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Investigational drugs can save or extend lives, and seriously ill patients not able to take part in clinical trials should have access to such drugs whenever possible. In a climate of increased public pressure for this access—often termed compassionate use—five states in the US have passed so-called 'right to try' legislation. These laws are ill advised, as they are not likely to substantially increase access and have the potential to compromise the clinical trial system.
This year's most notable research included studies that opened new avenues for regenerative medicine, paved the way to editing out vulnerability to disease and unraveled the genetic complexities underlying diseases such as leukemia and schizophrenia. Here are some of the papers that captured our attention and moved their fields forward in 2014.
Understanding the regenerative capacity of the adult mammalian heart and the cell types involved is essential for developing therapies for cardiac repair.
The authors report a mechanistic basis for intestinal polyp formation in patients with hereditary mixed polyposis syndrome that involves the aberrant epithelial expression of morphogens and leads to the formation of ectopic intestinal crypts by progenitor cells outside the stem cell niche, a mechanism that seems to also be involved in human ectopic serrated polyps.
The authors delineate a new pathway that regulates apoptosis, involving the known proapoptotic kinase PKCD, as well as the ubiquitin ligase Fbox protein FBXO25 and the mitochondrial antiapoptotic factor HAX-1, and report its alteration during lymphomagenesis.
Aftab Ansari and his colleagues show that antibody-mediated masking of α4β7 integrin impedes intravaginal transmission of simian immunodeficiency virus in macaques.
Hashizume et al. report a new therapeutic strategy for treating pediatric gliomas with mutations in the H3F3A gene by inhibiting histone demethylation.