$75,000* for Pharmacogenomics and Drug Screening Lead to a Novel Targeted Therapy with Potent and Specific Activity Against Mouse Models of MYCN-amplified Neuroblastoma
Anthony Faber, PhD - Virginia Commonwealth University
Using a combination of pharmacogenomics, drug screening and gene expression data mining, we have developed a novel targeted therapy combination that is specific for MYCN-amplified neuroblastoma. The goal of this grant is to further develop a combination targeted therapy to treat high-risk neuroblastoma.
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$75,000* for Preclinical Development of ATR Inhibitor VE-822, Delivered Systemically in Nanoparticles, for Medulloblastoma Therapy
Timothy Gershon, M.D./Ph.D. - University of North Carolina
This proposal will investigate the therapeutic potential of targeting the DNA damage response protein ATR for medulloblastoma therapy, using a small molecule inhibitor, VE- 822, in novel, nanoparticle formulation (pVE-822). Radiation and chemotherapy significantly extend survival for most patients with medulloblastoma, but produce significant long-term neuro-cognitive side effects.
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$75,000* for Small Molecule Inhibitors of ERG for Pediatric Leukemia and Sarcoma
John Bushweller, PhD - University of Virginia, School of Medicine
Current treatment for childhood AML, T-ALL, and Ewing’s sarcoma is limited in efficacy and has profound long- term side-effects due to the use of traditional cytotoxic agents rather than targeted drugs inhibiting specific drivers of the diseases. A targeted agent which inhibits ERG, clearly a driver of these diseases, has the potential to improve both survival and quality of life for children with AML, T-ALL, and Ewing’s sarcoma.
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