NSU students, professor part of research that could lead to cure for Huntington’s
June 13, 2018
ABERDEEN, S.D. –While her friends are working typical summer jobs, Northern State University student Cindy Venegas is spending her summer conducting research for a project that could lead to a cure for Huntington's disease.
"It's very cool," said Venegas, an Aberdeen native pursuing a bachelor's degree in biology and an associate in biotechnology. "It's amazing to be able to do research as a sophomore in college."
"That's an opportunity unique to NSU," said Dr. Jon Mitchell, associate professor of biology, who is working with Venegas. "That's the Northern advantage."
Mitchell and Venegas, along with NSU senior Zach Fleming, have been working for the past year to purify the sheep ganglioside GM1, which has been found to impact the effects of Huntington's disease and potentially other neurodegenerative diseases.
“That’s an opportunity unique to NSU. That’s the Northern advantage. It doesn’t happen very often that students get the opportunity to get a little bit of a wage and do some hard science. This is not easy stuff and no one’s doing it.”Dr. Jon Mitchell, NSU associate professor of biology
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It's advanced research, Mitchell said, and it's funded thanks to a South Dakota EPSCoR (Established Program to Stimulate Competitive Research) grant that pays for wages, some research equipment and consumable materials.
"It doesn't happen very often that students get the opportunity to get a little bit of a wage and do some hard science," Mitchell said. "This is not easy stuff and no one's doing it."
The work is a collaborative effort with GlycoScience Research Inc. (GRI), located near Brookings. GRI founders, Dr. Larry Holler, DVM, Ph.D., in the Veterinary and Biomedical Sciences Department at South Dakota State University, and Sue Holler, diagnostic specialist at SDSU, have been working on these sheep since 1998. They have identified a new sheep source for production of GM1 gangliosides that will exceed normal levels 30 to 40 fold, providing needed raw materials for downstream pharmaceutical applications against Huntington's disease, Parkinson's disease, Alzheimer's and dementia.
Patients with Huntington's disease are deficient in GM1 ganglioside, but the Hollers and GRI have discovered that treating Huntington's patients with this GM1 ganglioside has shown to drastically reduce its symptoms to the point of cure.
"These guys are trying to cure Huntington's," Dr. Mitchell explained, adding, "They want to convince the FDA that GM1 could be the answer." Additionally, this GM1 restoration may have a similar effect on patients with Parkinson's, Alzheimer's and dementia.
Dr. Mitchell partnered with the Hollers and GRI through I-Corps, a NSF funded program where faculty and undergraduates partner with a business to develop and execute a plan to generate innovative new technology.
In addition to the SD EPSCoR grant, Mitchell also credited Mel Ustad at the South Dakota Governor's Office of Economic Development (GOED) for professional support. This project also involves collaborations with Avanti Polar Lipids and Dr. Dan Engebretson, chair of the Biomedical Engineering Program at the USD Graduate Education and Applied Research (GEAR) Center in Sioux Falls.
Working Toward a Cure
While this treatment is in very early stages, its impact is certain.
"It is a cure," Venegas and Mitchell both said.
The Hollers are continuing their work toward getting GM1 approved by the Food and Drug Administration as a treatment. Mitchell and his students' involvement is more peripheral.
"Our big project is not to cure Huntington's. Our contribution consists of working on another important component: isolating and quantifying the GM1 ganglioside from afflicted/non-afflicted lamb muscle to definitively show no significant differences in GM1 levels between the two meat sources – basically, showing that the GM1 afflicted lamb meat would be safe to consume. Therefore, their data would support advocacy of GM1 lamb meat to be marketed through normal, USDA/FDA approved channels."
They're using a couple of different monitoring approaches. In one, they use a fluorescent molecule that will bind to the GM1 to quantify the amount. The other method is standard biochemistry, Mitchell said, monitoring levels using NSU's new nuclear magnetic resonance (NMR) spectrometer. This piece of equipment was purchased thanks to a $52,991 Research and Development Innovation Grant from the South Dakota Board of Regents.
The GM1 project is ongoing, and they're hoping EPSCoR funding will continue. They're also hoping to gather data and eventually send Venegas to a nationwide meeting to present on the project.
Other future possibilities include actually making GM1. Only a sugar separates GM1 from other gangliosides, Venegas said, so if they can isolate the GM2 or GM3 gangliosides and move some of the sugars around, they might be able to make GM1.
Mitchell said their work has involved picking up blood samples from a sheep farmer in Forbes, N.D., and it's great that the students are getting to see all aspects of research.
"You get to go out in the field, and you get to see it in the labs, and then you get to see the potential for helping folks," he said. "You get to see the end product."
Research Will Help with Career
Impacting people's health is amazing, Venegas said, and is in the area of what she wants to do for a career. She hopes to attend the University of California, Berkeley, pursuing a master's degree and Ph.D. in biological engineering, and possibly work in pharmaceuticals.
Her undergraduate research at Northern has given her a great start – and it's an opportunity not found at all colleges.
"In bigger schools, you don't really see that," Venegas said, adding, "Actually doing hands-on stuff is pretty great."
– Northern State University