baclofen Archives

20 Years of Advancing Fragile X Research: Progress Toward a Cure

FRAXA Research Foundation Bear, Mark • Research Updates September 13, 2021November 22, 2021arbaclofen, baclofen

Dr. Mark Bear joined the Fragile X field in 1999 when he received a research grant from FRAXA Research Foundation. At the time, we recognized the symptoms of Fragile X, and we knew its cause: a single missing protein. But we knew very little else. Dr. Bear traces the discoveries that now give us great optimism of finding effective treatments and ultimately a cure for Fragile X.

Developing Arbaclofen for Fragile X – Dr. Mark Bear 1:1 with FRAXA

FRAXA Research Foundation 1:1 with FRAXA • Bear, Mark • Research Updates December 9, 2020May 21, 2021arbaclofen, baclofen

Seven years ago, arbaclofen (STX209) was pulled from development, disappointing families around the US. Now MIT professor and FRAXA Investigator Dr. Mark Bear has founded Allos Pharma to bring it back. Dr. Bear sat down with FRAXA co-founder Katie Clapp to share the story and next steps.

Arbaclofen Returns After 7 Years!

FRAXA Research Foundation Bear, Mark • Research Updates November 19, 2020November 22, 2021arbaclofen, autism, baclofen, GABA, hyperexcitability

Allos Pharma Inc, a late-stage pharmaceutical company developing therapeutics for neurodevelopmental disorders, has announced the exclusive license rights on IP and documentation for arbaclofen in fragile X syndrome (FXS).

Healx Raises $56M to use AI to Find Treatments for Fragile X & Other Rare Diseases

FRAXA Research Foundation Drug Repurposing • Healx October 16, 2019October 16, 2019baclofen, drug repurposing, FRAXA-DVI, Healx, lithium, lovastatin, metformin, minocycline

Healx has secured $56M in new financing to build a clinical-stage portfolio for rare diseases, including treatments for Fragile X syndrome, and to launch a global Rare Treatment Accelerator program. Where the traditional drug discovery model takes more than a decade and can run into the billions of dollars, Healx’s AI-driven approach makes the process faster, more efficient and cost-effective.

Pharmacological Tolerance in the Treatment of Fragile X Syndrome

FRAXA Research Foundation 2018 Grants • 2019 Grants • 2020 Grants • Bear, Mark • Current Research Grants • Massachusetts Institute of Technology (MIT) • McCamphill, Patrick • Treatment Targets May 9, 2018May 21, 2021AFQ056, arbaclofen, baclofen, drug tolerance, GABA, GSK3, lithium, mGluR5, Novartis, Roche, seizure

With a $90,000 grant from FRAXA Research Foundation over 2018-2019, Dr. Patrick McCamphill, postdoctoral fellow in Dr. Mark Bear’s lab at Massachusetts Institute of Technology (MIT), is investigating drug tolerance to mGluR5 antagonists, arbaclofen, and other potential Fragile X treatments. He is also exploring ways to overcome it.

Repurposing Available Drugs to Treat Fragile X Syndrome – FRAXA Initiatives

Michael Tranfaglia, MD Drug Repurposing • Research Updates February 10, 2017December 26, 2019arbaclofen, autism, baclofen, drug repurposing, FRAXA-DVI, glutamate, GSK3, Healx, hypersensitivity, lithium, lovastatin, MAP1b, matrix metalloproteinase, metformin, mGluR, minocycline, NIH

FRAXA Research Foundation was founded in 1994 to fund biomedical research aimed at finding a cure for Fragile X syndrome and, ultimately, autism. We prioritize translational research with the potential to lead to improved treatments for Fragile X in the near term. Our early efforts involved supporting a great deal of basic neuroscience to understand the cause of Fragile X. By 1996, these efforts had already begun to yield results useful for drug repurposing. To date, FRAXA has funded well over $25 million in research, with over $3 million of that for repurposing existing drugs for Fragile X.

NIH Investigator Carolyn Beebe Smith, PhD, Looks to Improve Sleep in Fragile X Syndrome

Theodore Coutilish Research Updates • Smith, Carolyn B. November 17, 2016December 26, 2019Alzheimer's, autism, baclofen, FDA, lithium, NIH, sleep

Our sons with Fragile X Syndrome typically go to bed early and rise early. Sometimes they jump on us while we are sleeping at 3 a.m., excited to start their day. For heaven’s sake, why? The answer may come from Carolyn Beebe Smith, PhD, senior investigator, Section on Neuroadaptation and Protein Metabolism, National Institute of Mental Health, National Institutes of Health, Bethesda, Maryland. She is studying why children, in particularly boys, with FXS have problems sleeping.

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.

Social Behavior as an Outcome Measure for Fragile X Clinical Trials

FRAXA Research Foundation 2012 Grants • 2013 Grants • Clinical • Drug Repurposing • Erasmus University Rotterdam • FRAXA Research Grants • Research Grant Results • Treatment Targets • Willemsen, Rob September 13, 2013February 17, 2021AFQ056, AMPA, AMPAkines, autism, baclofen, ERK, mGluR5, minocycline, NMDA, Novartis

One of the features of the Fragile X mouse model which is relevant to the human Fragile X syndrome (and autism) is social behavior. Several tests show consistent social behavioral abnormalities in the Fragile X mouse model. With a $140,000 grant from FRAXA Research Foundation in 2012-2013, Dr. Willemsen at Erasmus University used social behavior tests to measure the effectiveness of several drug strategies.

Phase 2b Clinical Trial of Arbaclofen in Autism Has Disappointing Results

FRAXA Research Foundation Research Updates May 14, 2013December 13, 2019arbaclofen, autism, baclofen

Phase 2b clinical trial of Arbaclofen in Autism Seaside Therapeutics reports the study did not show improvement on the primary endpoint of social withdrawal, but it did demonstrate improvement on the Clinical Global Impression of Severity scale.

Genome-wide Epigenetic Markers in Fragile X

FRAXA Research Foundation 2006-2010 Grants • Completed Research Grants • Cornell University • Disease Mechanisms • Oh, Ji-eun • Toth, Miklos • Treatment Targets February 5, 2009January 16, 2019baclofen, epigenetics, miRNA, sound, startle

With $45,000 in grants from FRAXA Research Foundation over several years, Dr. Miklos Toth of Cornell University studied epigenetics (ie factors other than the gene itself) which can determine symptom severity in Fragile X.

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.