Clinical Trial of Combined Treatment of Minocycline and Lovastatin in Fragile X Syndrome

With a $66,714 grant from the FRAXA Research Foundation awarded over 2015-2017, Dr. Francois Corbin at the Universite of Sherbrooke will test the safety and synergistic effects of lovastatin and minocycline in patients with fragile X syndrome.

Francois CORBIN
$66,714 Grant
Francois Corbin, MD, PhD
Principal Investigator
Université de Sherbrooke
2015-2017 FRAXA Research Grant
$66,714 over 2 Years

This project aims to test the safety and synergistic effect of lovastatin and minocycline combined treatment in patients with fragile X syndrome. The hope is to improve behavior in FXS individuals. It will also seek to establish correlations between platelets biomarkers and clinical response to lovastatin and/or minocycline. Furthermore, the investigators will determine the effect of these drugs on brain activity and neurochemistry using functional magnetic resonance imaging (sMRI/fMRI) and transcranial magnetic stimulation (TMS), which may be able to assess synaptic plasticity (LTP, LTD) in patients.

Dr. Corbin presented the results of this trial at the 18th International Fragile X and Related Neurodevelopmental Disorders Workshop, October 12-16, 2017 in Saint-Alexis-des-Monts, Quebec, Canada.

Earlier Pilot Clinical Trial of Lovastatin

Dr. Corbin also ran the first clinical trial of lovastatin as a potential treatment for fragile X Syndrome, funded by FRAXA in 2012 for $44,000. Since this drug had never been tested in fragile X patients, an open-label trial on a limited number of patients was the first step toward evaluating its safety and efficacy. This was a 12 week trial, with 16 patients ages 10-25 at the Fragile X Clinic at the Centre Hospitalier Universitaire de Sherbrooke (CHUS), Quebec, Canada.

Why Lovastatin for Fragile X?

Dr. Mark Bear and colleagues at MIT published a paper in Neuron showing that Lovastatin can reverse many signs of disease in Fragile X mutant mice. The team’s multi-level preclinical validation strongly suggests that lovastatin could have disease-modifying effects in people with fragile X. Fortunately, these results have been known to FRAXA for some time, having been presented at the 2011 FRAXA Investigators Meeting. Shortly after that meeting, plans were laid for this pilot trial to investigate efficacy in humans.

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