Researchers at NCATS and the University of Nevada, Reno School of Medicine have demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy (DMD). The candidate drug, SU9516, represents a different kind of approach for treating DMD, a degenerative muscle disease that usually begins in childhood and has no known cure. It is caused by a faulty gene that leads to progressive muscle weakness, with death often occurring around age 25.
Ketamine Lifts Depression via a Byproduct of its Metabolism
NIH-funded team finds rapid-acting, non-addicting agent in mouse study. “Working in collaboration with NIH and academic researchers, NCATS chemists played a critical role in isolating the specific metabolite of ketamine responsible for fighting depression,” said Christopher P. Austin, M.D., NCATS director. “Overall, our collective efforts exemplify how a collaborative, team science approach can help advance the translational process in ways that help get more treatments to more patients more quickly.”
News Brief: Data Released on Drug Combinations to Treat Malaria
NCATS researchers tested and released data on thousands of combinations of known and newly identified drugs to potentially treat malaria.
NCATS-FDA team receives support from the HHS Innovation Ventures Fund — an HHS IDEA Lab program — to enhance the Web-based Collaborative Use Repurposing Engine (CURE). The highly competitive awards provide growth-stage funding and 15 months of mentoring as well as tools to help grow and sustain CURE, which supports crowdsourcing information on treating tropical diseases.
NCGC experts and University of Chicago researchers work together to create, adapt and validate an innovative 3-D ovarian cancer cell model used to identify small molecules that can prevent cancer cells from spreading to new sites in the body. The new model includes connective tissue cells and the extracellular matrix (a collection of supportive molecules outside cells), creating a more lifelike simulation of the human body environment.
A team of researchers from NCGC and the National Human Genome Research Institute make a major advance in the understanding and treatment of Gaucher disease, creating a new model that not only helps identify treatments for patients with this rare disorder, but also is useful in studying other diseases.
Scientists in NCGC work closely with academic, nonprofit and biotechnology researchers to pursue collaborative probe development projects, including the discovery of three novel small molecule chemical probes with the potential to treat diabetes, stroke and thrombosis.
A team that includes nine NCGC researchers identifies compounds that delay tumor formation in mice. The compounds target a specific form of pyruvate kinase, called PKM2, which governs how cancer cells use glucose.
Scientists from NCGC and the University of Wisconsin–Madison help identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease, a rare neurological disorder.
NCATS and Eli Lilly and Company jointly release an Assay Guidance Manual designed to provide step-by-step guidance through the complex process of turning a basic research finding into an assay (test) that will start the process of discovering pharmacological tools and drugs.