Senior Yuri Kwon has been a contributor to the ALS research in Peter King's lab for the past two and a half years. King has hosted many undergraduates in his lab, and Kwon "is one of the most dedicated students I've worked with," he said.For three decades, Peter King, M.D., has been treating patients with ALS — amyotrophic lateral sclerosis — also known as Lou Gehrig’s disease. During that time, research has revealed many secrets of this rare but fatal condition, which affects about 30,000 Americans, including identifying dozens of genetic mutations that seem to be involved. But inherited ALS only affects some 10 percent of patients; the rest are non-hereditary cases with no specific known cause. And for all patients, there are few treatment options. Only two drugs are FDA-approved for treatment “and neither is very effective,” said King, professor and vice chair of the UAB Department of Neurology and Chief of Neurology for the Birmingham Veterans Affairs Medical Center. “There’s nothing worse than going to see patients and having to tell them, ‘There’s nothing I can do to stop the disease.’”
There are few answers when it comes to prognosis as well. ALS comes in two varieties: fast-progressing and slow-progressing. Some patients deteriorate and die within a year or two of diagnosis, such as New York Yankees greats Lou Gehrig in the 1940s and Jim “Catfish” Hunter in the 1970s. Others follow the path more like that of physicist Stephen Hawking, who lived for 50 years after he was diagnosed with ALS in the early 1960s. In a project that began early this decade, King has been looking for predictors of the different types of ALS and what these biomarkers “can tell us about whether the disease will grow rapidly or slowly in an individual patient,” he said.
Remarkable success
King initiated the ALS biomarker project eight years ago using muscle samples from ALS patients treated at UAB. Using the latest technology in DNA sequencing, his research team identified more than 50 novel genes with the potential for being ALS biomarkers. Since then, the researchers have found that many of these biomarkers track disease progression in the mouse model of ALS. The work has enabled King to receive funding from the National Institutes of Health and the Department of Veterans Affairs to advance the biomarkers into the clinic.
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| “One metric of her success is the number of papers she has co-authored," King said. "It’s pretty remarkable for an undergraduate student to be on a publication, and Yuri has already been on three now.” |
“This is a great example of how a research idea is developed,” King said. Initiation of the project was made possible by seed money from philanthropy through UAB Advancement office, he noted. “The project then gained sufficient momentum to achieve federal funding.” The most recent grant award will validate these biomarkers in a large cohort of ALS patients from a national biorepository at the University of Miami. “I am excited about this project because an effective molecular marker would allow us to test new treatments more efficiently and effectively,” King said. “The biomarkers also provide clues to the underlying disease mechanisms and may lead to additional treatments.”
‘A great experience’
For the past two and a half years, Yuri Kwon has been one of the key contributors to the work. Kwon, a UAB senior majoring in biology and minoring in chemistry, also is a member of the Science and Technology Honors Program. King has hosted many undergraduates from the SciTech program, part of the UAB Honors College, in his lab. Kwon “is one of the most dedicated students I’ve worked with,” he said. “One metric of her success is the number of papers she has co-authored. It’s pretty remarkable for an undergraduate student to be on a publication, and Yuri has already been on three now and will probably be on one more. SciTech students are a premier group at UAB, but Yuri has been particularly successful and dedicated to becoming a scientist.”
Kwon, who was born in Seoul, South Korea, and went to high school in Madison, Alabama, has been selected to present her research at the American Academy of Neurology annual meeting in May. In the fall, she will begin work on a master’s degree in public health from the UAB School of Public Health and start applying to medical schools.
During her time in King’s lab, “I’ve learned to do a little bit of everything,” Kwon said. “I’m working with muscle samples at different time points, which allows us to see the changes in the biomarker expression as the disease progresses.” The biorepository of samples from ALS patients that King maintains allows the work to cross from the lab to the clinic. “I can do anything from genotyping, harvesting tissue to RNA and protein assays,” Kwon said. “It’s been a great experience.”
Training a new generation
A new endowed scholarship fund, created to honor one of the founders of UAB’s ALS program, will help additional undergraduates take part in groundbreaking research in the field, King noted. The Gwen Claussen, M.D., ALS Research Fund was created to honor the first director of the ALS Clinic at UAB, who trained generations of medical students in more than two decades at UAB. She was killed in a car accident in 2018.
| To donate to the Claussen ALS Research Fund, visit www.uab.edu/give/claussen. |
“We started the scholarship because of Gwen’s passion for teaching,” King said. The fund will provide promising young students the opportunity to explore medical research early in their careers, thus igniting their interests in ALS, King explained. There are plenty of burning questions to be solved: Why are military veterans diagnosed at twice the rate of those who haven’t served? Why is there an increased risk of ALS among endurance athletes? “No one knows what the trigger is,” King said. “This is a complex disease, and it’s going to take many minds coming together to figure it out.”
The Claussen scholarship is halfway to its funding goal, King said. “We want to train future generations of scientists and get them involved in basic biomedical research.”
