Fragile X clinical trial combines two available drugs

A pilot, randomized trial of minocycline and lovastatin combined treatment in Fragile X

2015 Clinical Trial funded for $66,714

This project aims to test the safety and synergistic effect of lovastatin and minocycline combined treatment. 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.

2012: Pilot Clinical Trial of Lovastatin

Funded for $44,000 in 2012

Dr. Corbin ran the first clinical trial of lovastatin as a potential treatment for Fragile X Syndrome. Since this drug has never been tested in FX patients, an open-label trial on a limited number of patients will be the first step toward evaluating its safety and efficacy. This study is 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.


Rationale for Lovastatin

by Michael Tranfaglia, 3/15/2013

The Mark Bear Lab 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. To prove efficacy in humans, clinical trials in Fragile X subjects are still necessary before lovastatin can be recommended as a new treatment.

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 a pilot trial and FRAXA authorized funding for an open trial of lovastatin directed by Francois Corbin at the Fragile X Clinic at University of Sherbrooke, Canada.