Can Small Molecule Agents Make a Big Difference?

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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Can Targeted Radiotherapy and a Combination of Antibodies Pack More Punch in Boosting the Immune System to Destroy Cancer Cells?

Can Targeted Radiotherapy and a Combination of Antibodies Pack More Punch in Boosting the Immune System to Destroy Cancer Cells? Project Title: A Phase I Study of 131-1 mIBG, Nivolumab, and Dinutuximab-beta in Relapsed / Refractory Neuroblastoma (MiNivAn) Researcher: Juliet Gray, MD Institution: University Hospital Southampton, University of Wisconsin-Madison, Greifswald, Germany Study Type: Phase I clinical…

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Can the Polio Virus Stop Deadly Brain Tumors?

The virus is delivered via a catheter directly into the tumor by a method known as convection-enhanced delivery.  This clinical project fills a significant unmet need for children with high-risk brain tumors. PVSRIPO for Recurrent Malignant Glioma in Children Project Title: Phase I study of an oncolytic polio/rhinovirus recombinant (PVS-RIPO) against recurrent pediatric high-grade glioma…

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Are Five Targets Better Than One? Using CTLs To Seek and Destroy

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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Can T-Cell Immunotherapy Work in Osteosarcoma and Neuroblastoma?

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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Can Nivolumab Treat Childhood Brain Tumors?

Recent results from clinical trials in adults with various tumor types  have shown exciting results. Nivolumab has not yet been studied in any children with brain tumors. Project Title: Anti-PD1 Nivolumab in Children With Brain Tumors Researcher: Ira Dunkel, MD Institution: Memorial Sloan-Kettering Cancer Center; Johns Hopkins University Hospital Study Type: Phase II Clinical Trial…

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Can High Frequency Ultrasound Be Used for Bulky Tumors?

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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Can Genetic Mutations Be Specifically Targeted for Neuroblastoma?

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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