Metformin treatment in young children with fragile X syndrome.

Abstract

BACKGROUND:

Metformin is a drug commonly used in individuals with type 2 diabetes, obesity, and impaired glucose tolerance. It has a strong safety profile in both children and adults. Studies utilizing the Drosophila model and knock out mouse model of fragile X syndrome (FXS) have found metformin to rescue memory, social novelty deficits, and neuroanatomical abnormalities. These studies provided preliminary evidence that metformin could be used as a targeted treatment for the cognitive and behavioral problems associated with FXS. Previously, a case series of children and adults with FXS treated with metformin demonstrated improvements in irritability, social responsiveness, language, and hyperactivity.

METHODS:

Here, we present nine children with FXS between 2 and 7 years of age who were treated clinically with metformin and monitored for behavioral and metabolic changes.

RESULTS:

Parent reports and developmental testing before and after metformin are presented. There were improvements in language development and behavior (such as lethargy and stereotypy) in most of the patients.

CONCLUSION:

These results support the need for a controlled trial of metformin in children with FXS under 7 years old whose brains are in a critical developmental window and thus may experience a greater degree of clinical benefit from metformin.

• Authors: Hazel Maridith B Biag, Laura A Potter, Victoria Wilkins, Sumra Afzal, Alexis Rosvall, Maria Jimena Salcedo-Arellano, Akash Rajaratnam, Ramiro Manzano-Nunez, Andrea Schneider, Flora Tassone, Susan M Rivera, Randi J Hagerman
• Journal: Molecular genetics & genomic medicine (Mol Genet Genomic Med) Vol. 7 Issue 11 Pg. e956 (11 2019) ISSN: 2324-9269 [Electronic] United States
• PMID: 31520524 (Publication Type: Case Reports, Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
• Copyright: © 2019 UC Davis MIND Institute. Molecular Genetics & Genomic Medicine published by Wiley Periodicals, Inc.

Chemical References

• Hypoglycemic Agents
• Metformin

Topics

• Child
• Child, Preschool
• Fragile X Syndrome (drug therapy, pathology)
• Humans
• Hypoglycemic Agents (therapeutic use)
• Male
• Metformin (therapeutic use)
• Neurodevelopmental Disorders (prevention & control)
• Prognosis