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Transl Res. 2017 Oct;188:27.e1-27.e14. doi: 10.1016/j.trsl.2017.08.001. Epub 2017 Aug 10.

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

Author information

1
Michigan Medicine, Department of Pharmacy Services, Ann Arbor, Mich.
2
Michigan Medicine, Department of Pharmacy Services, Ann Arbor, Mich; University of Kentucky Healthcare, Department of Pharmacy, Lexington, Ky.
3
University of Michigan Medical School, Ann Arbor, Mich.
4
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
5
Yale School of Medicine, New Haven, Conn.
6
University of Colorado Denver School of Medicine, Denver, Colo.
7
Seattle Children's Hospital, Seattle Wash.
8
Children's Hospital of Michigan, Detroit, Mich.
9
Anne and Robert H. Lurie Children's Hospital of Chicago, Chicago Ill.
10
Children's National Health System, Washington, DC.
11
Harvard Medical School, Boston, Mass.
12
University of Michigan Medical School, Ann Arbor, Mich. Electronic address: ckoschma@med.umich.edu.

Abstract

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 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.

PMID:
28860053
PMCID:
PMC5584679
DOI:
10.1016/j.trsl.2017.08.001
[Indexed for MEDLINE]
Free PMC Article

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