FRAXA Drug Validation Initiative (FRAXA-DVI)

FRAXA-DVI laboratory

Patricia Cogram, PhD
Principal Investigator

IEB, University of Chile

Robert Deacon, PhD
Co-Principal Investigator

University of Oxford

2021 Grant Funding: $60,000

Total Funding to date: $681,500

Summary

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing of potential Fragile X treatments. FRAXA-DVI uses in-vitro systems, behavior batteries, and gene expression and peripheral biomarker platforms to validate investigational new drugs and repurposed available compounds in Fragile X syndrome (FXS).

FRAXA Research Foundation has funded this resource every year since 2011.

FRAXA-DVI Team

Dr Patricia Cogram

Patricia Cogram, PhD

Dr Robert Deacon

Robert Deacon, PhD

The multidisciplinary team identifies novel compounds and biomarkers to bring forward new effective treatments for FXS.

Collaborators
  • Dr Paulina Carulo, FLENI Institute, Argentina – Biomarkers and pilot clinical trials in Fragile X
  • Sarah Lippe, Montreal, Canada – EEG studies
  • Peter Vanderklish, Scripps Institute, USA – OMICs
  • Julie Lauterborn, UCI, USA – Electrophysiology

Learn More About FRAXA-DVI

If you would like to learn more about working with or supporting FRAXA-DVI, please contact Patricia Cogram at FRAXA-DVI or Michael Tranfaglia at FRAXA.

Featured Treatment Development Programs and Clinical Trials Launched by FRAXA-DVI

This resource is having tremendous impact on the Fragile X field. FRAXA-DVI has tested over 100 compounds, generating new leads and enabling many development programs currently underway worldwide. Some programs have already launched clinical trials; others are in earlier stages along the drug development pipeline. Here are a few examples:

1

Preclinical

P70 S6 KINASE INHIBITOR – Epigen Biosciences
Preclinical studies are in progress.

Purposeful
This Greece-based company partners with FRAXA and FRAXA-DVI to identify drug repurposing candidates against Fragile X Syndrome.

Neuronascent
In partnership with FRAXA-DVI, this company is developing therapeutics aimed at neuron regeneration.

2

Phase II Clinical Trials

TROFINETIDE – Neuren Pharmaceuticals
Phase II Clinical Trial for Fragile X is complete; Trials in other rare diseases are underway.

Sulindac – Healx
Phase II Clinical Trial in Fragile X will launch in summer 2021.

3

Phase III Clinical Trials

BPN14770 – Tetra Therapeutics
Phase II Clinical Trial in Fragile X was successful, Phase III trial in Fragile X are now being planned

Highlighted Publications

The published article below are examples of some of the promising results generated at FRAXA-DVI. Many of these studies are leading to clinical trials of new treatments for Fragile X syndrome.

NNZ2591: common denominator to FXS, PTHS, AS, PWS and PMD

Hurley MJ, Deacon RMJ, Weistaub N, Sampieri G, Altimiras FJ, Angione C, Cogram P. Manuscript in preparation to be submitted to eLife.

Effects of the Sigma-1 Receptor Agonist Blarcamesine in a Murine Model of Fragile X Syndrome: Neurobehavioral Phenotypes and Receptor Occupancy

Reyes ST, Deacon RMJ, Altimiras FJ, Guo SG, Castillo JB, van der Wildt B, Morales AP, Park JH, Klamer D, Rosenberg J, Oberman LM, Rebowe N, Sprouse J, Missling CU, Cogram P, Kaufmann WI, Chin FT. Under Review, Scientific Reports, 2020

The cannabinoid-like compound VSN16R corrects behaviour in a mouse model of Fragile X syndrome

Hurley MJ, Deacon RMJ, Chan AWE, Baker D, Selwood DL, Cogram P. Under review, Brain, 2020

Altered gut microbiome composition in a Fragile X syndrome mouse model

Altimiras F, Garcia JA, Garcıa IP, Hurley MJ, Deacon RMS, Gonzalez B, Cogram P. Under review, Frontiers Neuroscience, 2020

Chronic bryostatin-1 rescues Autistic and cognitive phenotypes in the Fragile X mice

Cogram P, Alkon DL, Crockford D, Deacon RMJ, Hurley MJ, Altimiras F, Sun MK, Tranfaglia MR. Scientific Reports, 2020

Repurposing available drugs for neurodevelopmental disorders: The Fragile X experience

Tranfaglia MR, Thibodeaux C, Mason DJ, Brown D, Roberts I, Smith R, Guilliams T, Cogram P. Neuropharmacology. 2019

Gaboxadol Normalizes Behavioral Abnormalities in a Mouse Model of Fragile X Syndrome.

Cogram P, Deacon RMJ, Warner-Schmidt JL, von Schimmelmann MJ, Abrahams BS, During MJ. Front Behav Neurosci. 2019

Multiple Behavior Phenotypes of the Fragile-X Syndrome Mouse Model Respond to Chronic Inhibition of Phosphodiesterase-4D (PDE4D)

Gurney ME, Cogram P, Deacon RMJ, Rex C, Tranfaglia MR, Sci Rep. 2017

FRAXA-DVI laboratory

Explore Current Fragile X Research

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

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