The results proved so hopeful, that the Food and Drug Administration approved the use of the drugs in three clinical trials currently under review to test the effect of statins in children and adults born with NF1. The findings could help the estimated 35 million Americans who struggle with learning disabilities.
"Learning disabilities and mental retardation each affect five percent of the world population," said Dr. Alcino Silva, professor of neurobiology, psychiatry and psychology at the David Geffen School of Medicine at UCLA. "Currently, there are no treatment options for these people. That's why our findings are so exciting from a clinical perspective."
In an earlier study, Silva and his colleagues linked NF1's learning problems to a protein called Ras, a protein that regulates how brain cells talk to each other. This communication is what enables learning to take place. The NF1 mutation creates hyperactive Ras, which disrupts cellular conversation and undermines the learning process.
"The act of learning creates physical changes in the brain, like grooves on a record," said Silva. "But surplus Ras tips the balance between switching signals on and off in the brain. This interrupts the delicate cell communication needed by the brain to record learned information."
The UCLA team began searching for a safe drug that would zero in on Ras and overcome its hyperactivity without causing harmful side effects over long-term use.
"It became something of a Quixotic quest -- an impossible dream," Silva admitted.
Source:University of California - Los Angeles