Forbidden Fruit: Autism and Stem Cells

Anyone who has typed the word “autism” into an internet search engine will probably have seen those little pop-up adverts accompanying some of the search results offering stem cell therapy for autism. Normally offered at some clinic with a fancy title, stem cell therapy for lots of conditions brings to my mind Hollywood images of genetic rearrangement such as those portrayed in the James Bond film ‘Die Another Day’ tinged with questions about safety, efficacy and potential side effects. Ethics is another factor; and those important debates about the use of embryonic stem cells. Indeed, government and politics have been inextricably tied into stem cell research almost since its conception.

Outside of such factors, the past few years have quietly seen the topic of stem cell use in cases of autism discussed in the peer-reviewed research literature. Phrases such as cellular reprogramming [1] and induced pluripotent stem cells (iPSCs) [2] have appeared alongside the suggestion that autism and various other neuropsychiatrically-defined conditions might benefit from such research.

2012 has, to my mind, marked a bit of a turning point in the discussions about stem cells and autism with several important findings and announcements being reported. Here are a few examples:

Prof. Paul Patterson and colleagues published an important paper in July 2012 [3].  Previously suggesting that a mouse model of stimulated immune activation during pregnancy resulted in behavioural effects on offspring which seemed to overlap with the core symptoms of autism [4], Hsiao and colleagues [3] described similar immune related effects to offspring mice following maternal immune activation. Furthermore, irradiation and transplantation of “immunologically normal” bone marrow seemed to correct some of the behaviours noted in offspring mice including repetitive actions and anxiety-type behaviours. The authors went to great lengths to reiterate that this was a study of mice not people and not an advert for bone marrow / stem cell transplantation for autism or anything else at this time. Discussions also remain as to whether the irradiation process prior to transplantation may have also have played some role in the presented results.

At the same time, news is also emerging of a new trial of cord blood stem cells being undertaken for autism [5] based at The Sutter Neuroscience Institute in Sacramento. The trial “Autologous Cord Blood Stem Cells for Autism” [6] headed by Dr Michael Chez is looking to see whether infusions of umbilical cord stem cells can affect language skills and behaviour alongside other markers of the immune system including pro- and anti-inflammatory cytokines in cases of autism. Initial results are expected late 2013.

Many questions still remain about this controversial area of research and what benefits / consequences it may ultimately bring to conditions like autism. Such research potentially offers some interesting insights into autism, reinforcing the possibility that the immune system and behavioural presentation of autism, some autism, may be linked and opening the door to a potential behavioural effect from immunotherapy (in whatever way, shape or form this may take) at least in some cases.

The question of safety, short- and long-term safety, remains a key priority and whether such biological “tinkering” may predispose participants to other adverse risks and events as a result of such procedures. Treading very cautiously however and without forming any opinion on this area of investigation, one cannot discount the results from groups like Prof. Patterson’s, and the important need for further investigation in this area related to autism and beyond.



[1] Ming GL. et al. Cellular reprogramming: recent advances in modeling neurological diseases. The Journal of Neuroscience. 2011; 31: 16070-16075.

[2] Vaccarino FM. et al. Induced pluripotent stem cells: a new tool to confront the challenge of neuropsychiatric disorders. Neuropharmacology. 2011; 60: 1355-1363.

[3] Hsiao EY. et al. Modeling an autism risk factor in mice leads to permanent immune dysregulation. PNAS. 2012; 109: 12776-12781.

[4] Malkova NV. et al. Maternal immune activation yields offspring displaying mouse versions of the three core symptoms of autism. Brain, Behavior & Immunity. 2012; 26: 607-616.

[5] The Modesto Bee. Sutter Neuroscience Institute launching trial of cord blood stem cells in autistic children. (Accessed: 21 August 2020).

[6] Autologous Cord Blood Stem Cells for Autism. NCT: 01638819. (Accessed: 21 August 2020).

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