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Study advances use of stem cells in personalized medicine
Date:11/26/2012

grammed to analyze the effects, the researchers quickly identified eight compounds for further testing, of which one SKF-86466 ultimately showed promise for stopping or reversing the disease process at the cellular level.

Lee says a clinical trial with SKF-86466 might not be feasible because of the small number of Riley-Day patients worldwide, but suggests that a closely related version of the compound, one that has already been approved by the U.S. Food and Drug Administration for another use, could be employed for the patients after a few tests.

The implications of the experiment reach beyond Riley-Day syndrome, however. "There are many rare, 'orphan' genetic diseases that will never be addressed through the costly current model of drug development," Lee explains. "We've shown that there may be another way forward to treat these illnesses."

Another application of the new stem cell process could be treatments tailored not only to an illness, but also to an individual patient, Lee says. That is, iPSCs could be made for a patient, then used to create a laboratory culture of, for example, pancreatic cells, in the case of a patient with type 1 diabetes. The efficacy and safety of various drugs could then be tested on the cultured cells, and doctors could use the results to help determine the best treatment. "This approach could move much of the trial-and-error process of beginning a new treatment from the patient to the petri dish, and help people to get better faster," says Lee.


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Contact: Shawna Williams
shawna@jhmi.edu
410-955-8236
Johns Hopkins Medicine
Source:Eurekalert

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