Where’s the funding for translational research in autism?

Autism scientists are well aware of the importance of translational research — work that translates basic findings in animal models into treatments for people.

Unfortunately, potential autism treatments that work well in animals often do not work when tested in people. For researchers desperate to find solutions, these failures are extremely disappointing. Failed clinical trials can also spark doubt in the public and in funding agencies, potentially affecting future research.

Take, for example, the experimental drug arbaclofen. In 1991, researchers identified an abnormal expansion of FMR1, the gene mutated in fragile X syndrome. They then created a mouse model that expresses the mutated protein and found a drug that corrects the abnormal features of neurons and autism-like behaviors in the mice. But when scientists tested the drug in people with fragile X syndrome, it did not diminish their autism features.

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23rd January 2018