Improving Memory Reversal Testing and Treatment with AMPAkines in the Fragile X Knock-Out Mouse
W. Ted Brown, MD, PhD
Principal Investigator
NYS Institute For Basic Research
Staten Island, NY
1997-1998 Grant Funding: $62,000
Summary
This was a first attempt to use smart drugs to treat Fragile X.
The Science
The goal of this grant was to develop an improved test to show learning deficits in the FMR1 knock-out mouse model of Fragile X, and then to test the effects of experimental drugs (Ampakines) that may be effective in treating these deficits.
Ampakines are a class of drugs that enhance the activity of AMPA receptors in the brain, which are involved in synaptic plasticity and learning and memory. Later studies, including the following pilot clinical trial of CX516 which was funded by FRAXA, suggested that ampakines could improve cognitive function and behavior in individuals with Fragile X Syndrome.
Berry-Kravis, E., Krause, S. E., Block, S. S., Guter, S., Wuu, J., Leurgans, S., … & Brown, W. T. (2006). Effect of CX516, an AMPA-modulating compound, on cognition and behavior in fragile X syndrome: a controlled trial. Journal of Child Neurology, 21(10), 849-855. doi: 10.1177/08830738060210101301
https://pubmed.ncbi.nlm.nih.gov/17069542/
