Targeted Therapies for High-Risk Pediatric Brain Tumor Subtypes

Home » Research » Acceleration Initiative » Active Grants » Targeted Therapies for High-Risk Pediatric Brain Tumor Subtypes

CureSearch Acceleration Initiative Award, 2016-2018

Dr. Richard Gilbertson

Richard Gilbertson, MD, PhD

University of Cambridge
Research Focus: High-risk pediatric brain tumor subtypes: Medulloblastoma, Ependymoma and Choroid Plexus Carcinoma

Project Overview:

Brain cancers are the most common solid tumors in children, accounting for 20 percent of all childhood cancers. Many of these cancers are high-risk, meaning that they are prone to relapse and have a low 5-year survival rate. Each type of brain tumor also includes subtypes with radically different origins, biology and prognoses. Current treatments – a combination of surgery, radiation and conventional chemotherapy, are not effective at curing these cancers.

Dr. Gilbertson, one of the world’s leading experts in pediatric brain tumors, received a CureSearch Acceleration Initiative Award to discover and develop new therapies for three high-risk pediatric brain cancers: medulloblastoma, ependymoma and choroid plexus carcinoma.

Project Update:

Dr. Gilbertson’s team tested new and existing drugs for these specific diseases and identified several promising therapies to guide immediate clinical testing. Results of this project contributed to the launch of a phase 1 clinical trial testing combination therapies for malignant brain tumors with Dr. Gilbertson’s research directly impacting patients with recurrent, refractory medulloblastoma. The trial, sponsored by St. Jude Children’s Research Hospital, opened March 5, 2019.

In addition to the progress that Dr. Gilbertson has made towards promoting clinical trials, he has performed a high-throughput drug screen, assessing over 1.2 million compounds for effectiveness against high-risk brain cancers and has developed 15 novel animal models using tissue from pediatric brain cancer patients.

Notably, thanks in part to the strength of the work supported by CureSearch, Dr. Gilbertson’s institution was designated as the first Cancer Research UK Children’s Brain Tumour Centre of Excellence.

Hear Dr. Gilbertson speak about the project after receiving his CureSearch grant:

Pin It on Pinterest

Scroll to Top