mTOR Archives

Metabolic and Mitochondrial Crosstalk in Insulin and cAMP Pathways in Fragile X Syndrome

August 28, 2025

Fragile X syndrome research explores insulin and cAMP pathway crosstalk, mapping brain metabolism to guide treatment strategies.

Targeting Adiponectin to Treat Fragile X Syndrome

October 29, 2019

Boosting adiponectin, a hormone that regulates metabolism, may improve cognition and behavior in Fragile X. Early results suggest it can restore brain plasticity.

Spectrum News – Newly Discovered Aspects of Fragile X Spur Next Wave of Drugs

September 27, 2018

Many drugs for Fragile X syndrome have failed in large clinical trials, but candidates that target new aspects of the condition may fare better.

Autophagy is a Novel Therapeutic Target of Impaired Cognition in Fragile X Syndrome

September 27, 2017

FRAXA’s $90K grant enabled Dr. Zukin to link impaired autophagy to Fragile X. Boosting autophagy restored synaptic proteins and reversed cognitive deficits in mice.

Biomarker Discovery and Validation for Fragile X Syndrome

March 24, 2017

This grant supported discovery of protein-based biomarkers for Fragile X to create objective outcome measures that translate from mouse studies to human trials.

PIKE as a Central Regulator of Synaptic Dysfunction in Fragile X Syndrome

February 2, 2017

With $255,000 from FRAXA Research Foundation, Dr. Suzanne Zukin at Albert Einstein College of Medicine studied signalling pathways in Fragile X syndrome.

Peter Vanderklish, PhD, at Scripps Research Institute, FRAXA research grant

Targeting AMP-Activated Protein Kinase Pathway in Fragile X Syndrome

September 13, 2015

With this grant from FRAXA, Dr. Peter Vanderklish explored AMPK activators to treat Fragile X. Both metformin and resveratrol, found in red wine, are AMPK activators.

Dr. Tom Jongens and Dr. Sean McBride study Fragile X Fruit Flies

Fruit Flies to Model and Test Fragile X Treatments

September 13, 2015

Boosting cAMP signaling restores memory and fixes brain-signaling defects in Fragile X models, suggesting diabetes drugs like metformin may help.

Effects of Metformin in Fmr1 Knockout Mouse Model of Fragile X Syndrome

April 8, 2015

Metformin, a safe diabetes drug, activates AMPK to rebalance protein synthesis. FRAXA-funded work investigated its potential to treat Fragile X.

Phase 1 Clinical Trial of Mega Green Tea Extract in Fragile X Syndrome

September 13, 2014

An early trial of green tea extract EGCG improved cognition in Fragile X. It targets ERβ and reduces overactive PI3K/mTOR/ERK signaling linked to FXS symptoms.

Iryna Ethell UC Riverside, FRAXA researcher

Fragile X Syndrome Treatment Target: MMP-9

July 24, 2014

Dr. Ethell was awarded FRAXA Research Foundation funding from 2008-2011 and 2012-present. This latest work shows that human Fragile X tissues have elevated levels of the extracellular enzyme MMP-9, as well as an increase in the active fraction of that protein (like most enzymes, MMP-9 can exist in an inactive form which can be switched on rapidly; this kind of regulation is important in most biological pathways.)

The mTOR Pathway in Fragile X Syndrome

September 6, 2013

FRAXA-funded research showed that blocking S6K1 in Fragile X mice improves social, behavioral, and physical symptoms.

Genetic and Pharmacologic Manipulation of PI3K Activity in FXS: Assessing Potential Therapeutic Value

September 2, 2012

Targeting the PI3K/mTOR cascade — specifically p110β — in Fragile X mice reversed neural and behavioral dysfunctions, validating it as a treatment pathway.