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From Technology Review @MIT-
New Drug Being Tested for Autism
Last year, Mark Bear and Gul Dolen, of MIT, published an exciting finding for the autism community: they described correcting many of the symptoms of fragile X syndrome, a genetic disorder that is the most common form of heritable mental retardation and a leading cause of autism, by changing the activity of a specific gene in mice. Drugs that target the same gene are now being tested in humans, Dolen revealed at the conference this week. This is great news, given the lack of existing or even experimental therapies for autism. Dolen declined to give further details on the tests but says she hopes that the same approach will work for more common types of autism, which do not share the gene mutation that underlies fragile X.
To correct fragile X deficits in mice, researchers started with mice that carry the mutation responsible for the disorder and show many of the same symptoms. They then engineered the mice to have lower levels of a receptor called metabotropic glutamate receptor 5 (mGluR5), which improved abnormal brain development and faulty memory and reduced seizures--some of the defining symptoms of the disorder.
Pharma companies have been developing drugs that target this class of receptors, which are involved in the brain's primary excitatory signaling mechanism, for a number of disorders, including schizophrenia, chronic pain, Parkinson's, and anxiety. (I wrote a piece on this new drug class several years ago but haven't heard much about them since then. According to the NIH clinical trials database, none are in publicly funded clinical trials.) Maybe the autism findings will breathe new life into their development.
Last year, Mark Bear and Gul Dolen, of MIT, published an exciting finding for the autism community: they described correcting many of the symptoms of fragile X syndrome, a genetic disorder that is the most common form of heritable mental retardation and a leading cause of autism, by changing the activity of a specific gene in mice. Drugs that target the same gene are now being tested in humans, Dolen revealed at the conference this week. This is great news, given the lack of existing or even experimental therapies for autism. Dolen declined to give further details on the tests but says she hopes that the same approach will work for more common types of autism, which do not share the gene mutation that underlies fragile X.
To correct fragile X deficits in mice, researchers started with mice that carry the mutation responsible for the disorder and show many of the same symptoms. They then engineered the mice to have lower levels of a receptor called metabotropic glutamate receptor 5 (mGluR5), which improved abnormal brain development and faulty memory and reduced seizures--some of the defining symptoms of the disorder.
Pharma companies have been developing drugs that target this class of receptors, which are involved in the brain's primary excitatory signaling mechanism, for a number of disorders, including schizophrenia, chronic pain, Parkinson's, and anxiety. (I wrote a piece on this new drug class several years ago but haven't heard much about them since then. According to the NIH clinical trials database, none are in publicly funded clinical trials.) Maybe the autism findings will breathe new life into their development.