Nature Medicine is the premier journal for biomedical research. Respected internationally for the quality of its papers on areas ranging from infectious disease to cancer and neurodegeneration, Nature Medicine aims to bridge the gap between basic research and medical advances and is consistently ranked the number one journal by the Institute of Scientific Investigation in the Medicine, Research and Experimental category.
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.
At first glance, 2014 may seem like a year dominated by disease outbreaks, from polio's reemergence in parts of Asia to the Ebola epidemic that still continues to make headlines. But a closer look indicates 2014 was replete with regulatory rulings, big investments in genomic medicine and activism in the name of research funding.
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.
This year's newsworthy drugs made major strides against infectious diseases, cancer and more. Some others received attention for controversies they stemmed or stomped. Here is a look at the therapies that leapt forward, some that are in limbo, and others that fell by the wayside.
Diffuse intrinsic pontine glioma is a uniformly lethal malignant tumor of infancy with no effective therapies. A new study reveals that inhibition of JMJD3 has robust antitumor activity in diffuse intrinsic pontine glioma xenografts.
Increasing evidence points to a role for the immune system in the regulation of metabolism. Two new studies in mice indicate treatment with interleukin-22 restores mucosal immunity in diabetes and alleviates metabolic disease, resulting in improved glycemic control.
Frontotemporal dementia (FTD) is a neurodegenerative disease that causes social dysfunction and other symptoms. A new study suggests that social dysfunction in FTD is due to decreased microRNA-124 expression and resulting changes in glutamate receptor composition in the prefrontal cortex.
Vascular endothelial growth factor A (VEGF-A) is a potent proangiogenic cytokine elevated in patients with peripheral artery disease (PAD). A new study links impaired vascular regrowth in PAD to increased expression of an antiangiogenic splice variant of VEGF-A.
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.
Alternative splicing of the gene encoding VEGF-A under ischemic conditions generates an antiangiogenic isoform of the protein that impairs revascularization under conditions of metabolic dysfunction in mice, and this isoform is found at elevated levels in patients with peripheral artery disease.