Search This Blog

Tuesday, September 3, 2013

Testing a Drug

Although some medications may help with the co-occurring conditions of autism (e.g., anxiety), there is no drug for autism itself. The Boston Globe reports on tests of a medication that might alleviate some symptoms of autism:
Researchers frequently use diseases that involve simple, clear gene mutations, like the ones in tuberous sclerosis, to better understand more complex conditions such as autism that involve hundreds of genes. Because so many genes underlie the disorder, “one pessimistic point of view would be that you’d need 500 different drugs to help individuals with autism,” said Dr. Mustafa Sahin, who is leading the trial at Boston Children’s. “But maybe, these 500 genes converge on certain common pathways and they can be rescued by common drugs.”
Autism advocates are cautiously optimistic that rapamycin, the medication Sahin’s team is testing, will be that common drug. “I keep my fingers crossed,” said Daniel Smith, senior director of discovery neuroscience at the research and advocacy organization Autism Speaks, which helped fund the research.
In the study, 50 children with tuberous sclerosis are taking rapamycin — used for decades to prevent rejection of transplanted organs — or a placebo for six months. The goal is to combat symptoms of the disease. Roughly one-third of tuberous sclerosis patients develop epilepsy, particularly in early childhood, so researchers also hope to learn more about that condition, Sahin said.