Alt Text
Office of Neuroscience Research > Did you know? > A promising future

A promising future

From the WashU Newsroom...

At Washington University, training the next generation of leaders in translational medicine is a key focus. “The young people in our labs are smart and dedicated, and they know that what we’re doing will change medicine — 20 years, 10 years, 5 years from now,” Skip Virgin says. “It’s actually already happening, and they’re trying to figure out how to do it yesterday.”

Cheryl Leyns and Tien-Phat “Phat” Huynh are two of the promising graduate students spread out across the School of Medicine. Members of David Holtzman’s lab, they research pathologies of Alzheimer’s disease. Alzheimer’s is the most common cause of dementia and currently affects an estimated 5.5 million Americans. While the exact cause of the disease is unknown, it is characterized by the appearance of two types of protein aggregates in the brain. The amyloid-beta protein clumps together to form plaques outside of brain cells, while the tau protein forms tangles within the cells. Together, these are thought to drive the loss of brain cells, or neurodegeneration, that occurs in Alzheimer’s patient brains and cause the steady cognitive decline that clinically characterizes the disease.

Leyns is a doctoral candidate in the Molecular Cell Biology program in the Division of Biology and Biomedical Sciences. In the lab, she focuses on the tau protein, a driver of Alzheimer’s that also contributes to 25 other neurodegenerative diseases. And she’s worked on two key projects: 1. investigating novel therapies, and 2. examining novel mouse models, studying the risk factor TREM2 and how it’s impacting tau pathology and tau-mediated neurodegeneration.

Leyns spent her first three years designing gene therapy approaches to deliver biologics, namely monoclonal antibody constructs, to target tau protein. The benefits of gene therapy are that researchers can get past the blood-brain barrier and deliver the drug directly to its target. Leyns says the research was exciting but also frustrating at times, when the research team experienced technical issues and setbacks along the way. Today, the monoclonal antibody, licensed now to AbbVie, is in Phase II clinical trials...

...Tien-Phat “Phat” Huynh, an MD/PhD student, has completed two years of medical school and three years of research in Holtzman’s lab. In the lab, Huynh has been working on the ApoE gene and its impacts on the production of amyloid beta (Abeta), which increases the risk for Alzheimer’s by building up in plaques in the brain.

Huynh is now revising a paper for submission to a major journal based on unexpected findings in his study.

The experiment hypothesized that an ASO (antisense oligonucleotide) drug, which Huynh designed with Ionis Pharmaceuticals, would target and reduce the ApoE gene, thus diminishing the amount of Abeta protein in the brain and reducing plaques. The findings, however, didn’t match the hypothesis.

Visit the Source for the complete story.