Projects

First-in-Class Immunotherapy for Relapsed and Refractory Pediatric Brain Tumors

Project Title:  Phase 1 study of APX-005M CD40 agonist antibody in children with CNS tumor Researcher: Ira Dunkel, MD Institutions: Pediatric Brain Tumor Consortium Study Type: Phase I   This phase I clinical trial is a collaboration to evaluate dosage and side effects of APX005M, an investigational immune activating compound that targets CD40, in pediatric patients…

Read More

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…

Read More

Can The Measles Virus Help Topple Deadly Brain Tumors?

Project Title: Ph I Measles OV MV-NIS for recurrent Medullo/ATRT Investigators:  Sabine Mueller, MD, PhD, MAS Institutions:  UCSF Helen Diller Cancer Center, PNOC Consortium Study Type: Two arm Phase I clinical trial Status:  Recruiting   For children with recurrent medulloblastoma or AT/RT, outcomes remain poor. Preclinical studies have shown that treatment with modified, attenuated measles virus (MV-NIS) leads…

Read More

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…

Read More

Will a Three-Pronged Approach be the Better Approach?

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.…

Read More

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…

Read More

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…

Read More

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,…

Read More

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 1 Clinical Trial…

Read More

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…

Read More