Dr. Neil Dawson PhD Senior Lecturer Lancaster University

Genetic Lead Suggests Ketamine May Benefit Autism, Tourette’s and Schizophrenia

MedicalResearch.com Interview with:

Dr. Neil Dawson PhD Senior Lecturer Lancaster University

Dr. Dawson

Dr. Neil Dawson PhD
Senior Lecturer
Lancaster University

MedicalResearch.com: What is the background for this study?

Response: Deletions on chromosome 2p16.3, involving deletion of the NEUREXIN1 gene, dramatically increase the risk of developing a wide range of neurodevelopmental disorders including autism, Tourette’s syndrome and schizophrenia. We don’t fully understand the mechanisms involved. In our study we wanted to understand how the genetic deletion impacts on brain function and the ability of brain regions to communicate with one another, as these are known to be impaired in these neurodevelopmental disorders. We also wanted to determine how the genetic deletion impacts on the function of neurotransmitter systems involved in these disorders, and whether drugs targeting these neurotransmitter systems could restore some of the deficits in brain function seen.

MedicalResearch.com: What are the main findings?

Response: We found that 2p16.3 deletion significantly altered the function of key brain regions known to be affected in the different neurodevelopmental disorders. We found that the genetic deletion altered the function and impaired the connectivity of thalamic brain regions. This is important, as these thalamic brain regions play a key role in allowing other brain regions to communicate with one another. Interestingly, we also found that treatment with a low dose of ketamine, a drug used at higher doses as an anaesthetic, was able to restore the function and connectivity of these thalamic brain regions. This suggests that ketamine, or related drugs, may have some therapeutic potential in the treatment of neurodevelopmental disorders or for people with the 2p16.3 deletion. But we have to be cautious about this suggestion and much more research is needed. 

MedicalResearch.com: What should readers take away from your report?

Response: Our work gives us new insight into how the 2p16.3 genetic deletion impairs brain function and sets up the brain to fail, contributing to an increased risk of developing neurodevelopmental disorders.

The work also shows that ketamine, or similar drugs, may have some therapeutic potential in the treatment of autism, Tourette’s or schizophrenia. This work is also preclinical, so it also highlights the important translational value and impact of preclinical work in developing new effective treatments for the neurodevelopmental disorders.

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

 Response: As ketamine interacts with many different molecular targets we now want to determine which of these are most affected by the genetic deletion. We are also interested in testing the ability of drugs that are related ketamine, that are more selective or last for a longer time in the body, to restore the impact of the genetic deletion on brain function. The ultimate aim would be to test the efficacy of these drugs in individuals with the 2p16.3 deletion and in people with autism, Tourette’s or schizophrenia. 

Citation:” Ketamine Restores Thalamic-Prefrontal Cortex Functional Connectivity in a Mouse Model of Neurodevelopmental Disorder-Associated 2p16.3 Deletion” by Rebecca B Hughes, Jayde Whittingham-Dowd, Rachel E Simmons, Steven J Clapcote, Susan J Broughton and Neil Dawson, 8 December 2019, Cerebral Cortex.
DOI: 10.1093/cercor/bhz244


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Last Updated on December 11, 2019 by Marie Benz MD FAAD