NEJM Highlights March 2015: Progress against Crohn’s, PCSK9 inhibitors coming through, comparative effectiveness for diabetic macular edema, Eric Lander encourages the FDA on genomic testing regulation
Marc Herant
April 1, 2015
A promising agent for Crohn’s Disease, a miserable illness Crohn’s is an inflammatory bowel disease that is notoriously unpredictable; flares can affect any part of the digestive tract and lead to grave complications. In this double-blind phase 2 study, patients were dosed with mongersen (licensed by Celgene) an anti-sense oligonucleotide that down-regulates the expression of a protein implicated in the inflammatory cascade. In general these classes of medications have to be given parenterally but in this case the target is the gut so it can be taken orally. At two weeks