New drug offers hope for autism-related condition

June 23rd, 2008 - 12:54 pm ICT by IANS  


Washington, June 23 (IANS) An FDA-approved drug has been found to reverse brain dysfunction, caused by a genetic disease called tuberous sclerosis complex, that also afflicts half of autism patients. The finding offers new hope for addressing learning disorders caused by autism.

Using a mouse model for tuberous sclerosis complex (TSC), scientists at the University of Calfornia at los Angeles (UCLA) tested the ability of the FDA-approved rapamycin drug to fight tissue rejection after organ transplants.

TSC is a devastating disorder that disrupts how the brain works, often causing severe mental retardation. Even in mild cases, learning disabilities and short-term memory problems are common.

Half of all TSC patients also suffer from autism and epilepsy. The disorder strikes one in 6,000 people, making it twice as common as Huntington’s or Lou Gehrig’s disease.

Rapamycin is well-known for targeting an enzyme involved in making proteins required for memory. The UCLA team chose the drug because the same enzyme is also regulated by TSC proteins.

“This is the first study to demonstrate that Rapamycin can repair learning deficits related to a genetic mutation that causes autism in humans.”

“The same mutation in animals produces learning disorders, which we were able to eliminate in adult mice,” explained principal investigator Alcino Silva.

The study and other recent studies suggest that some forms of mental retardation can be reversed, even in the adult brain.

“These findings challenge the theory that abnormal brain development is to blame for mental impairment in tuberous sclerosis,” said co-author Dan Ehninger.

“Our research shows that the disease’s learning problems are caused by reversible changes in brain function — not by permanent damage to the developing brain.”

“After only three days of treatment, the TSC mice learned as quickly as the healthy mice,” said Ehninger.

“Rapamycin corrected the biochemistry, reversed the learning deficits and restored normal hippocampal function, allowing the mice’s brains to store memories properly.”

The findings of the study have been published online in the latest edition of the journal Nature.

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