New Autism Study Safety Data to be Presented at the Translational Neuroscience Cell Symposium

SAN BRUNO, CA – November 14, 2014 – Cord Blood Registry® (CBR®) announced today that Dr. Michael G. Chez, principal investigator at Sutter Neuroscience Institute, will present data from the first ever FDA-registered study on the safety of autologous umbilical cord blood (AUCB) stem cells in treating autism at the Translational Neuroscience: Bridging the Gap Between Basic Research Discoveries and Clinical Applications Cell Symposium on November 14 in Arlington, Virginia.


This single-center, randomized, placebo-controlled, blinded study is designed to assess safety, language and behavior outcomes after an infusion of AUCB stem cells from cord blood stored by CBR client families. Results to date show that it is feasible and safe to perform this type of study in the autism population. Efficacy results will be reported at a future medical congress.


"Families who have children with autism are very interested in the promise of stem cell therapy and this is the first trial in the U.S. aimed to assess the safety and efficacy of using a child's own cord blood as a potential treatment," said Dr. Michael G. Chez, principal investigator. "I'm excited to present preliminary safety data on the first 18 subjects who have received both cord blood and placebo infusions. We have shown that this type of treatment is not only safe, but also possible to do within this patient population. I'm looking forward to the completion of the study next year when data will be unblinded and analyzed further."


"As the leader in the cord blood industry, CBR recognizes the importance of clinical research and is committed to investigating the potential use of newborn stem cells in regenerative medicine," said Geoff Crouse, President and Chief Executive Officer of Cord Blood Registry. "We are excited to support Dr. Chez's research at Sutter Neuroscience Institute and look forward to future results."


About the Trial

This study enrolled 30 subjects aged 2-7, both male and female, who were diagnosed with Autistic Disorder according to the DSM-IV-TR criteria and ADOS. The duration of participation for each study subject was approximately 55 weeks, including a screening visit over a period of approximately six weeks, one visit for baseline testing, one day for infusion of autologous umbilical cord blood (AUCB) stem cells containing a minimum of 10 million cells per kilogram or placebo control (saline) followed by 24 weeks of follow-up. A second baseline visit was conducted at week-24, at which time subjects were crossed over to receive either a stem cell infusion or placebo. Both groups were tested again 24-weeks after infusion. Results are blinded as to the infusion sequence. More information on this trial is available at www.clinicaltrials.gov (NCT01638819).