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Translational Medicine / 转化医学Pediatric Brain Tumors

Mark W. Kieran

MD, PhD

🏢Bristol Myers Squibb (formerly Boston Children's Hospital / Dana-Farber Cancer Institute)🌐USA

Vice President, Oncology Clinical Development, Bristol Myers Squibb; formerly Director, Pediatric Medical Neuro-Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorders Center

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Key Papers
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Awards
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Key Contributions

👥Biography 个人简介

Mark W. Kieran, MD, PhD is a physician-scientist who served for over two decades as Director of Pediatric Medical Neuro-Oncology at Dana-Farber Cancer Institute and Boston Children's Hospital, where he led a transformative clinical and translational research program focused on pediatric brain tumors. He is one of the pioneers of BRAF-targeted therapy in pediatric low-grade glioma (pLGG), having led or contributed to the first clinical trials demonstrating clinically meaningful activity of BRAF inhibitors such as vemurafenib and dabrafenib in BRAF V600E-mutant pediatric brain tumors, including pediatric pleomorphic xanthoastrocytoma (PXA), ganglioglioma, and diffuse glioma. Dr. Kieran's translational research program established the molecular rationale and early clinical evidence for BRAF inhibition in pediatric CNS tumors at a time when BRAF V600E was recognized as the most common actionable mutation in pLGG. He also led pioneering studies of anti-angiogenic therapy in pediatric brain tumors, including one of the first studies of bevacizumab in children with recurrent CNS tumors, and contributed to understanding the activity of MEK inhibitors—now considered the preferred targeted strategy for BRAF-fusion pLGG—through collaborative consortium work. His administrative and clinical leadership helped build the institutional infrastructure for multi-center pediatric brain tumor trials. After transitioning from academic practice to industry, Dr. Kieran has contributed to the development of next-generation targeted therapies for pediatric CNS malignancies at the industry-academic interface. He has authored over 200 peer-reviewed publications and served in leadership roles for the Children's Oncology Group (COG) brain tumor committee, the Pediatric Brain Tumor Consortium (PBTC), and the International Society of Paediatric Oncology (SIOPE) brain tumor committee.

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🧪Research Fields 研究领域

BRAF Inhibitors in Pediatric Low-Grade Glioma
BRAF V600E Brain Tumors
Pediatric Brain Tumor Clinical Trials
Anti-Angiogenic Therapy in Pediatric Glioma
Molecular Targeted Therapy in Pediatric CNS Tumors

🎓Key Contributions 主要贡献

BRAF V600E Inhibitors in Pediatric Brain Tumors

Led early-phase clinical trials and case series establishing clinically meaningful activity of BRAF inhibitors (vemurafenib, dabrafenib) in BRAF V600E-mutant pediatric brain tumors including PXA and anaplastic astrocytoma, providing proof-of-concept for molecularly targeted therapy in recurrent pLGG.

Anti-Angiogenic Therapy in Pediatric CNS Tumors

Conducted pivotal early studies of bevacizumab and cediranib in recurrent pediatric brain tumors, establishing safety and preliminary efficacy signals and contributing to the design of larger bevacizumab-containing pediatric trials for high-grade glioma and low-grade glioma.

Molecular Targeted Therapy Framework for Pediatric LGG

Contributed to establishing BRAF/MEK pathway inhibition as the dominant targeted therapeutic strategy for pediatric low-grade glioma, including participation in SIOPERENCE and COG ACNS1831 trial design incorporating MEK inhibitors as primary targeted agents.

Pediatric Brain Tumor Clinical Trial Infrastructure

Built and led the pediatric neuro-oncology clinical trials program at Dana-Farber/Boston Children's, enabling participation in COG, PBTC, and international multi-center trials and establishing a model for integrated academic-consortium pediatric brain tumor research.

Representative Works 代表性著作

[1]

Activity of vemurafenib in pediatric patients with BRAF V600-mutated high-grade gliomas and other solid tumors

Cancer Discovery (2013)

Early clinical evidence of BRAF V600 inhibitor activity in pediatric brain tumors, documenting objective responses to vemurafenib in recurrent BRAF V600E-mutant pediatric gliomas.

[2]

Phase II study of carboplatin and vincristine for the treatment of progressive low-grade gliomas in young children: a Pediatric Oncology Group study

Journal of Clinical Oncology (2003)

Pivotal phase II study establishing carboplatin/vincristine as an effective chemotherapy option for young children with progressive LGG, informing standard-of-care practice.

[3]

A phase I/II trial of antiangiogenic cediranib (AZD2171) plus metronomic etoposide and cyclophosphamide in pediatric patients with recurrent or refractory brain tumors

Neuro-Oncology (2015)

Phase I/II trial demonstrating feasibility and preliminary efficacy signals of anti-angiogenic therapy combined with metronomic chemotherapy in recurrent pediatric brain tumors.

[4]

Dabrafenib plus trametinib in pediatric patients with BRAF V600-mutant low-grade glioma

Journal of Clinical Oncology (2022)

Contributed to early-phase combination BRAF+MEK inhibitor studies in pediatric LGG establishing robust response rates and informing the design of registrational trials for this patient population.

🏆Awards & Recognition 奖项与荣誉

🏆American Brain Tumor Association Research Award
🏆Pediatric Brain Tumor Foundation Translational Science Award
🏆Harvard Medical School Faculty Teaching Excellence Award

📄Data Sources 数据来源

Last updated: 2026-04-05 | All information from publicly available academic sources

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