NCGC staff collaborate with more than 200 investigators at NIH and in the academic, biopharmaceutical and nonprofit sectors to generate probes for studying a diverse cross-section of human biology, focusing specifically on new targets and untreatable diseases. Read the latest news about these collaborations below.
NCATS scientists and their colleagues have demonstrated how an investigational drug works against a rare, fatal genetic disease, Niemann-Pick type C1 (NPC1). They found that a closely related compound will activate an enzyme, AMPK, triggering a cellular “housekeeping” system that helps reduce elevated cholesterol and other accumulated fats that in the brains and livers of NPC1 patients, which are hallmarks associated with severe neurological problems.
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.
International scientific teams find potential approach against parasites. Led by NCATS and University of Tokyo scientists, the teams sorted through more than 1 trillion small protein fragments to uncover two that could shut down the enzyme. The finding, reported April 3, 2017, in Nature Communications, could set the stage for the potential development of new types of antimicrobial drugs.
NIH scientists and grantees have discovered that ketamine lifts depression via a byproduct of its metabolism in mouse study.
NCATS researchers and collaborators from the NIH’s National Institute of Allergy and Infectious Diseases, Georgetown University, and the University of California, San Francisco, have released a large dataset of potential drug combinations for malaria. Using NCATS’ state-of-the-art high-throughput combination drug-screening platform, the Center’s researchers tested 13,910 combinations of known and newly identified antimalarial drugs in three malaria parasite lines.
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.