Auburn veterinary medicine uses online giving to fund GM1 research

Published: February 27, 2015
Updated: March 02, 2015
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AUBURN UNIVERSITY – The Auburn University College of Veterinary Medicine, in cooperation with the Auburn University Foundation, is using a crowdsourcing site to raise awareness and financial support for its GM1 research.

Associate Professor Dr. Doug Martin said he believes that if funding is raised through the site, 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 in Auburn's College of Veterinary Medicine has researched GM1 gangliosidosis in felines, as GM1 occurs naturally in cats. The goal is to find a cure for feline GM1 and, through research partners across the United States, successfully apply therapies that translate into human medicine.

Martin and his team at Scott-Ritchey have successfully extended the life expectancy 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.

Treated cats are living years past the life expectancy of non-treated cats and their survival time continues to increase. Martin's goal is to model that same success in human clinical trials.

"We believe 100 percent that the research we are doing will help children like Porter Heatherly," said Martin.

Porter Heatherly, the son of Auburn alumni Sara and Michael Heatherly, has GM1 gangliosidosis, 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 2 on 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 United States and the United Kingdom.

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 at https://www.crowdrise.com/offerporterhopecureg/fundraiser/auburnuniversityfoun or through the Scott-Richey Research Center at www.vetmed.auburn.edu/srrc 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 at www.curetay-sachs.org and National Tay-Sachs and Allied Diseases Association Inc. at www.ntsad.org.

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