Another FRAXA research grant kicks off major funding aimed at curing Fragile X syndrome. In 2023, FRAXA awarded Dr. Raymond Turner’s lab at the University of Calgary $100,000 to refine their groundbreaking approach to replacing the missing Fragile X protein, FMRP. The Canadian Institutes of Health Research (CIHR) has just awarded Dr. Turner’s lab $1 million CAD over the next five years to accelerate this promising work.
From Vein to Brain: Delivering FMRP to Reverse Fragile X
FRAXA has supported Dr. Ray Turner’s lab at the University of Calgary in Alberta, Canada, since 2018. The team’s innovative approach focuses on replacing FMRP, the protein missing in Fragile X, with a modified version designed to safely and effectively restore brain function.
With their first FRAXA grant, Dr. Turner and his colleagues developed a unique system to transport a version of FMRP across the blood-brain barrier in mice. A shortened version of FMRP is coupled with a small protein called Tat, which acts as a delivery system, carrying the therapeutic protein to the brain. Using this combination is less toxic and more efficient than attempting to replace the full-length FMRP protein.
Dr. Turner and FRAXA Postdoctoral Fellow Dr. Xiaoqin Zhan showed that injecting the Tat-FMRP protein into a mouse tail vein rapidly delivers it to the brain, where it restores synaptic plasticity and reduces hyperactivity for at least 24 hours! Mice treated with the Tat-FMRP protein showed behavioral improvements at low doses and experienced no toxic effects, even at much higher doses.
Building on this success, FRAXA awarded the Turner lab another grant for 2023–2025 to refine and optimize their curative therapy for Fragile X syndrome: the Tat-FMRP protein delivery system.. The team is now testing the effects of these peptides on cortical circuit function by recording EEG over several days in the mice, as a potential biomarker for FXS.
Scaling Up Fragile X Syndrome Research with CIHR Funding & Industry Support
Two developments are moving this Fragile X treatment approach forward:
- A new company, Bowen’s FX Therapeutics, has been formed in Canada by Jeff Parr and named after his grandson Bowen, who has Fragile X. Their goal is to develop and commercialize this therapy.
- The Canadian Institutes of Health Research (CIHR) has awarded the Turner lab a $1 million (CAD) grant over five years to expand and accelerate their groundbreaking work.
FRAXA research grants can provide a big return on investment, leveraging much larger funding opportunities to move the field forward to improve the lives of families affected by Fragile X!
