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Cell therapy weekly: cord blood for autism and promising gene therapy safety data

This week: first double-blind crossover study of children with autism demonstrates improvement after cord blood treatment and Celgene commences Juno buy-out.

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Feb 09, 2018
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2 Comments

Go to the profile of James L. Sherley, M.D., Ph.D.

The trial of  Chez et al., reported in SCTM to evaluate possible beneficial effects of UCB on autism spectrum diseases, has two potentially confounding design features.

1) A cross-over design is ill-advised for investigations of stem cell treatments, which if in fact efficacious might have long-lived effects.   Long-lived treatment effects will confound the comparison of treated versus placebo in a cross-over study.

2) DMSO the cryoprotectant for UCB units, is also a potent cell differentiation agent, including differentiation of neuronal cells.   This study did not control for effects of DMSO.   If beneficial effects are present, though not detected with statistical confidence in the reported trial, they might be due to DMSO instead of the cells in the treatment.  Differentiation effects are also long-lived and will be confounded in a cross-over study.

James Sherley

Asymmetrex

Go to the profile of Freya Leask
Freya Leask 13 days ago

Interesting thoughts, James - thanks for sharing!