Near-term use for iPS cells: repurposing existing drugs

George Daley opened the World Stem Cell Summit in West Palm Beach, Florida

Geoff Lomax is CIRM’s Senior Officer to the Standards Working Group

Dr. Daley kicked off the World Stem Cell Summit by describing the strengths and opportunities for the field. To illustrate near-term strengths, he described his research on  a disease that results in bone marrow dysfunction — Shwachman Diamond Syndrome. They used cells taken from people with the disease and turned those into an embryonic-like stem cell called an iPS cell to study in a lab dish how this disease can result in bone marrow failure.

Based on what this research taught them about how the disease forms, they found an existing drug that could prevent the disease from taking place in those cells in the lab dish. These studies suggest that the drug may be a good therapy for Shwachman Diamond Syndrome patients. His group is now trying to learn whether the drug that works so well in the lab dish might be able to treat the disease in people.

Dr. Daley’s bigger message was that the greatest near-term opportunity for iPS cell research is their ability to help us repurpose existing drugs to treat diseases.

G.L.

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