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Abstract * d0 F B7 w; e
Background 4 W8 F% J! ~# l( l' ]: u" O y
Autism is a pervasive neurodevelopmental disorder. At present there are no defined
/ o9 N% A, L+ V# I9 ]6 Y" ^mechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem 5 V# p4 J2 F' n" B
cell transplantation may offer a unique treatment strategy for autism due to immune and 0 Z4 g' a& } R! M9 p! R$ ~
neural dysregulation observed in this disease. This non-randomized, open-label, single center
& U6 e2 h& W$ j. jphase I/II trial investigated the safety and efficacy of combined transplantation of human cord
5 A& |( a, p4 H+ J! _blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells
6 Z. Y/ J7 N0 a* H. k# m(UCMSCs) in treating children with autism. 8 K/ A1 ^4 B1 ^4 ?
Methods * ^- O% z* x. J& H; J: I
37 subjects diagnosed with autism were enrolled into this study and divided into three groups: 8 C9 U" E5 v7 x" S/ S1 m
CBMNC group (14 subjects, received CBMNC transplantation and rehabilitation therapy),
8 \' V) g4 `: D7 W9 s7 jCombination group (9 subjects, received both CBMNC and UCMSC transplantation and ' X6 Y+ I- k B9 _! Q
rehabilitation therapy), and Control group (14 subjects, received only rehabilitation therapy). 4 W) d! d/ c" f6 d+ A8 E! U4 d n
Transplantations included four stem cell infusions through intravenous and intrathecal ) o) D4 p2 p$ P& B/ c+ s
injections once a week. Treatment safety was evaluated with laboratory examinations and
m' c- j, L3 _1 k9 w. R+ Kclinical assessment of adverse effects. The Childhood Autism Rating Scale (CARS), Clinical * C7 M+ g; c D
Global Impression (CGI) scale and Aberrant Behavior Checklist (ABC) were adopted to
# ~% Q- j/ k- o. H# h0 Qassess the therapeutic efficacy at baseline (pre-treatment) and following treatment.
' j* y/ T# i: H3 z, \2 ]1 G* sResults
0 x% m% v3 |+ J3 IThere were no significant safety issues related to the treatment and no observed severe " j. o9 T" y5 c2 X. Y" C
adverse effects. Statistically significant differences were shown on CARS, ABC scores and 5 i/ t3 @6 @( G& V1 l& @7 o6 s
CGI evaluation in the two treatment groups compared to the control at 24 weeks post-+ z9 E; K2 z& Q9 _0 b4 }
treatment (p < 0.05).
/ s" K4 J5 E9 `Conclusions
8 z* P, U: a" F, ]4 `$ X4 xTransplantation of CBMNCs demonstrated efficacy compared to the control group; however,
8 N3 V Q! P! Y2 E. N3 o* Q1 \the combination of CBMNCs and UCMSCs showed larger therapeutic effects than the
4 a" ]; u3 q8 [$ w& h5 p+ ACBMNC transplantation alone. There were no safety issues noted during infusion and the
3 t1 ^' s" H* j8 B% Iwhole monitoring period. Trial registration 9 Q9 C# O4 y q8 |8 y
ClinicalTrials.gov: NCT01343511, Title “Safety and Efficacy of Stem Cell Therapy in
$ k( }, f0 r4 m# v$ j$ n0 zPatients with Autism”.
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发表于 2013-9-2 15:29
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发表于 2013-11-23 22:18
