Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial

Jessica M. Sun, Allen W. Song, Laura E. Case, Mohamad A. Mikati, Kathryn E. Gustafson, Ryan Simmons, Ricki Goldstein, Jodi Petry, Colleen McLaughlin, Barbara Waters-Pick, Lyon W. Chen, Stephen Wease, Beth Blackwell, Gordon Worley, Jesse Troy, Joanne Kurtzberg

Research output: Contribution to journalArticlepeer-review

99 Scopus citations


Cerebral palsy (CP) is a condition affecting young children that causes lifelong disabilities. Umbilical cord blood cells improve motor function in experimental systems via paracrine signaling. After demonstrating safety, we conducted a phase II trial of autologous cord blood (ACB) infusion in children with CP to test whether ACB could improve function (ClinicalTrials.gov, NCT01147653; IND 14360). In this double-blind, placebo-controlled, crossover study of a single intravenous infusion of 1–5 × 10 7 total nucleated cells per kilogram of ACB, children ages 1 to 6 years with CP were randomly assigned to receive ACB or placebo at baseline, followed by the alternate infusion 1 year later. Motor function and magnetic resonance imaging brain connectivity studies were performed at baseline, 1, and 2 years post-treatment. The primary endpoint was change in motor function 1 year after baseline infusion. Additional analyses were performed at 2 years. Sixty-three children (median age 2.1 years) were randomized to treatment (n = 32) or placebo (n = 31) at baseline. Although there was no difference in mean change in Gross Motor Function Measure-66 (GMFM-66) scores at 1 year between placebo and treated groups, a dosing effect was identified. In an analysis 1 year post-ACB treatment, those who received doses ≥2 × 10 7 /kg demonstrated significantly greater increases in GMFM-66 scores above those predicted by age and severity, as well as in Peabody Developmental Motor Scales-2 Gross Motor Quotient scores and normalized brain connectivity. Results of this study suggest that appropriately dosed ACB infusion improves brain connectivity and gross motor function in young children with CP. Stem Cells Translational Medicine 2017;6:2071–2078.

Original languageEnglish
Pages (from-to)2071-2078
Number of pages8
JournalStem cells translational medicine
Issue number12
StatePublished - Dec 2017

Bibliographical note

Funding Information:
The authors report grant funding from The Julian Robertson Foundation (PI: J.K.), The Marcus Foundation (PI: J.K.), The Dana Foundation (PI: J.M.S.), and the NIH (PI: A.W.S.), and fees from Duke University (B.B., S.W.). J.K. serves as the Director of the Carolinas Cord Blood Bank, a public cord blood bank located at Duke University. J.K. and J.M.S. have a patent pending for Cord Blood Therapy for Cerebral Palsy. S.W. and B.B. are employees of The Emmes Corporation and received research funding from Duke University. J.D.T. is a consultant to the Emmes Corporation, has honorarium for serving on DSMB for Gamida Cell, and research funding from Seattle Genetics. The other authors indicated no potential conflicts of interest.

Publisher Copyright:
© 2017 The Authors Stem Cells Translational Medicine published by Wiley Periodicals, Inc. on behalf of AlphaMed Press


  • Autologous stem cell transplantation
  • Cellular therapy
  • Clinical Trials
  • Cord blood
  • Human cord blood
  • Nervous system
  • Umbilical cord blood

ASJC Scopus subject areas

  • Medicine (all)


Dive into the research topics of 'Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial'. Together they form a unique fingerprint.

Cite this