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

作者：Marini Bernard L; Benitez Lydia L; Zureick Andrew H; Salloum Ralph; Gauthier Angela C; Brown Julia; Wu Yi Mi; Robinson Dan R; Kumar Chandan; Lonigro Robert; Vats Pankaj; Cao Xuhong; Kasaian Katayoon; Anderson Bailey; Mullan Brendan; Chandler Benjamin; Linzey Joseph R; Camelo Piragua Sandra I; Venneti Sriram; McKeever Paul E; McFadden Kathryn A; Lieberman Andrew P; Brown Noah; Shao Lina; Leonard Marcia A S; Junck Larry; McKean Erin; Maher Cormac O

来源：Translational Research, 2017, 188: 27-39.

DOI：10.1016/j.trsl.2017.08.001

Targeted chemotherapeutics provide a promising new treatment option in neurooncology. 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 multidisciplinary translational program established at the University of Michigan for evaluating the central nervous system (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.

• 出版日期2017-10