WEDNESDAY, April 11 (HealthDay News) — An experimental drug reversed many of the major symptoms of Fragile X syndrome in mice, researchers report.
Fragile X syndrome is a leading cause of autism, and its symptoms include developmental delays, hyperactivity, learning and memory difficulties, low IQ, seizures and social and communication deficits.
Prior research suggested that inhibiting mGlu5, a receptor for a neurotransmitter known as glutamate, might help alleviate some symptoms.
In the new study, researchers in Switzerland and at the Massachusetts Institute of Technology treated adult mice genetically engineered to have Fragile X syndrome with a compound called CTEP, which inhibits mGlu5.
“We found that even when treatment with CTEP was started in adult mice, it reduced a wide range of [Fragile X] symptoms,” study author Dr. Lothar Lindemann, of F. Hoffmann-La Roche Ltd. in Basel, said in a journal news release.
Symptoms that showed improvement included learning and memory challenges and hypersensitivity to sounds.
Although studies involving animals can be useful, they frequently fail to produce similar results in humans, experts noted. The study’s authors also pointed out that CTEP was not developed for use in people. The findings, however, could help scientists pursue a similar treatment for people with Fragile X.
“The most important implications of our study are that many aspects of [Fragile X] are not caused by an irreversible disruption of brain development, and that correction of the altered glutamate signaling can provide widespread therapeutic benefit,” said Mark Bear, of MIT’s Picower Institute for Learning and Memory.
The study is published in the April 12 issue of the journal Neuron.
The U.S. National Institutes of Health provides more information on Fragile X syndrome.
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