This article by Spectrum, “Newly discovered aspects of fragile X spur next wave of drugs“, presents several potential treatments based on new discoveries beyond the mGluR theory which launched large scale clinical trials several years ago. “Researchers began looking at other proteins and pathways that may offer targets to treat Fragile X — including those that control the birth of neurons and fine-tune the strength of synapses.”
Four scientists featured in this article began their Fragile X studies with FRAXA funding:
- Dr. Gary Bassell was funded from 2001-2012, first at Albert Einstein College of Medicine and then at Emory University School of Medicine, to explore the PI3K/mTOR signaling complex as a therapeutic target.
- Dr. Mark Bear, Picower Professor at Massachusetts Institute of Technology (MIT), is a member of our Scientific Advisory Board and has received several FRAXA grants up to the present. His lab also fielded two teams in the inaugural FRAXA Biotech Games.
- Dr. Emily Osterweil began her Fragile X career as a FRAXA Fellow in Dr. Bear’s lab. She won a FRAXA Pioneer Award for work demonstrating that lovastatin could reverse signs of Fragile X in mice. She is now pursuing further studies at her own laboratory at the University of Edinburgh.
- Dr. Xinyu Zhao received a FRAXA Grant in 2012-2013 and that led to a large grant from the John Merck Fund ($1 million over 4 years), showing once again how targeted pilot funding can open doors to support from additional sources.
It’s great to see all of these dedicated researchers continue to pave the path to effective treatments and ultimately a cure for Fragile X.