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Future Oncol. 2018 Sep;14(21):2115-2129. doi: 10.2217/fon-2018-0121. Epub 2018 Mar 29.

Accelerating drug development in pediatric cancer: a novel Phase I study design of venetoclax in relapsed/refractory malignancies.

Author information

1
Dana-Farber/Boston Children's Cancer & Blood Disorders Center, Boston, MA, USA.
2
School of Medicine, Emory University, Atlanta, GA, USA.
3
Cancer Research Center, University Children's Hospital & Children's Research Center, Zurich, Switzerland.
4
Department of Pediatrics, Benioff Children's Hospital & the Helen Diller Comprehensive Cancer Center, University of California, San Francisco, CA, USA.
5
School of Medicine & Children's Hospital Colorado, University of Colorado, Aurora, CO, USA.
6
Department of Paediatrics, Hospital for Sick Children & University of Toronto, Toronto, ON, Canada.
7
AbbVie Inc., North Chicago, IL, USA.
8
Genentech Inc., South San Francisco, CA, USA.
9
Department of Pediatric Oncology/Hematology, Erasmus MC-Sophia Children's Hospital, Rotterdam, & Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
10
Wolfson Childhood Cancer Research Centre, Northern Institute for Cancer Research, Newcastle University & Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Abstract

Venetoclax is a highly selective, potent BCL-2 inhibitor that is approved for some patients previously treated for chronic lymphocytic leukemia, and has shown promising activity in adult studies across several hematologic malignancies. Preclinical studies have demonstrated venetoclax activity in pediatric patient-derived xenograft models and cell lines; however, clinical studies in pediatric patients have yet to be conducted. The prognosis is poor for children with most relapsed/refractory malignancies, and limited treatment options result in unmet clinical need. Herein, we describe the rationale and design of the first study of venetoclax in pediatric patients with relapsed/refractory malignancies: a Phase I trial investigating the safety and pharmacokinetics of venetoclax monotherapy followed by the addition of chemotherapy (Trial registration: EudraCT 2017-000439-14; NCT03236857).

KEYWORDS:

ALL; AML; BCL-2; NHL; neuroblastoma; pediatric cancer; relapsed/refractory; venetoclax

PMID:
29595064
DOI:
10.2217/fon-2018-0121
[Indexed for MEDLINE]
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