Alzheimer’s disease is the most common form of dementia, affecting memory, thinking and behavior. According to the Alzheimer’s Association, over 5 million Americans suffered from Alzheimer’s in 2015, and numbers are expected to grow.
Despite decades of tremendous effort, researchers have yet to come up with a successful treatment for Alzheimer’s, as many promising drug candidates have fallen by the wayside due to safety concerns or lack of efficacy. Some researchers have attributed these failures to limitations of the models used to study the disease—mainly transformed cell lines and animals that have been engineered to overexpress proteins implicated in the disease.
A recent article in the Wall Street Journal addressed this issue, stating, “Mice and other lab animals make for poor models in which to test new medications in many neurodegenerative disease like Alzheimer’s. Lab animals don’t get these types of diseases naturally, and the disease that scientists have genetically engineered into mice, for instance, translates imperfectly into what happens in humans.”
As a result, many researchers in the field are shifting toward use of stem cells in their Alzheimer’s research to overcome some of the limitations with existing models. The ability to derive neurons from human induced pluripotent stems cells from patients with Alzheimer’s and individuals without dementia has given researchers a useful and human-relevant model for disease modeling as well as preclinical drug discovery and development.
Use of these models has already provided valuable insight into the biological changes that take place within neurons in individuals with Alzheimer’s to help explain how the disease develops and progresses. These approaches offer a number of advantages over animal models used to study the disease—including human-relevance and improved recapitulation of disease pathology—while producing results more quickly, cheaply and humanely than animal models.