Does Size Matter? Can a Nanoparticle Deliver Something Really Big?

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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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? 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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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 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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Will an Antibody Recognizing a New Target in Neuroblastoma Kill Tumor Cells?

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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