Blood brain barrier-adapted precision medicine therapy for pediatric brain tumors

Publication date: Available online 10 August 2017
Source:Translational Research
Author(s): Bernard L. Marini, Lydia L. Benitez, Andrew H. Zureick, Ralph Salloum, Angela C. Gauthier, Julia Brown, Yi-Mi Wu, Dan R. Robinson, Chandan Kumar, Robert Lonigro, Pankaj Vats, Xuhong Cao, Katayoon Kasaian, Bailey Anderson, Brendan Mullan, Benjamin Chandler, Joseph R. Linzey, Sandra I. Camelo-Piragua, Sriram Venneti, Paul E. Mc Keever, Kathryn A. McFadden, Andrew P. Lieberman, Noah Brown, Lina Shao, Marcia A.S. Leonard, Larry Junck, Erin McKean, Cormac O. Maher, Hugh J.L. Garton, Karin M. Muraszko, Shawn Hervey-Jumper, Jean M. Mulcahy-Levy, Adam Green, Lindsey M. Hoffman, Katie Dorris, Nicholas A. Vitanza, Joanne Wang, Jonathan Schwartz, Rishi Lulla, Natasha Pillay Smiley, Miriam Bornhorst, Daphne A. Haas-Kogan, Patricia L. Robertson, Arul M. Chinnaiyan, Rajen Mody, Carl Koschmann
Targeted chemotherapeutics provide a promising new treatment option in neuro-oncology. The ability of these compounds to penetrate the blood brain barrier is crucial for their successful incorporation into patient care. "CNS Targeted Agent Prediction" (CNS-TAP) is a multi-institutional and multi-disciplinary translational program established at the University of Michigan for evaluating the CNS activity of targeted therapies in neuro-oncology. In this report, we present the methodology of CNS-TAP in a series of pediatric and adolescent patients with high-risk brain tumors, for which molecular profiling (academic and commercial) was sought and targeted agents were incorporated. Four of five of the patients had potential clinical benefit (partial response or stable disease greater than 6 months on therapy). We further describe the specific drug properties of each agent chosen and discuss characteristics relevant in their evaluation for therapeutic suitability. Finally, we summarize both tumor and drug characteristics that impact the ability to successfully incorporate targeted therapies into CNS malignancy management.



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