Growth factor works again in another mouse model of autism features

A report from the recent meeting of the International Congress of Human Genetics – posted on the website of the Simons Foundation Autism Research Initiative (SFARI 10/19/11) – contains exciting news about the use of insulin-like growth factor-1 (IGF-1) in a mouse model. As discussed in Chap. 9, it had been previously shown in a mouse model of Rett syndrome, a disorder with some of the features of autism, that administering IGF-1 systemically can improve locomotor function, partially alleviating breathing and heart rate problems, and increase life span by 50%. These positive results in the mouse model led to a safety and efficacy trial being led by Omar Khwaja at Children’s Hospital Boston. In a study of 12 girls with Rett, IGF-1 was administered for 2 weeks and appears to improve breathing and heart rate. No serious side effects were found, although this treatment was for a very short time period. The FDA had previously approved the use of IGF-1 for children of short stature, so safety issues are not a major worry at this point.

In the new experiments, IGF-1 treatment was used in another mouse model of a gene associated with autism, SHANK3. This protein is known to be important for forming and maintaining synapses. When this gene is knocked out in mice, signaling defects are found at certain synapses, leading to behavioral deficits. As reported by Joe Buxbaum (Mt. Sinai School of Medicine) at the meeting, administering IGF-1 to these mice greatly improves their synaptic function. This has prompted another clinical trial of IGF-1, this time in children with Phelan-McDermid syndrome. These kids have severe motor and language deficits, and the plan is to monitor speech patterns and their distinctive gait abnormality during IGF-1 treatment. Stay tuned…

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