Pyruvate Kinase M2 Activators for the Treatment of Cancer

NIH investigators have discovered a series of small compounds with the potential to treat a variety of cancers as well as hemolytic anemia. Contrary to most cancer medications, these molecules can be non-toxic to normal cells because they target a protein specific to the metabolic pathways in tumors, thus representing a significant clinical advantage over less-specific chemotherapeutics.

Methods and Devices for Transcatheter Cerclage Annuloplasty

The invention relates to techniques and devices for cardiovascular valve repair, particularly annuloplasty techniques and devices in which tensioning elements are positioned to treat regurgitation of the mitral valve or tricuspid valve. More specifically, the technology pertains to a new device for myocardial septal traversal ("cerclage reentry") that also serves to capture (ensnare) and externalize the traversing guidewire.

Salen-Manganese Compounds for Therapy of Viral Infections

Salen-manganese compounds are synthetic, stable, low toxicity, low cost agents that may provide protection from immune reaction-related oxidative cell damage associated with many illnesses. In particular, oxidative cell damage has been associated with many viral infections including influenza. This invention demonstrates that treating mice with salen-manganese compounds, after lethal pandemic influenza virus infection, significantly enhances survival. Salen-manganese treatment also reduces lung pathology and also improved cellular recovery and repair.

Novel Tocopherol and Tocopheryl Quinone Derivatives as Therapeutics for Lysosomal Storage Disorders

Novel tocopherol derivatives and tocopheryl quinone derivatives useful in the decrease of lysosomal substrate accumulation, the restoration of normal lysosomal size, and the treatment of lysosomal storage disorders (LSDs) are provided. The inventors have discovered that tocopherol and tocopheryl quinone derivatives with side chain modifications (such as terminal tri-halogenated methyl groups) exhibit improved pharmacokinetics, modulation of mitochondrial potential and restoration of some LSDs phenotypes.

Device for Non-Surgical Tricuspid Valve Annuloplasty

This is a non-surgical tricuspid annuloplasty to treat functional tricuspid valve regurgitation, meaning regurgitation with intact valve leaflets. The device is delivered using novel catheter techniques into the pericardial space and positioned along the atrioventricular groove. A compression member is positioned along the tricuspid annular free wall and tension applied through a variably-applied tension element. In the best embodiment, the compression member has an M shaped portion with at least two inflection points between the segments of difference curvatures.

Monoclonal Antibodies That Recognize the Human Type I Interferon Receptor and Block Interferon Signaling

Type I interferons play a critical role in both innate and adaptive immunity through the stimulation of the IFNAR1 which initiates interferon signaling in response to viral and bacterial infections. However, abnormal interferon signaling is associated with human diseases, such as lupus. The present invention discloses six hybridomas that produce mouse monoclonal antibodies specific for the extracellular domain of human IFNAR1. Two of the monoclonal antibodies are able to bind IFNAR1 and reduce interferon signaling.

Human iPSC-Derived Mesodermal Precursor Cells and Differentiated Cells

Cells, cell culture methods, and cell culture media compositions useful for producing and maintaining iPSC-derived cell lines that are of higher purity and maintain cell type integrity better than current iPSC-derived cell lines are disclosed. Human induced pluripotent stem cells (hiPSCs) can be generated by reprogramming somatic cells by the expression of four transcription factors. The hiPSCs exhibit similar properties to human embryonic stem cells, including the ability to self-renew and differentiate into all three embryonic germ layers: ectoderm, endoderm, or mesoderm.