Phase II Clinical Trial Data Establishes Effectiveness of using a Child's Own Cord Blood to Treat Cerebral Palsy

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Key Discoveries in the Therapeutic Application of Cord Blood: Improvement of Motor Function in Children with Cerebral Palsy

WALTHAM, Mass.–(BUSINESS WIRE)– Research published in Stem Cells Translational Medicine shows encouraging developments related to using a child’s own cord blood to treat childhood brain injury known as cerebral palsy (CP).

The research from a Phase II clinical trial indicates that patients with CP who were treated with an infusion of an adequate dose of their own cord blood, which had been collected and stored at birth, saw a significant improvement in their motor function. Of the 63 children receiving treatment, those who were given a dose higher than 20 million cells per kilogram showed improvements in motor function measured on a validated scale for assessing motor function in children with CP called the Gross Motor Function Measure (GMFM-66).

“This is the first demonstration of a therapy producing durable improvements in motor function in young children with CP. We are very encouraged by these results. Our research has indicated that autologous cord blood infusions are feasible, safe and easily administered in the outpatient setting,” said Joanne Kurtzberg, MD, Director, Carolinas Cord Blood Bank and Pediatric Blood and Marrow Transplant Program; Chief Scientific Officer, Robertson Clinical and Translational Cell Therapy Program, Duke University, who led the clinical trial.

The purpose of this Phase II trial was to determine the efficacy of using a child’s own (autologous) cord blood for the treatment of pediatric patients with cerebral palsy. The primary measure of efficacy was a significant change in standardized measure of motor function over what would have been expected at one year without the treatment. Further details can be found here.

“Before our son Patrick’s cord blood treatment, things were so overwhelming for him and our family. Patrick struggled with therapy, school and the normal daily activities you take for granted,” said GiGi and Kevin Rooney, the parents of a child who participated in the study. “Today, after his cord blood treatment, Patrick has great stamina and endurance and the confidence to try new things. Having his cord blood and being in this study has tremendously impacted our lives for the better and we are truly grateful.”

These findings in children with CP follow the publication in April 2017 of results from a Phase I trial testing the safety of using a child’s own cord blood to treat Autism Spectrum Disorder (ASD) published in Stem Cells Translational Medicine. Duke continues to study the use of cord blood to treat children with Autism Spectrum Disorder in a Phase II Clinical Trial using both autologous and allogeneic cord blood, and recently completed a Phase I study in children with CP who were treated with cord blood from a sibling.

“For more than 20 years, ViaCord has collaborated on projects to determine the clinical utility of cord blood stem cells. These initial findings mark an exciting development in the potential use of cord blood stem cells for treating Cerebral Palsy, and a significant step in establishing the role of cord blood beyond transplant medicine and into regenerative medicine, including Autism,” said Morey Kraus, Chief Scientific Officer, ViaCord. “We are proud that ViaCord families were able to participate in this historic study by having access to their own cord blood, and we look forward to helping many more families.”

ViaCord, PerkinElmer’s (NYSE:PKI) cord blood and cord tissue preservation business, has preserved the umbilical cord stem cells of more than 350,000 newborns and released over 350 cord blood units for treatment and clinical research studies. It has released more than 200 cord blood units for use in the treatment of nearly 80 diseases including acute lymphoblastic leukemia and sickle cell disease and more than 150 cord blood units for use in clinical research studies including this study for Cerebral Palsy and Autism Spectrum Disorder.

Please visit www.viacord.com for more information. Follow ViaCord on Twitter @ViaCord.

About PerkinElmer

PerkinElmer is a global leader committed to innovating for a healthier world. Our dedicated team of 9,000 employees worldwide are passionate about providing customers with an unmatched experience as they help solve critical issues especially impacting the diagnostics, discovery and analytical solutions markets. Our innovative detection, imaging, informatics and service capabilities, combined with deep market knowledge and expertise, help customers gain earlier and more accurate insights to improve lives and the world around us. The Company reported revenue of approximately $2.1 billion in 2016, serves customers in more than 150 countries, and is a component of the S&P 500 Index. Additional information is available through 1-877-PKI-NYSE, or at www.perkinelmer.com.

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For PerkinElmer
Elizabeth Tripp, 646-556-9324
etripp@apcoworldwide.com

Source: PerkinElmer

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