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Rhabdomyosarcoma (RMS) is the most common type of soft tissue sarcoma in children and makes up 3% of all childhood cancers. Aveloar Rhabdomyosarcoma is the most aggressive subtype and is primarily established through a chromosomal translocation resulting in the fusion protein PAX3-FOXO1. Despite aggressive therapy, the 5-year survival rate for patients with high risk or recurrent Fusion Positive RMS (FP-RMS) is low (~30% and ~17%, respectively). Therefore, new therapies targeting the PAX3-FOXO1 oncogenic driver are urgently needed.  

Angiogenesis is the formation of new blood vessels from pre-existing blood vessels. Angiogenesis occurs during normal growth and development (physiological angiogenesis) and during the growth of solid tumors (pathological angiogenesis). CD276, also known as B7-H3, is a cell surface tumor endothelial marker that is highly expressed in the tumor vessels of human lung, breast, colon, endometrial, renal, and ovarian cancer, but not in the angiogenic vessels of normal, healthy tissue.

Cancer diagnosis depends on the assessment of patient biopsies to determine tumor type, grading, and stage of malignancy. Pathologists visually review specimens and count mitotic figures (MF) in a variety of cancer types to help gauge aggressiveness, guide treatment, and inform patient prognosis. Current technology for recording MF counts in surgical pathology is lacking in objectivity, and enumeration of MF by microscopy can be error prone. In particular, a lack of systematic means for recording contributes to recognized variability.

Gene therapy research has yielded FDA-approved treatments for an array of diseases. However, challenges facing nucleic-acid based therapeutics include non-specific delivery and degradation of the nanoparticles. NCI investigators have developed a solution to address these challenges in their novel nucleic-based therapy based on the conditional activation strategy. 

Hydrogels represent an attractive controlled drug-delivery system that have been used in various clinical applications, such as: tissue engineering for wound healing, surgical procedures, pain management, cardiology, and oncology. High-water content of hydrogels confers tissue-like physical properties and the crosslinked fibrillar network enables encapsulation of labile small molecule drugs, peptides, proteins, nucleic acids, proteins, nanoparticles, or cells.

Epidermal growth factor receptor variant III (EGFRvIII) is a variant of EGFR that is an excellent target for immunotherapy because of its expression in cancer cells and not in normal cells. 

The Hippo signaling pathway regulates a multitude of biological processes including cell proliferation, apoptosis, differentiation, tissue homeostasis, and stem cell functions. This axis has been recently listed as one of the top 10 signaling pathways altered in human cancer. Its role in modulating cell growth and proliferation is mediated by the activation of Yes-associated protein 1 (YAP1) and transcriptional co-activator with PDZ-binding domain (TAZ).

Human cancers contain genetic mutations that are unique to each patient. Some of the mutated peptides are immunogenic, can be recognized by T cells, and therefore, may serve as therapeutic targets.

Previously described epidermal growth factor receptor- (EGFR) driven tumor mouse models develop diffuse tumors, which are dissimilar to human lung tumor morphology and difficult to measure by CT and MRI scans. Scientists at the National Cancer Institute (NCI) have developed and characterized a genetically engineered mouse (GEM) model of human EGFR-driven tumor model (hEGFR-TL) that recapitulates the discrete lung tumor nodules similar to those found in human lung tumor morphology.

Primary mediastinal B-cell lymphoma (PMBCL) is an aggressive type of non-Hodgkin lymphoma that mostly occurs in people between the ages of 30-40. It accounts for 5-7% of all aggressive lymphomas. The diagnosis of PMBCL is challenging as the histological features of PMBCL overlap with diffuse large B-cell lymphoma (DLBCL), another most common type of non-Hodgkin lymphoma. Available evidence suggests that PMBCL responds much more favorably to the DA-EPOCH-R chemotherapy regimen than to the standard R-CHOP regimen used to treat DLBCL.