Sun JM, Song AW, Case LE, Mikati MA, Gustafson KE, Simmons
R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen LW, Wease S,
Blackwell B, Worley G, Troy J, Kurtzberg J. Effect of Autologous Cord Blood
Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral
Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med. 2017 Oct
28. doi:10.1002/sctm.17-0102. [Epub ahead of print]
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.
Courtesy of: https://neurologistconnect.com/posts/5a0b28a0634e884e0b8b4615?SKUID=6656d46c04553656b04bf4a8e0248071&mkt_tok=eyJpIjoiTlRBMk5EZGlObVE0WkdSaCIsInQiOiJtckt4a3N6YVZRdFpSS0NibEpORVV4K1ByRzhCVjk1Nk1aMlwvaHZ0am8wd3h2TDc0ZWd4dFVYQUNZTXNKaDgwQmFhSFFUR1RmWlNcL1BWR29xZytcL1N2d0t4Y1BPZXhqQUVrOU5JK3J5WjZaSytRRzFPaUVUZ0FpNnNraHN1c1lYRyJ9
See: http://childnervoussystem.blogspot.com/2015/12/autologous-cord-blood-infusion-for.html
See: http://childnervoussystem.blogspot.com/2015/12/autologous-cord-blood-infusion-for.html
A colleague comments: The study is good and promising, but the gains are not exceptional when the source data is viewed.
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