In a recent study, researchers demonstrated a unique method that utilizes stem cells as targets for disease treatment. The study, led by Gabsang Lee, D.V.M., Ph.D., was performed at the Johns Hopkins University School of Medicine. Several years ago, Lee wanted to see if stem cells could be used to form specialized diseased cells, which would then serve as easy targets to evaluate the effectiveness of drug therapies. He investigated a patient with Riley-Day syndrome, a rare genetic nerve cell disease. Because these damaged nerve cells are essentially impossible to obtain directly, Lee instead extracted skin cells to use for analysis and test his hypotheses.¬†These skin cells were biochemically altered to form induced pluripotent stem cells (iPSCs) that have the potential to develop into any cell type in the body. Lee then induced the iPSCs to develop into nerve cells. Specifically, the DNA content of the IPSCs drove the formation of Riley-Day nerve cells. In the current study, the researchers used these damaged nerve cells to test various drugs, one of which (SKF-86466) showed promise of reversing the effects of Riley-Day syndrome. While this finding may lead to better treatment for the condition, the greater success of the study comes from the process that Lee and his colleagues demonstrated. In cases of genetic disease, cells from an easily accessible part of the body can be removed, converted to IPSCs, and induced to form the specific type of damaged cell type where they can be studied in culture. As a result, drug therapies can be cheaply and easily tested, avoiding the costly drug development model that is currently in place. This will especially broaden the study of rare diseases and individually tailored drug treatments, making these investigations significantly more convenient.