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expert reaction to study on anti-cancer drug romidepsin and reversal of social deficits in mice

A new study, published in Nature Neuroscience, examined the effect of the anti-cancer drug romidepsin on animal models of autism.


Dr Grainne McAlonan, Deputy Head, Sackler Centre for Translational Neurodevelopment at King’s College London, said:

“This is potentially really exciting and the work presented here seems to be thorough.

“The model is an informative one for autism spectrum disorder (ASD) but the caution is that while many individuals with a SHANK3 mutation have autism, not many people with ASD have a SHANK3 mutation. Thus in thinking about how this translates to people with ASD one must first ask the question does it translate to the human SHANK3 deficiency syndrome before seeking to generalize results to the etiologically and clinically diverse population that is autism.

“But, we should definitely be doing these kind of experiments in animal models first; and the approach builds upon an accumulating evidence that epigenetics have a role to play in ASD and therefore could be a treatment target.”


Dr Rosa Hoekstra, Lecturer in Psychology, Institute of Psychiatry Psychology & Neuroscience, King’s College London (IoPPN), said:

“This study examined the effect of romidepsin on social deficits in a particular mouse model: mice with a deficiency in the SHANK3 gene. It is important to emphasise that this study is still very far removed from having any direct relevance to people with autism. Changes in the SHANK3 gene have been implicated in only a small minority (about .5 to 2%) of people with autism. It is unknown whether the findings presented here will ever have any relevance to the vast majority of people with autism, who do not have abnormalities in the SHANK3 gene.”


Dr Georgina Warner, Research Manager, Autistica, said:

“Autism is a very diverse condition with many different genes playing a role so developing a mouse model to represent it is challenging.  While mouse studies like this one can play an important role in research, findings can’t easily be applied to humans. The Shank 3 gene this research focuses on represents a syndrome in which autism spectrum disorder (ASD) is common but, as the authors admit, the model only accounts for 0.5 to 2 per cent of ASD and intellectual disability cases. The study reports interesting findings about the drug romidepsin, but it’s far too early to say whether it would have any effect on autistic people’s social skills. It’s also not clear that all autistic people would welcome a drug which aimed to improve their social ability, especially if it had other side-effects. Autistic people and carers of children with autism should not consider using the drug other than for its approved use as a cancer treatment.”


* ‘Social deficits in Shank3-deficient mouse models of autism are rescued by histone deacetylase (HDAC) inhibition’ by Luye Qin et al. will be published in Nature Neuroscience on Monday 12 March 2018.


All our previous output on this subject can be seen at this weblink:


Declared interests

Dr Rosa Hoekstra: I am a lecturer in Psychology at King’s College London; my research is funded by MRC (UK) and Autism Speaks. My research is in a different field of autism (only concerning humans). I therefore believe I do not have a conflict of interest in relation to the study reported.

None others received.

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