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Stem Cells Transl Med. 2017 Dec;6(12):2071-2078. doi: 10.1002/sctm.17-0102. Epub 2017 Oct 28.

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

Author information

1
The Robertson Clinical and Translational Cell Therapy Program, Duke University, Durham, North California, USA.
2
The Brain Imaging and Analysis Center, Duke University, Durham, North California, USA.
3
Department of Physical and Occupational Therapy, Duke University, Durham, North California, USA.
4
Division of Pediatric Neurology, Duke University, Durham, North California, USA.
5
Department of Psychiatry, Duke University, Durham, North California, USA.
6
Division of Neonatology, Duke University, Durham, North California, USA.
7
Stem Cell Transplant Laboratory, Duke University, Durham, North California, USA.
8
The Emmes Corporation, Rockville, Maryland, USA.

Abstract

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 × 107 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 × 107 /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.

KEYWORDS:

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

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