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Childhood Brain Tumors: A Review of Strategies to Translate CNS Drug Delivery to Clinical Trials

Rahman, Ruman; Janowski, Miroslaw; Killick-Cole, Clare L.; Singleton, William G. B.; Campbell, Emma; Walczak, Piotr; Khatua, Soumen; Faltings, Lukas; Symons, Marc; Schneider, Julia R.; Kwan, Kevin; Boockvar, John A.; Gill, Steven S.; Miguel Oliveira, J.; Beccaria, Kevin; Carpentier, Alexandre; Canney, Michael; Pearl, Monica; Veal, Gareth J.; Meijer, Lisethe; Walker, David A.

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Miroslaw Janowski

Clare L. Killick-Cole

William G. B. Singleton

Emma Campbell

Piotr Walczak

Soumen Khatua

Lukas Faltings

Marc Symons

Julia R. Schneider

Kevin Kwan

John A. Boockvar

Steven S. Gill

J. Miguel Oliveira

Kevin Beccaria

Alexandre Carpentier

Michael Canney

Monica Pearl

Gareth J. Veal

Lisethe Meijer

David A. Walker


Brain and spinal tumors affect 1 in 1000 people by 25 years of age, and have diverse histological, biological, anatomical and dissemination characteristics. A mortality of 30–40% means the majority are cured, although two-thirds have life-long disability, linked to accumulated brain injury that is acquired prior to diagnosis, and after surgery or chemo-radiotherapy. Only four drugs have been licensed globally for brain tumors in 40 years and only one for children. Most new cancer drugs in clinical trials do not cross the blood–brain barrier (BBB). Techniques to enhance brain tumor drug delivery are explored in this review, and cover those that augment penetration of the BBB, and those that bypass the BBB. Developing appropriate delivery techniques could improve patient outcomes by ensuring efficacious drug exposure to tumors (including those that are drug-resistant), reducing systemic toxicities and targeting leptomeningeal metastases. Together, this drug delivery strategy seeks to enhance the efficacy of new drugs and enable re-evaluation of existing drugs that might have previously failed because of inadequate delivery. A literature review of repurposed drugs is reported, and a range of preclinical brain tumor models available for translational development are explored.


Rahman, R., Janowski, M., Killick-Cole, C. L., Singleton, W. G. B., Campbell, E., Walczak, P., …Walker, D. A. (2023). Childhood Brain Tumors: A Review of Strategies to Translate CNS Drug Delivery to Clinical Trials. Cancers, 15(3), Article 857.

Journal Article Type Article
Acceptance Date Jan 26, 2023
Online Publication Date Jan 30, 2023
Publication Date Jan 30, 2023
Deposit Date Feb 3, 2023
Publicly Available Date Feb 14, 2023
Journal Cancers
Electronic ISSN 2072-6694
Publisher MDPI AG
Peer Reviewed Peer Reviewed
Volume 15
Issue 3
Article Number 857
Keywords Cancer Research; Oncology
Public URL
Publisher URL


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