080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

New Pfizer unit to take on autism

The Autism News | English

Currently, only one medication – Johnson & Johnson’s antipsychotic drug Risperidone – has been cleared by the Food and Drug Administration for use in autistic children. But autism advocacy groups are looking eagerly at Pfizer’s new model in the hopes that it can develop novel medicines to help alleviate autism symptoms – or, perhaps more important, delve into a number of its already established drugs that target neural pathways affecting autistic patients.

”Presumably, some drugs already developed could be helpful,” said Dr. Michael Tranfaglia, medical director and chief scientific officer of the FRAXA Research Foundation, a Newburyport, Mass.-based organization that funds research and does advocacy work for people with a form of autism known as Fragile X.

Tranfaglia said major pharmaceutical firms like New York-based Pfizer used to spend most of their time chasing the biggest blockbusters but are now gradually coming to realize they may have a treasure trove of older drugs that could target smaller populations, such as those with Fragile X. Fragile X is a rare form of autism in which one key gene shuts down, failing to produce an important protein, leading to learning problems and anxiety, among other symptoms.

Anabella Villalobos of East Lyme, the head of the neuroscience research unit at the Groton labs, said Fragile X gives scientists at Pfizer a relatively easy early target to investigate, since only one gene is involved. Most of the autism disorders – called a spectrum because they are so wide-ranging – involve multiple genes.

”This is all a work in progress,” said Villalobos, who is overseeing the autism unit on an interim basis.

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