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2014 Grants - Yuan
“In Vitro Clinical Trial” with GSM for the Treatment of FAD Carriers
Shauna Yuan, M.D.
University of California, San Diego
San Diego, California
2014 New Investigator Research Grant
Several potential treatments for Alzheimer’s disease have been studied in human clinical trials after showing promising results in the laboratory. However, to date none of the potential treatments have been successful in Phase III human trials. One possible interpretation of these events is that better models are needed to identify and test potential therapies before moving to clinical testing in humans.
Shauna Yuan, M.D., and colleagues have developed a new model for testing potential drug therapies; this new model may give a more accurate assessment of some of the effects of drugs on human nerve cells. The model is referred to as an ‘in vitro’ model, indicating that the testing occurs outside the body.
The in vitro model developed by Dr. Yuan and colleagues involves collection of stem cells from the skin of people who have genetically inherited forms Alzheimer’s, known as familial Alzheimer’s disease (FAD). The researchers are then able to convert these stem cells into nerve cells in the laboratory, and then use those nerve cells to study potential drug therapies.
Dr. Yuan’s team has proposed to use their in vitro model to test a series of drugs known as gamma-secretase modulators (GSMs). Gamma-secretase is a protein that performs one of the critical steps in the production of beta-amyloid, a protein fragment that accumulates into amyloid plaques, one of the characteristic features Alzheimer’s disease in the brain. Using this model, the researchers hope to identify GSMs that are highly suited for further testing in clinical trials of people with Alzheimer’s disease. These studies may improve the efficiency and overall success of the scientific process for discovering and developing new treatments for Alzheimer’s disease.