Exceptional response to the ATR inhibitor, camonsertib, in a patient with ALT+ metastatic melanoma

Abstract A key hallmark of cancer tumorigenesis is the maintenance of telomere length, which occurs canonically through the reactivation of telomerase. Alternative lengthening of telomeres (ALT) is an atypical, non-canonical telomere maintenance mechanism that uses homologous recombination (HR) to m...

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Main Authors: Natalie Y. L. Ngoi, Ian M. Silverman, Adrienne Johnson, Chenfeng Meng, Joseph D. Schonhoft, Michal Zimmermann, Danielle Ulanet, Hyeyeon Kim, Carlos Torrado, Carolina Salguero, Christian Valladolid Brown, Jordi Rodon, Victoria Rimkunas, Maria Koehler, Timothy A. Yap
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:npj Precision Oncology
Online Access:https://doi.org/10.1038/s41698-025-01025-1
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Summary:Abstract A key hallmark of cancer tumorigenesis is the maintenance of telomere length, which occurs canonically through the reactivation of telomerase. Alternative lengthening of telomeres (ALT) is an atypical, non-canonical telomere maintenance mechanism that uses homologous recombination (HR) to maintain telomere length and is associated with replication stress and defects in genome maintenance. In preclinical models, ALT positivity (ALT+) sensitizes tumor cells to ataxia telangiectasia and Rad3-related (ATR) inhibitors. Camonsertib is a novel potent, and highly selective ATR inhibitor that is synthetic lethal with genomic alterations affecting HR and DNA damage response (DDR). Here we describe a case of confirmed clinical and molecular response to pharmacological ATR inhibition through camonsertib, in a patient with ALT+ metastatic melanoma. To our knowledge, this is the first clinical report of synthetic lethal targeting of a confirmed ALT+ tumor with an ATR inhibitor.
ISSN:2397-768X