Results Published in Annals of Neurology Entitled, "Sustained Improvement of Spinal Muscular Atrophy Mice Treated with Trichostatin A Plus Nutrition"

The paper, published by the research group of Dr. Charlotte Sumner at Johns Hopkins University and partially funded by Families of SMA, shows for the first time sustained survival in SMA mice after using a specific drug regimen.

This regimen entailed early treatment with the histone deacetylase inhibitor trichostatin A (TSA), starting on day 2 post birth and nutritional support including infant formula by mouth and subcutaneous fluids, starting on day 8 post birth.  Average survival time was extended by 170%, while in experiments using just TSA treatment alone survival was extended by 40% and by 19% when TSA alone began later on day 5 post birth.  Nutritional support alone did not extend survival times. 

TSA is not suitable for human use, but other potent second generation HDAC inhibitors are currently being explored as possible treatments for SMA.

Click here to see the journal site.