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Stem Cell Models for Alzheimer’s Disease Enable Drug Discovery and Development

Feb 3, 2016 1:00:37 PM / by Karina Palomares

 Neurons derived from patient iPSCs would provide a more relevant model for Alzheimer's disease. Image credit: evaluate new treatments of Alzheimer’s disease on neurons derived from iPSCs from patients.

Alzheimer’s disease is the leading cause of dementia, and has affected approximately 48 million people worldwide. Despite decades of research, we have yet to come up with a successful treatment. One major challenge in drug development for neurological diseases is access to a clinically relevant disease model. As stated in a recent WSJ article, results from animal testing often do not predict well what will happen in humans [1].  As a result, many promising drug candidates have been removed from further development because of safety concerns or lack of efficacy.

Scientists are shifting toward using stem cells for Alzheimer's disease to overcome some of the limitations of current disease models. The ability to derive neurons from human induced pluripotent stem cells (hiPSCs) from patients with Alzheimer’s disease would provide a more relevant model for preclinical drug discovery and development. Current stem cell research models allow more efficient testing of experimental drugs in the lab rather than in costly, time-consuming clinical trials.

Scientists at UCSD have developed a stem cell model to study Alzheimer’s disease [2]. They examined neurons derived from hiPSCs from patients carrying mutations in presenilin 1, a gene involved in the majority of early onset, familial Alzheimer’s disease. Their model identified a new biomarker signature that differs from other models of this disease, and they expected it to more accurately reflect drug response in patients. Indeed, testing of one experimental drug that was previously withdrawn from a clinical trial revealed that the dosage used in the trial was likely ineffective. Thus, in vitro testing of drugs in stem cell-derived neurons could potentially identify accurate doses for clinical trials, as well as reveal which compounds should not move forward in development.

As demonstrated above, patient-specific stem cells are well-suited for generating disease models. We at HemaCare look forward to the application of these models to understanding disease pathogenesis, as well as to the development of novel therapies. To learn more about the various stem cell types for your research purposes, contact us today at (877) 397-3087.



[2]       Liu, Q. et al. Effect of potent gamma-secretase modulator in human neurons derived from multiple presenilin 1-induced pluripotent stem cell mutant carriers. JAMA neurology 71, 1481-1489, doi:10.1001/jamaneurol.2014.2482 (2014).




Topics: Drug Discovery, Stem Cells

Karina Palomares

Written by Karina Palomares

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