In an early-phase clinical trial of a new oral drug, selumetinib, children with the common genetic disorder neurofibromatosis type 1 (NF1) and plexiform neurofibromas, tumors of the peripheral nerves, tolerated selumetinib and, in most cases, responded to it with tumor shrinkage. NF1 affects 1 in 3,000 people. The study results appeared Dec. 29, 2016, in the New England Journal of Medicine [1].
Nationwide Study Reports Shifts in Americans’ Use of Natural Products
A nationally representative survey shows that natural product use in the United States has shifted since 2007, with some products becoming more popular and some falling out of favor. Overall, natural products (dietary supplements other than vitamins and minerals) remain the most common complementary health approach.
HHS, NIH Take Steps to Enhance Transparency, Public Access to Clinical Trial Results
Last week, the U.S. Department of Health and Human Services (HHS) issued a Notice of Proposed Rulemaking (NPRM), which proposes regulations to implement reporting requirements for clinical trials that are subject to Title VIII (Clinical Trial Databases) of the Food and Drug Administration Amendments Act of 2007 (FDAAA). The proposed rule clarifies requirements to clinical researchers for registering clinical trials and submitting summary trial results information to ClinicalTrials.gov, a publicly accessible database operated by the National Library of Medicine, part of the National Institutes of Health. The NIH also announced a proposal to apply the proposed requirements to all NIH-funded clinical trials, whether subject to FDAAA or not. The proposed policy would require that all NIH-funded clinical trials be registered in ClinicalTrials.gov and that summary results be posted to the database in a timely matter. Both documents are open for a 90-day public comment period, and comments will be taken into consideration before final regulations and a final NIH Policy are issued.
Most Risk for Autism Comes from Common Genetic Variation, Not Rare Genes
Most of the genetic risk for autism comes from versions of genes that are common in the population rather than from rare variants or spontaneous glitches, researchers funded by the National Institutes of Health have found. Heritability also outweighed other risk factors in this largest study of its kind to date.
Protein Overload May Kill Brain Cells as Parkinson’s Progresses
Scientists may have discovered how the most common genetic cause of Parkinson’s disease destroys brain cells and devastates many patients worldwide.