Auburn Using Social Media to Fund GM1 Research
Auburn University College of Veterinary Medicine, in cooperation with the Auburn University Foundation, is using a social media funding site to raise awareness and financial support for GM1 research.
Auburn is using CrowdRise to raise awareness – and hopefully financial support – for potentially life-saving research being conducted at the College of Veterinary Medicine’s Scott-Ritchey Research Center on GM1 gangliosidosis and to tell the story of 29-month-old Porter Heatherly, who has the fatal disease.
Associate Professor Dr. Doug Martin believes that if funding is raised through CrowdRise, there could be a breakthrough in treatment for the rare, fatal neurological disease. “If we could get started on the studies that could lead to eventual FDA approval, we believe we could see real results in clinical trials,” he said.
For decades, the Scott-Ritchey Research Center has researched GM1 gangliosidosis in felines, as GM1 occurs naturally in cats. Martin’s research has successfully treated cats who suffer from GM1.
Martin and his team have successfully extended the life expectancy of cats by more than five times compared to non-treated GM1 cats with the use of gene therapy, a non-harmful viral vector to produce enzymes missing in GM1 and Tay-Sachs-related diseases.
Martin’s goal is to model that same success in human clinical trials and, through research partners across the United States, successfully apply similar therapies that translate into human medicine.
“We believe 100 percent that the research we are doing will help children like Porter,” said Martin.
Porter, the son of Auburn alumni Sara and Michael Heatherly, was diagnosed with GM1 at four months old. GM1 is an inherited disease that progressively destroys nerve cells in the brain and spinal cord. There is no known cure and unfortunately for children like Porter, who have infantile-onset GM1, the life expectancy is just two years. Porter turned two Sept. 14. The Heatherlys hope Porter’s story will inspire support for Martin’s research.
Auburn is part of an international team – the Tay-Sachs Gene Therapy Consortium – established in 2007 by researchers from Scott-Ritchey, Cambridge University, the University of Massachusetts Medical School, Boston College, Massachusetts General Hospital and New York University. The consortium (www.tsgtconsortium.com) has pooled its efforts to devise the most effective gene therapy approach to treat Tay-Sachs disease and bring it into simultaneous clinical trials in the U.S. and U.K.
Martin believes the research is ready for clinical trials in humans, but before that can happen, the college must secure $500,000 in funding so Scott-Ritchey scientists can pursue research leading to studies needed for FDA approval.
Donations made through CrowdRise will be received by the Auburn University Foundation as tax-deductible contributions, and will be directed to fund GM1 research in the college.
For more information, go to Cure Tay-Sachs Foundation www.curetay-sachs.org and National Tay-Sachs & Allied Diseases Association, Inc. at www.ntsad.org