment of cerebral palsy in pediatric patients ages one through six. Improvement in the study was measured using the Gross Motor Function Measure (GMFM-66), a standardized assessment to evaluate motor function (e.g. walking ability).  Whole brain connectome (neural mapping) exploratory analysis was also performed using magnetic resonance imaging (MRI) of the brain. Sixty-three children were randomized into a treatment (n = 32) or placebo (n = 31) group. Of the 32 children receiving treatment, those who were given a dose greater than 20 million total nucleated cells per kilogram showed statistically significant, and clinically meaningful improvements in motor function measured on a validated scale for assessing motor function in children with cerebral palsy compared to children who received lower doses of cord blood (p=0.05) or the placebo (p=0.04). Additional analysis showed that children who received higher cord blood cell doses also showed a greater increase in normalized whole brain connectivity compared to children who received lower cell doses. While these results are encouraging, limitations of the study include a small sample size, heterogeneity of the participants, and greater than historically observed gains in motor function observed in all groups.

More than one-third of the children in this study—or 22 out of 63 total study participants—had their cord blood preserved at birth with CBR. CBR is one of the only private cord blood banks that collects health status information from families storing in order to facilitate participation in clinical research. CBR aims to advance families’ access to new clinical applications of cord blood stem cells for conditions that currently have no cure through regenerative medicine and is committed to advancing the science on the use of cord blood stem cells. The results of this study highlight the need for continued research of autologous cord blood infusions to treat cerebral palsy. CBR is partnering with research institutions to support FDA-regulated clinical trials investigating the use of cord blood in regenerative medicine applications across a wide variety of conditions, including autism, acquired hearing loss, and two clinical trials on cerebral palsy at Augusta University and The University of Texas Health Sciences Center.

“We are excited about these results from this early trial regarding the potential for autologous cord blood in children with cerebral palsy and the impact it may have on both the participants and their families,” said Heather Brown, vice president, Scientific and Medical Affairs, CBR. “It is encouraging to see that the results of this trial suggest that in some of the children with cerebral palsy, when dosed =2 × 107 cells per kg, an infusion of a child’s own cord blood improves whole brain connectivity and motor function.”