Project Title: Optimizing SN38 nanoparticles to treat children with high-risk tumors Investigator:  Garrett Brodeur, MD Institution: Children’s Hospital of Philadelphia Study Type: Preclinical   Nanotechnology is a rapidly emerging field with the potential to revolutionize drug delivery, and it is expected to affect every major arena of drug development in the coming years. Advances in this area…

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This international collaboration of researchers and charities will rapidly introduce a new investigational drug to children and potentially result in significant clinical responses. Project Title: A Phase I/II study of PF-06463922, a next-generation ALK inhibitor, in children with relapsed / refractory ALK-driven neuroblastoma Investigators: Araz Marachelian, MD; Yael Mosse, MD; Lou Chesler, MD Institutions: NANT…

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A promising way to further improve outcomes in this disease appears to be the development of adjuvant therapeutic strategies. This trial will treat children with high-risk neuroblastoma by combining anti-GD2 with mIBG therapy and a new antibody (Nivolumab). Project Title: A Phase 1 Study of 131-1 mIBG, Nivolumab and ch14.18/CHO in relapsed / refractory neuroblastoma.…

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This immunotherapy treatment uses cytotoxic T lymphocytes (CTLs) for the first-time for children with neuroblastoma. CTLs are the killer T-cells of the immune system whose role is to seek out and destroy abnormal cells. Project Title: Phase I Clinical Trial of Tumor Associated Antigen-Specific CTLs for Neuroblastoma Researcher: Chris DeRenzo, MD Institution: Baylor College of…

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This trial studies the side effects and best dose of activated T cells armed with GD2 bispecific antibody and how well they work in treating patients with neurblastoma, osteosarcoma, and other GD2+ solid tumors. Project Title: Activated T-Cells Armed With GD2 Bispecific Antibody in Children and Young Adults With Neuroblastoma and Osteosarcomas Researcher: Maxim Yankelevich,…

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This clinical trial uses a new technology for childhood cancer called “High Intensity Focused Ultrasound guided by Magnetic Resonance.” This clinical trial will be the first time this noninvasive new technology will be tried in children with abdominal tumors that cannot be surgically removed. Project Title: Pilot Study of High Intensity Focused Ultrasound (HIFU) for…

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By understanding the genetic features of a tumor, researchers are able to use specific drugs that interfere with certain cell processes that cause unrestricted growth. This is the first precision medicine trial for children that will robustly analyze the genomics and epigenomics of cancer and use combinations of investigational drugs to target specific mutations and…

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In the lab, an antibody targeting ALK expression kills neuroblastoma cells. Researchers believe that an anti-ALK antibody that targets the surface antigen will alert the immune system to attack the cancer, reducing the risk of a future recurrence. This research is to develop, test, and produce clinical grade anti-ALK antibody. Project Title: Preclinical Development of…

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