Greenwood, SC – GGC’s Associate Director of Research, Heather Flanagan-Steet, PhD, has been awarded a Translational Science Grant from the STAR (Salla Treatment and Research) Foundation. The $48,000 grant will support a yearlong project to screen zebrafish models for drugs to treat Free Sialic Acid Storage Disorder (FSASD).
FSASD is a group of rare genetic disorders caused by the accumulation of a specific sugar molecule called sialic acid. The accumulation of sialic acid in brain cells is thought to underlie the serious and irreversible damage to the central nervous system experienced by FSASD patients. Symptoms typically begin in infancy and progress to include developmental delay, seizures, and motor impairments.
Scientists in GGC’s Allin Aquaculture Facility have developed two lines of zebrafish which have variants in the SLC17A5 gene that cause sialic acid to accumulate in brain cells and lead to swimming defects in the affected fish.
“This project will allow us to test two drugs which are FDA-approved for use in other diseases, but that could potentially be repurposed to treat FSASD,” said Flanagan-Steet. “Our work will investigate if these drugs will improve the swim defects in the fish and provide information for future pre-clinical studies to determine if the drugs could eventually be beneficial for treating patients.”
“The STAR Foundation believes that no disease is too rare for viable research and treatment options. Through the generosity of our wonderful donors, we are delighted to establish this important partnership with Dr. Flanagan-Steet through the Translational Science Grant,” said STAR Foundation President Jessica Foglio. “This vital research allows families and patients to have hope for a brighter, healthier future.”
“It is always very meaningful to receive research funding from family-led organizations like the STAR Foundation who work tirelessly to raise money to fund research for a condition that is personal to them,” said Mike Lyons, MD, Chief Genomics Officer and Curry Chair in Translational Genomics and Therapeutics at GGC. “It is truly humbling to be the recipient of such grants and know that we have earned the trust of patients and their families. We are grateful that our work is providing hope to so many who are impacted by rare diseases like FSASD.”
About the STAR Foundation The STAR (Salla Treatment and Research) Foundation is a 501 (c) 3 tax-exempt charitable organization dedicated to supporting and advancing Salla disease treatments, research, education, awareness, and family networks. STAR was established in 2018 by Ben Foglio’s parents, Jessica and Michael Foglio, to accelerate the prospects for effective medical treatments for those affected with free sialic acid storage disorders including Salla disease. To learn more visit https://www.star-foundation.io/.