The U.S. Food and Drug Administration took a significant step forward in driving the efficient development of novel diagnostic technologies that scan a person’s DNA to diagnose genetic diseases and guide medical treatments. For the first time, the agency has formally recognized a public database that contains information about genes, genetic variants and their relationship to disease.
Meeting the goals of the Paris Agreement could save about a million lives a year worldwide by 2050 through reductions in air pollution alone. The latest estimates from leading experts also indicate that the value of health gains from climate action would be approximately double the cost of mitigation policies at global level, and the benefit-to-cost ratio is even higher in countries such as China and India.
An interim analysis of a large phase 3 clinical trial found that the combination of ibrutinib plus rituximab was superior to standard treatment for patients age 70 and younger with previously untreated chronic lymphocytic leukemia (CLL). The trial met its primary endpoint of an improvement in progression-free survival (the length of time patients live before their disease worsens). The combination also improved overall survival, the trial’s secondary endpoint. In general, patients in the ibrutinib–rituximab arm were less likely to experience serious side effects than those in the standard treatment arm. Until now, the standard treatment for previously untreated CLL has been a six-month course of FCR, which combines the chemotherapy drugs fludarabine and cyclophosphamide with rituximab.
A scientist from the National Institutes of Health will present promising, early results from a human clinical trial testing a novel gene replacement therapy in people with severe sickle cell disease. Preliminary findings suggest that the approach has an acceptable level of safety and might help patients consistently produce normal red blood cells instead of the sickle-shaped ones that mark this painful, life-threatening disease.
The National Institutes of Health announced that enrollment for the Adolescent Brain Cognitive Development (ABCD) Study is now complete and, in early 2019, scientists will have access to baseline data from all ABCD Study participants.
The U.S. Food and Drug Administration today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS.
The U.S. Food and Drug Administration approved Truxima (rituximab-abbs) as the first biosimilar to Rituxan (rituximab) for the treatment of adult patients with CD20-positive, B-cell non-Hodgkin’s lymphoma (NHL) to be used as a single agent or in combination with chemotherapy. Truxima is the first biosimiliar to be approved in the U.S. for the treatment of non-Hodgkin’s lymphoma.
The U.S. Food and Drug Administration approved Xospata (gilteritinib) tablets for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test. The FDA also approved an expanded indication for a companion diagnostic, to include use with Xospata. The LeukoStrat CDx FLT3 Mutation Assay, developed by Invivoscribe Technologies, Inc., is used to detect the FLT3 mutation in patients with AML.
An international research team has begun patient enrollment in a clinical trial testing multiple investigational Ebola therapies in the Democratic Republic of the Congo (DRC). The randomized, controlled trial is enrolling patients of any age with confirmed Ebola virus disease (EVD) at a treatment unit in the city of Beni operated by ALIMA(link is external) (The Alliance for International Medical Action), a medical humanitarian organization.
Scientists at the National Eye Institute (NEI) have found that neurons in the superior colliculus, an ancient midbrain structure found in all vertebrates, are key players in allowing us to detect visual objects and events. This structure doesn’t help us recognize what the specific object or event is; instead, it’s the part of the brain that decides something is there at all. By comparing brain activity recorded from the right and left superior colliculi at the same time, the researchers were able to predict whether an animal was seeing an event. The findings were published in the journal Nature Neuroscience. NEI is part of the National Institutes of Health.