This $503,194 Project Grant award from the National Cancer Institute's Cancer Treatment Research program (CFDA 93.395) supports research at Seattle Children's Hospital aimed at developing an innovative multispecific T cell engager (MTE) therapy to treat neuroblastoma and other pediatric solid tumors. The key products and services being delivered under this 2-year award include: 1) Determining the efficacy of the R2P3 MTE, which binds to the ROR1 and PD-L1 tumor targets as well as CD3 on T cells,...
This $4,653,104 Cooperative Agreement award from the National Cancer Institute (CFDA 93.395 - Cancer Treatment Research) supports research to discover and develop small molecule agents that can modulate the PAX3-FOXO1 fusion oncoprotein driving the highly fatal fusion-positive alveolar rhabdomyosarcoma (FP-RMS) cancer. The project involves a multi-institutional team with expertise in high-throughput screening, chemoproteomics, molecular biology, medicinal chemistry, and pharmacology. Key goals...
This federal Project Grant award from the National Cancer Institute (CFDA 93.395 Cancer Treatment Research) provides $792,367 to Nanopharmaceuticals LLC to develop a novel dual-targeting ligand, NP855, for the treatment of high-risk neuroblastoma. The key products and services to be delivered under this grant include: Scaled-up manufacturing and analytical evaluation of the NP855 drug substance to enable formulation of the drug product. In vitro and in vivo studies to confirm the efficacy of the...
The National Cancer Institute (NCI) awarded a $177,000 Project Grant under the Cancer Biology Research program (CFDA 93.396) to The Children's Hospital Corporation, doing business as Boston Children's Hospital. The funding supports research to investigate the role of the NLRC4 inflammasome and caspase-8 activation in the pathogenesis of fibrolamellar carcinoma (FLC), a rare and aggressive form of liver cancer. The researchers will use mouse liver organoids engineered with the DNAJB1-PRKACA...
This Project Grant award from the National Cancer Institute (CFDA 93.395 - Cancer Treatment Research) provides $1,237,513 to The Washington University to develop a novel combination molecular radiotherapy approach for treating relapsed and refractory neuroblastoma, the most common extracranial solid cancer in children. The research aims to evaluate the efficacy and biological effects of two high linear energy transfer radiopharmaceuticals, 77Br-MBBG targeting the norepinephrine transporter and...
This Project Grant award, valued at $1,723,610.00 and awarded by the U.S. Food and Drug Administration (FDA) under the FDA Research program (CFDA 93.103), will support the development of novel gene therapy treatments and outcome measures for Leber Congenital Amaurosis (LCA) in pediatric patients. The award has two key objectives: 1) Conduct a Phase I/II clinical trial of a gene augmentation therapy for LCA5, a severe form of LCA, in adolescent patients aged 13-18 years, and 2) Develop new...
This $156,136 Project Grant award from the National Center for Advancing Translational Sciences (NCATS), CFDA 93.350, aims to develop biochemical reagents and assays to analyze the structure and function of the PAX3-FOXO1 fusion protein that drives the rare disease alveolar rhabdomyosarcoma. Specifically, the project will generate recombinant PAX3-FOXO1 constructs and conduct preliminary structural screening using innovative nuclear magnetic resonance techniques. It will also establish in...
The National Cancer Institute (NCI), through the Cancer Treatment Research Federal Grant Program (CFDA 93.395), awarded a $219,351 Project Grant (R21CA296696) to The Institute for Cancer Research (ICR) to assess the efficacy and toxicity of T3 (the active form of thyroid hormone) for the treatment of medulloblastoma, the most common malignant brain tumor in children. The research aims to evaluate the toxicity, pharmacokinetics, and pharmacodynamics of T3 in tumor-bearing mice, as well as examine...
This Project Grant award from the National Cancer Institute (CFDA 93.398 - Cancer Research Manpower) provides $143,788 to the Broad Institute, Inc. to investigate genetic dependencies in KIAA1549-BRAF rearranged pediatric low-grade gliomas. The key objectives are to: Test the hypothesis that protein O-mannosyltransferase (POMT) enzymatic activity is necessary for the survival of KIAA1549:BRAF rearranged pediatric low-grade glioma cells. Evaluate whether O-mannosylation of the KIAA1549:BRAF...
This Project Grant award from the National Cancer Institute (CFDA 93.395 - Cancer Treatment Research) provides $194,013 to Sanford Research, a non-profit biomedical research organization, to develop the first genetically engineered mouse models that accurately replicate key features of human high-risk rhabdomyosarcoma. The goal is to use these advanced preclinical models to explore the role of the NOTCH1 signaling pathway as both a driver and potential therapeutic target for this aggressive form...