Fibroblast Cell Lines (with L444P/RecNci1 Genotype) for the Screening of Small Molecules for Gaucher Disease Treatment

This technology includes two human fibroblast cell lines to be used to study the defects in GBA1 gene and protein and to screen small molecules for involvement in Gaucher disease. Glucocerebrosidase (GBA1 or GCase or beta-glucosidase) is a lysosomal enzyme, responsible for breakdown of a fatty material called glucocerebroside (or glucosyl ceramide). Deficiency or malfunction of GBA1 leads to the accumulation of insoluble glucocerebrosides in tissues, which is a major symptom of Gaucher disease.

Monoclonal Antibodies for the Recognition of Oncogene Fusions and Alveolar Rhabdomyosarcoma (ARMS) Diagnosis

This technology includes monoclonal antibody (mAb) that binds to the junction region of the PAX3-FOXO1 and PAX7-FOXO1 fusion protein for the diagnosis of Alveolar Rhabdomyosarcoma (ARMS). Specifically, two monoclonal antibodies (PFM.1 and PFM.2) have been isolated that recognize the 92kDa bands found uniquely to the pediatric striated muscle tumors of the type Alveolar Rhabdomyosarcoma (ARMS) carrying the characteristic t(2;13)(q35;q14) or t(1;13)(p36;q14) chromosomal translocations.

MLL3 (KMT2C), MLL4, PA1, UTX And PTIP Antibodies for the Treatment of Development Diseases and Cancers

This technology includes polyclonal antibodies against MLL3 (KMT2C), MLL4, PA1, UTX And PTIP for the development of treatments for development diseases and cancer. Enhancers play a central role in cell-type-specific gene expression and are marked by H3K4me1/2. Active enhancers are further marked by H3K27ac. However, the methyltransferases responsible for H3K4me1/2 on enhancers remain elusive. Furthermore, how these enzymes function on enhancers to regulate cell-type-specific gene expression is unclear.

A Novel Carbohydrate Antibody to GalNac1-3Gal and Its Application for Cancer Diagnostic and Prognosis

Cervical cancer is one of the most common cancers among women worldwide. Currently, physical descriptors such as tumor size and depth are the primary factors used for deciding the course of treatment. Despite significant efforts to identify prognostic biochemical markers or therapeutic targets to improve diagnosis and treatment, none have achieved routine clinical use. An example of one previously identified biomarker is the Tn antigen, a carbohydrate moiety composed of a GalNAc residue linked to serine or threonine.

Cancer Vaccines against POTE for Treating Solid Tumors

POTE is a novel tumor antigen expressed in a variety of cancers including breast, prostate, colon, lung, ovary, and pancreas cancers.  POTE has limited expression in normal tissues and therefore a specific target for cancer treatments, including immunotherapy.  The researchers seek statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize immunogenic peptides. 

Natural product-based anti-cancer agents: aza-Englerin analogues

Chemotherapy resistance in a wide array of cancers is often associated with enhanced glucose uptake and dysregulation of the insulin signaling pathway.  Therapeutics capable of inhibiting insulin signaling would be valuable as a stand-alone treatment and for sensitizing resistant tumors to standard chemotherapy regiments.  Researchers at NCI’s Genitourinary Malignancies Branch have synthesized and developed a series of Englerin-A ana

T-Cell Immunotherapy that Targets Aggressive Epithelial Tumors

Metastatic cancers cause up to 90% of cancer deaths, yet few treatment options exist for patients with metastatic disease. Adoptive transfer of T cells that express tumor-reactive T-cell receptors (TCRs) has been shown to mediate regression of metastatic cancers in some patients. Unfortunately, identification of antigens expressed solely by cancer cells and not normal tissues has been a major challenge for the development of T-cell based immunotherapies. Thus, it is essential to find novel target antigens differentially expressed in cancer versus normal tissues.

Isotopes of Alpha Ketoglutarate and Related Compounds for Hyperpolarized MRI Imaging

This technology includes 1-13C-ketoglutarate which can be used for imaging the conversion to hydroxyglutarate (HG) or Gln in cancer cells with an IDH1 mutations by hyperpolarized MRI. The ability to detect the status of IDH1 mutations is clinically prognostic for multiple cancers. These exciting observations are limited by two factors, the major one being that the natural abundance of 13C at position C5 overlaps with 1-13C-2-hydroxyglutarate peak, which limits the sensitivity of analysis and prevents simultaneous observations of HG and Gln formation.

LZK and DLK Inhibitors to Target LZK and Suppress MYC Expression, Inhibit AKT Activation, and Promote Cancer Cell Death and Tumor Regression

This technology includes the use of LZK and DLK inhibitors to be used for the treatment of head and neck squamous cell carcinoma (HNSCC) or lung squamous cell carcinoma (LSCC). Specifically, we demonstrate that inhibitors that can be repurposed to target LZK suppresses LZK kinase-dependent stabilization of MYC and activation of the PI3K/AKT pathway. In vivo preclinical cell line xenograft mouse model demonstrates that targeting LZK will suppress tumor growth. We also demonstrate that several additional compounds potently inhibit LZK and could serve as new therapeutic modalities.

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