Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is the most common motor neuron disease worldwide. It affects as many as 30,000 people in the United States, with 5,000 new cases diagnosed each year.
Lynn George, associate professor of Molecular Biology at Montana State University Billings, says ALS patients lose the connections between the muscles and the neurons that innovate those muscles.
“And without that information and use they begin to waste away," she said. "So, patients gradually lose their ability to eat, to speak, and even their larger motor functions like breathing become difficult.
"I don't know from the science perspective, but of all the motor neuron diseases, it seems to be incredibly awful.”
George and three research students were initially conducting research on a disease related to ALS — called familial dysautonomia
— when they noticed something surprising.
“The two diseases overlap in that the same molecular complex goes haywire," she said. "We initially thought that it might be a contaminant and that it maybe was a mistake, essentially.”
“But, with lots of follow up experiments, we one hundred percent validated that this protein is present in neurons and there are some characteristics about the protein that make it a particularly interesting candidate for having a role in ALS.”
It is a role that provides crucial insight into the disease by helping researchers understand the cause of ALS.
“Therapeutic design is based on understanding the pathways that cause disease, and if you don't understand those pathways, you can't design therapeutics," George said. "So, the more we understand about the molecular pathways that go wrong inside the neuron, then the more effective therapeutics we can design for tackling it.”
George received grants totaling $385,000 to expand her research on ALS from the National Institutes of Health and the National Institute of Neurological Disorders and Stroke. Her grant proposal scored in the top one percentile, which is laudable, given the highly competitive nature of NIH funding.
The next step for George is collaborating with other researchers.
“We are actually making a mouse model where this protein of interest will be knocked out, is what we say in the field. And so, it will be a new model for ALS," she said. "Using that model, we can verify the connection between this protein and the disease, and then potentially it could be used for testing and potential therapeutics as well.”
George says as she learned about the underlying kind of molecular mechanisms that are contributing to both diseases, she was compelled to start working more on a disease that impacts more people.
“I think it's every scientist's goal to make a discovery, to see something that has never been seen before or to make a connection," she said, "especially in the medical field that may actually make a difference in treating a disease like ALS.”
Yellowstone Public Radio is licensed to MSU Billings.
