Stem cell transplantation and autism: early days

My attention was recently caught by the publication of the study by Yong-Tao Lv and colleagues* (open-access) reporting results based on the use of stem cell transplantation in a small group of children diagnosed with an autism spectrum disorder (ASD).

I know that this research area is still a little bit of a hot potato when it comes to a heterogeneous, behaviourally-defined condition like autism (sorry, the autisms). As I intimated in a previous article about some related study in this area (see the ClinicalTrials.gov entry here) there are still quite a lot of unanswered questions about whether stem cell therapy is 'right' for autism or not; the progress of which is not helped by the historical propagation of the therapy via all those Google ads for specialist clinics offering stem cell services.

The new paper is open-access and includes a notable addition to the authorship group - one Paul Ashwood from UC Davis who has appeared more than once on this blog - and basically reports the results of a trial investigating "the safety and efficacy of combined transplantation of human cord blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells (UCMSCs) in treating children with autism". Without trying to rehash the authors findings, they basically suggested that (a) aside from some cases of transient fever, stem cell therapy was relatively 'safe' when it came to the monitoring of various measures, at least over the course of the investigative period and (b) compared with a control group who only received "professional sensory integration and behavioral rehabilitation therapy", two other groups of children who received CBNMC and CBMC + UCMSC transplantation in addition to behavioural intervention showed various "improvements" according to the behavioural measures used over the course of the 24 week trial. Indeed, the combination therapies CBMC + UCMSC transplantation "showed larger therapeutic effects than the CBMNC transplantation alone". That being said, this was quite a small trial and the trial was non-blinded and non-randomised.

I'm going to reiterate my oft-cited caveat about this blog not giving medical or clinical advice particularly when it comes to something as 'new' as stem cell therapy being suggested for autism. I am inclined to point you towards another archive post where stem cell transplants were mentioned with autism (or at least mouse models of autism) in mind, based on the impressive work coming out of Paul Patterson's laboratory (see here) and how immune function, development and behaviour might be players at the same table for some cases of autism. The more recent paper has gone beyond mouse models....

Every therapeutic option which is put forward for 'managing' the characteristics of autism needs to start somewhere when it comes to testing for safety and efficacy and stem cell therapy is no exception. Whilst questions still remain about the long-term safety and effects of such transplantations and indeed, whether there is any appetite for such an intervention, the research toe has well and truly been dipped into the experimental waters.

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* Yong-Tao Lv. et al. Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism. Journal of Translational Medicine 2013, 11:196

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Yong-Tao Lv (2013). Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism Journal of Translational Medicine DOI: 10.1186/1479-5876-11-196