In Mice, Drug Reverses Symptoms of Condition Linked to
WEDNESDAY, April 11 (HealthDay News) -- An experimental drug
reversed many of the major symptoms of Fragile X syndrome in mice,
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
Prior research suggested that inhibiting mGlu5, a receptor for a
neurotransmitter known as glutamate, might help alleviate some
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
"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
The U.S. National Institutes of Health provides more information
Fragile X syndrome.
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