Autism poses drug R&D challenges, nonprofits upping therapeutics game

At Swiss drug giant Novartis ($NVS), it's taken a diverse team of scientists and physicians working together to advance an experimental drug against Fragile X, the most common genetic cause of autism. And the development of medicines for patients with autism is likely to call for a similar mix of expertise and collaboration.

It's still the early days for autism drug R&D. When Boston venture capitalist Alan Crane and his fellow founders of the nonprofit Autism Consortium began talking to drug companies about 6 years ago about advancing treatments for the developmental disorder, he said, there was interest from pharma groups yet very little action. Now there are at least three Big Pharma companies engaged in R&D related to autism, in addition to smaller research outfits such as Seaside Therapeutics with programs in clinical trials.

There's definitely a market carrot for developers to chase in autism or autism spectrum disorders--which can lead to symptoms such as trouble interacting with others, slow development of language skills and a range of abnormal behaviors. It's estimated that one in 110 children in the U.S. are diagnosed with autism today, according to the nonprofit Autism Speaks. And despite the incidence and rise in autism cases, there are no FDA-approved drugs that treat the core symptoms of the disorder.

Yet unlike drugging against illnesses such as cancer, development of autism medicines faces some big challenges--such as a scarce number of validated drug targets, a lack of model systems of the disease in which to test new compound and low participation among biotech and pharma companies to advance new treatments. Adding to those challenges is the fact that the cause of autism spectrum disorders isn't really known.     

"There's a lot of scientific advances [in autism research] that we think are intriguing, but not yet the whole package together that you would need to do a drug development program tomorrow," said Donald Johns, VP and global head of neuroscience translational medicine for Swiss drugmaker Novartis ($NVS). "But we're intrigued by the profound unmet medical need and the emerging scientific basis, particularly some of the genetic studies."

Autism poses drug R&D challenges, nonprofits upping therapeutics game

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