Cell therapy approaches to autism: a review of clinical trial data.
Jack Price
PMID 32448347WHAT IT FOUND
The only placebo-controlled trial found no behavioural improvement in autistic children given stem cells.
Open-label studies reported gains, but these match natural development or lack control groups. The review concludes current evidence does not justify further uncontrolled trials.
Key findings
01The sole placebo-controlled trial found no significant change in any behavioural test after autologous cord blood cell injections.
02Improvements in open-label studies aligned with natural history data from untreated cohorts, suggesting they may reflect normal development rather than treatment effects.
03Intrathecal administration carries risks including seizures, spinal headaches and pain, and was used without adequate preclinical safety data or biodistribution evidence.
STILL TO COME
How it was doneWhat they found
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What it does not show
The review is narrative and did not use systematic search methods or formal risk-of-bias assessment. Only five of thirteen trials had published data, so most registered studies could not be evaluated. The author's conclusions about ethics and study design are interpretive rather than derived from a formal appraisal tool. Trial heterogeneity in cell types, dosing and patient ages makes direct comparison difficult.
Declared interests
The author declares no competing interests. However, the review notes that one of the trials analysed (Riordan et al.) was a pay-to-participate study where authors declared financial conflicts of interest, and the review questions the validity of such trials.
The easy way to misread this
Do not interpret the positive results from open-label trials as evidence that stem cell therapy works. These studies lacked control groups, and their reported improvements match the natural progression seen in untreated autistic children.