Pilot Clinical Trial of Lithium in Fragile X Shows Promising Results

With a $65,000 grant from FRAXA Research Foundation in 2005, Dr. Berry-Kravis at the Rush University Medical Center conducted a pilot clinical trial of lithium in 15 patients with Fragile X syndrome.

Results Published 2008: Open-label treatment trial of lithium to target the underlying defect in fragile X syndrome
Elizabeth Berry-Kravis, MD, PhD, Fragile X researcher
$65,000 Grant
Elizabeth Berry-Kravis, MD, PhD
Principal Investigator
Rush University Medical Center
2005 FRAXA Research Grant

by Elizabeth Berry-Kravis

Fifteen subjects with Fragile X will be treated with lithium to get pilot data to justify a larger trial of lithium. The lithium treatment will be added on to other medications the individuals enrolled are already taking, and treatment will be for at least two months and up to a year if the lithium is helpful.

Tests will be given at the beginning of the study, after two months of treatment, and after a year of treatment for those who are treated a full year. Side effects will be monitored closely to make sure that lithium is safe in the Fragile X population. We will use a battery of behavioral and thinking tests which we are validating specifically for use in medication trials in Fragile X. We will try new physiological tests to measure over-stimulation and eye aversion, a special blood test that may serve as a biomarker for improvement in the cellular defect in Fragile X, and some new tests of associative learning.

These new tests, in combination with some of the tests that worked best in our previous Ampakine study, should allow us to know if lithium is improving some of the behaviors and learning deficits seen in Fragile X syndrome. If the new tests are able to measure a medication response in this study, we can then use them for other future studies.

Results of this trial are here.

A 2014 review of the studies on minocycline for Fragile X by Zhonghua Liu and Carolyn Beebe Smith at the National Institute of Mental Health is here.

Elizabeth Berry-Kravis, MD, PhD, Fragile X researcher

Explore Current Fragile X Research

FRAXA-funded researchers around the world are leading the way towards effective treatments and ultimately a cure.

Share via
Copy link