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Progress report on precision therapies for autism

Today in Science magazine: What’s next in the development of individualized treatments for autism?
October 15, 2015

Today in Science magazine, Harvard neurologist Mustafa Sahin and MIT brain scientist Mriganka Sur summarize scientific progress toward delivering precision treatments for autism spectrum disorder (ASD).

“Given the large number of genes that potentially confer ASD risk, the genetic heterogeneity of ASD presents a substantial obstacle to development of one-size fits all therapies,” they write. “Biomarkers can be crucial for predicting subjects most likely to respond [to tailored treatments].”

Biomarkers can include the results of genetic testing, blood work, brain imaging and other medical assessments. In particular, Drs. Sahin and Sur call for research aimed at identifying individual differences in the brain signaling molecules and brain networks that likely produce autism symptoms.

While biomarkers provide the most promising targets for future medicines, in most cases such treatments will be in addition to – not instead of – behavioral therapies, they add.

Going forward, Drs. Sahin and Sur call for a new generation of clinical trials that recruit participants based on common biomarkers and provide interventions that combine pharmacological and behavioral treatments.

“The successful development of new therapies absolutely depends on adoption of the viewpoint put forth by Drs. Sahin and Sur,” agrees Mathew Pletcher, Autism Speaks head of genomic discovery. “Our MSSNG program was created with the goal of providing the data necessary to enable this precision medicine approach for ASD.”

MSSNG is a groundbreaking collaboration between Autism Speaks and Google that is creating the world’s largest genomic database on autism and making it freely available for global research.


Read more about MSSNG here.

Read an abstract of Dr. Sahin and Sur’s paper – "Genes, Circuits, and Precision Therapies for Autism and Related Neurodevelopmental Disorders" – here. (Access to the full article requires a one-time fee or subscription.)