With a 2017 grant from FRAXA Research Foundation of $90,000, Dr. Andreas Frick’s team at Neurocentre Magendie, in France, will test non-invasive imaging using magnetic resonance imaging (MRI) as a potential biomarker for future fragile X syndrome clinical trials.
There is an urgent need for improved outcome measures for fragile X clinical trials. Behavioral ratings can be subjective and hard to quantify, so we need tests that can evaluate a potential treatment’s biological effects in an objective and easily measurable way. Really good measures might be used for stratifying patients likely to respond to a given treatment. Ideally, they should work in both mouse models and human patients.
Dr. Frick’s team will explore non-invasive imaging approaches using magnetic resonance imaging (MRI). They will first measure defects in the structural and functional connectivity of key brain networks in fragile X mice. With these mice, they will test the therapeutic potential of three promising molecules targeting known aspects of fragile X physiopathology.
If they are able to correct connectivity defects by chronic pharmacological intervention, they will evaluate changes in behaviors following treatment to correlate behavioral parameters with their connectivity measures.
If this project is successful, we will have objective outcome measures to evaluate new treatments, both in mice bred to mimic fragile X and in human patients.
Kamila Castro Grokoski