Doctors Hope That New Drug Will Treat Autism

Katie Mills is a 7-year-old-girl who suffers from tuberous sclerosis complex, a rare genetic disorder which causes half of those afflicted to develop autism.

Katie recently joined a clinical trial at Boston Children, where she was offered several multivitamin-size white pills which, caused her to speak in complete sentences for the first time, and also extended her 30-second attention span to several minutes. Her mother, Susan, said that she was finally able to retain information from one day to the next.

“We’d had a child who had basically been 2 years old for four years,” said Mills.  “But after four months, she was communicating in ways she had never done before. It was amazing. It was like it was a totally different child.”

Doctors hope that if this treatment can work for children with tuberous sclerosis, then it can also work for those on the autism spectrum.  But Dr. Mustafa Sahin points out that a solution would not be so simple.  So many genes underlie autism that “you would need 500 different drugs to help [them].”  On a more optimistic note, Sahin says that it’s possible that the 500 genes “converge on certain common pathways and they can be rescued by common drugs.”

Autism advocates anticipate that rapamycin (the medication which Sahin’s team is testing) will be one of those common drugs.  “I keep my fingers crossed,” said Daniel Smith, who is the senior director of discovery neuroscience at the research and advocacy organization Autism Speaks, which helped fund the research.

Scientists conducted tests that showed that mice with tuberous sclerosis gene mutations behaved more normally and their tumors shrunk when they were given rapamycin.

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