December 6, 2012

"Disease in a Dish"


The latest episode of Developments to Watch, a collaborative video series produced byMedscape, is now available online: Disease in a Dish: The Ultimate Personalized Medicine.
In the video, Sanford-Burnham CEO John Reed, M.D., Ph.D., talks to Michael Jackson, Ph.D., vice president of drug discovery and development, about the Institute’s work on creating personalized “disease in a dish” models using stem cells derived from patients. They also talk about drug repurposing—finding new applications for existing therapeutic drugs in order to get treatments to patients faster.
Here’s an excerpt:
Reed: Tell us about this new breakthrough in stem cell technology that allows you to create “disease in a dish” models using cells derived from stem cells. What is it all about?”

Jackson: It’s a very exciting new advancement. It allows us to take patients’ own cells and, if a patient has a disease that affects those cells, to measure that defect. This is done through advances in quantitative microscopy, mixed with or alongside of robotic drug screening; the two together are quite a spectacular shortcut to be able to see whether a medicine we already have today that is being prescribed for one disease might have utility in another.
Now, you say, how could that be? There are a number of very spectacular examples out there of drugs that have been used or developed for one indication and then later on have been found to have a different clinical utility. In the specific example we’re talking about here with disease in a dish, we’re initially focusing on rare diseases. With rare diseases, the children’s cells all have a particular defect, and it’s that defect that we can actually recapitulate and rebuild in a dish, the so-called disease in a dish. Then, using robotics and screening technology and using the kind of equipment you’re seeing behind you here, to search in a systematic way and see whether any preexisting drugs that are already approved could move the needle and improve the defect in the child’s cells.

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