PRF-funded study Identifies Rapamycin as Possible Treatment for Progeria

Researchers at the National Institutes of Health and Massachusetts General Hospital in Boston, MA published a new study today in Science, Translational Medicine that may lead to a new drug treatment for children with Progeria.*
Rapamycin is an FDA approved drug that has previously been shown to extend the lives of non-progeria mouse models. This new study demonstrates that rapamycin decreases the amount of the disease-causing protein progerin by 50%, improves the abnormal nuclear shape, and extends the lifespan of progeria cells. This study provides the first evidence that rapamycin may be able to decrease progerin's damaging effects in children with progeria.
There is tremendous media coverage on this! Click below for links to media stories:
Wall Street Journal Health Blog
US News and World Report
Science Magazine
Boston Globe
The Progeria Research Foundation was delighted to provide cells for this project from the PRF Cell & Tissue Bank, and help fund the research through our grants program.
This exciting new study demonstrates the remarkable pace of progeria research, while providing further insight into the aging process that affects us all.
*"Rapamycin Reverses Cellular Phenotypes and Enhances Mutant Protein Clearance in Hutchinson-Gilford Progeria Cells"
Kan Cao, John J. Graziotto, Cecilia D. Blair, Joseph R. Mazzulli, Michael R. Erdos, Dimitri Krainc, Francis S. Collins
29 June 2011 Vol 3 Issue 89
Epub ahead of print


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