The Scott-Ritchey Research Center
The Scott-Ritchey Research Center is an integral part of Auburn University and the College of Veterinary Medicine and for more than 30 years.
THIS IS HOPE FOR A CURE.
For more than four decades, Scott-Ritchey researchers have sought a cure for GM1 gangliosidosis, a rare, neurodegenerative disease found in animals and humans. Today, Dr. Doug Martin and a team of Scott-Ritchey Research Center scientists believe they have found a cure for the disease. His research, an extension of work that began by mentors Dr. Henry Baker and the late Dr. Nancy Cox, is a gene therapy to produce enzymes found missing in GM1 and Tay-Sachs-related diseases.
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.
“There is no question now the disease can be treated successfully,” Dr. Martin said. “We have seen how it has woreked in cats with the disease and how it is has extended their lives as healthly animals.”
Now, the goal is human clinical trials, slated for 2017. The Tay-Sachs Gene Therapy Consortium, an international collaborative group of scientists committed to searching for a cure, have developed and tested the vector based on the adeno-associated virus (AAV). Read about the most recent research effort.
In 2013, Michael and Sara Heatherly, both Auburn alumni, received the devastating GM1 diagnosis for their son, Porter, at just four months old. Soon after, they learned of the ongoing research at Scott-Ritchey and believe that their Auburn Connection can be a lifesaving cure. Sadly, Porter Heatherly died on Nov. 10, 2016, but the fight continues. Scientists believe they will be in clinical trials in 2017 with a treatment.
Now, a second child in Alabama was diagnosed with GM1. Meet Clara, the daughter of Ryan and Jenny Bragg of Hoover, is spunky and full of life. Diagnosed with juvenile GM1, the Braggs are on a mission to raise money for the clinical trial, which they believe will help Clara. Watch their video.
Read the latest about families raising money to find a cure for GM1.
How can you help?
Make a donation to support GM1 research online through the Auburn University Foundation. Gifts given to support GM1 research will be designated to “Porter’s Fund” in honor and memory of the late Porter Heatherly, who passed away Nov. 10, 2016 from GM1.
Or, gifts by mail should be made out to Auburn University Foundation and sent to the attention of:
Auburn University Foundation
Attn: Gift Processing
RE: Scott-Ritchey GM1 Research
317 South College Street
Auburn, Alabama 36849