Research And Grants

Developing Novel Combination Therapies for Diffuse Intrinsic Pontine Glioma

This project will develop novel combination therapies for the most aggressive childhood cancer, diffuse intrinsic pontine glioma (DIPG). Our team performed a comprehensive high-throughput drug screen, testing 3,570 drugs for their ability to prevent DIPG cell growth and found ACT001 to be one of the most effective drugs tested. ACT001 is known to have both antioxidant and anti-inflammatory properties and readily crosses the blood brain barrier. We have initiated a world first Phase 1 pediatric clinical trial of ACT001 for children with relapsed/refractory solid or CNS tumors, including patients with DIPG/DMG. Currently, fifteen patients, seven of which are DIPG/DMG patients, are enrolled and no dose-limiting toxicities have been observed. Excitingly, clinical activity has been demonstrated in three DIPG/DMG patients including improvement in the appearance of the tumor on MRI imaging, as well as improvement in patient symptoms. Using our preclinical models, we now seek to determine precisely how ACT001 works in DIPG and then use this information to evaluate potential ACT001 combination therapies, to enhance the effectiveness of ACT001. As such, this project will provide valuable information for developing new combination treatments that may lead to ACT001 as the first active treatment for children suffering from DIPG.