drug combinations Archives • Fragile X Research

Versatile Drug Screening Platform for Fragile X Syndrome

FRAXA Research Foundation 2022 Grants • Current Research Grants • Drug Repurposing • Kooy, Frank • University of Antwerp, Belgium • van der Lei, Mathijs November 2, 2022January 26, 2023drug combinations

Many experts believe that combinations of drugs may be needed to best treat Fragile X syndrome. How can we find the best combinations in the ideal doses? This project — a collaboration between a top university research team and an innovative AI startup both based in Belgium — tackles this challenge.

New Fragile X Clinical Trial Announced by Healx

Holly Roos Healx • Research Updates October 21, 2021November 22, 2021drug combinations, drug repurposing, FDA, FRAXA-DVI, Healx

Healx’s AI-driven approach makes finding the right combination therapies more efficient, cost-effective, and rapidly ready for testing at FRAXA-DVI. It was this successful process that has brought Healx to its recent announcement sharing that it has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for the Phase 2a clinical study of HLX-0201.

Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

FRAXA Research Foundation 2021 Grants • Coulson, Rochelle • Current Research Grants • Mourrain, Philippe Jacques • Stanford University • Treatment Targets • Wang, Gordon March 9, 2021March 10, 2021drug combinations, drug tolerance, Roche

FRAXA Research Foundation has awarded a $90,000 research grant to Stanford University principal investigators Dr. Philippe Jacques Mourrain and Dr. Gordon Wang, along with postdoctoral fellow, Dr. Rochelle Coulson. They are evaluating additive effects of combinatorial drug treatments to correct a broad spectrum of deficits observed in Fragile X syndrome.

Repurposing Study II: Evaluating Combinations of Drugs to Treat Fragile X

FRAXA Research Foundation 2016 Grants • 2017 Grants • 2018 Grants • Drug Repurposing • Healx • Research Grant Results February 8, 2018November 11, 2022drug combinations, drug repurposing, FDA, FRAXA-DVI, Healx

FRAXA Research Foundation initially partnered with Healx in 2016 to identify existing drugs with potential to treat Fragile X syndrome, using machine learning algorithms and computational biology. The study produced results, and now FRAXA and Healx have launched a new round of studies to evaluate combinations of compounds, including both drugs and natural products.

Combinatorial Drug Treatment in a Model of Fragile X Syndrome using Novel Biomarkers

FRAXA Research Foundation 2016 Grants • 2017 Grants • Biomarkers • Lovelace, Jonny • Razak, Khaleel • Research Grant Results • Treatment Targets • University of California at Riverside October 3, 2017May 15, 2020drug combinations, EEG, hypersensitivity, matrix metalloproteinase, mGluR5, minocycline, MMP9, sound, Tetra Therapeutics

With a $90,000 grant from FRAXA Research Foundation awarded over 2016-2017, University of California researchers Khaleel Razak, PhD, and Jonathan W. Lovelace, PhD, are exploring drug combinations to limit hypersensitivity to sounds in Fragile X mice.

Meltdown no more? Targeting Hypersensitivity in Fragile X

Theodore Coutilish Research Updates June 5, 2016December 26, 2019drug combinations, hypersensitivity, sound

We’ve all been there. Our child with Fragile X hears something and becomes excited. Very excited. Hand flapping follows with non-stop jumping and ear-piercing squawking. Nothing helps. No meds. No iPhone. No magic toy. Several minutes go by. Sometimes longer. How many times have you apologized in a grocery store — or restaurant — or at the mall? Wouldn’t it make our lives better if this unpredictable excitability was minimalized or eliminated? That’s the premise behind research being conducted at University of California, Riverside. Principal Investigator Khaleel Razak, PhD, and postdoctoral fellow Jonathan W. Lovelace, PhD, are studying mice genetically altered to mimic the genetic characteristics of humans with Fragile X Syndrome.

Why Did Fragile X Clinical Trials of mGluR Antagonists Fail?

Michael Tranfaglia, MD Research Updates • Trials and Studies August 15, 2014November 19, 2019AFQ056, arbaclofen, baclofen, drug combinations, drug tolerance, lithium, mGluR, mGluR5, minocycline, NIH, Novartis

by Michael Tranfaglia, MD. In my opinion, the Fragile X clinical trials of AFQ056 sponsored by Novartis failed because of a dose range that was inadequate for Fragile X, and because of the unexpected development of tolerance.

Baclofen: GABA(B) Receptor Supersensitivity and Normalization of Behavioral Abnormalities by Various GABA(B) Agonists Including Baclofen in FMRP Deficient Mice

FRAXA Research Foundation 2001-2005 Grants • 2006-2010 Grants • Completed Research Grants • Cornell University • Disease Mechanisms • Oh, Ji-eun • Toth, Miklos • Treatment Targets October 16, 2006January 16, 2019baclofen, drug combinations, drug tolerance, GABA, seizure, sound, startle

With $110,000 in grants from FRAXA Research Foundation over several years, Dr. Miklos Toth from Cornell University discovered increased startle response in Fragile X mice and that baclofen can correct this phenotype.