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Recent Studies on Ponatinib in Cancers Other Than Chronic Myeloid Leukemia
Francesca Musumeci1, Chiara Greco2, Giancarlo Grossi3
1Department of Pharmacy, University of Genova, Viale Benedetto XV 3, 16132 Genova, Italy. francesca.musumeci@unige.it.
Abstract:
Ponatinib is a third line drug for the treatment of chronic myeloid leukemia patients, especially those that develop the gatekeeper mutation T315I, which is resistant to the first and the second line drugs imatinib, nilotinib, dasatinib and bosutinib. The compound was first identified as a pan Bcr-Abl and Src kinase inhibitor. Further studies have indicated that it is a multitargeted inhibitor that is active on FGFRs, RET, AKT, ERK1/2, KIT, MEKK2 and other kinases. For this reason, the compound has been evaluated on several cancers in which these kinases play important roles, including thyroid, breast, ovary and lung cancer, neuroblastoma, rhabdoid tumours and in myeloproliferative disorders. Ponatinib is also being tested in clinical trials to evaluate its activity in FLT3-ITD acute myelogenous leukemia, head and neck cancers, certain type of lung cancer, gastrointestinal stromal tumours and other malignancies. In this review we report the most recent preclinical and clinical studies on ponatinib in cancers other than CML, with the aim of giving a complete overview of this interesting compound.
Insights
Ponatinib effectively targets various kinases beyond Bcr-Abl, showing promise in treating cancers like leukemia, thyroid, and lung cancer. This review highlights its recent preclinical and clinical applications in non-chronic myeloid leukemia malignancies.
Area of Science:
- Oncology
- Pharmacology
- Molecular Biology
Background:
- Ponatinib is a third-line treatment for chronic myeloid leukemia (CML), particularly effective against the T315I mutation resistant to other tyrosine kinase inhibitors.
- Initially identified as a pan Bcr-Abl and Src kinase inhibitor, ponatinib exhibits multitargeted activity against various kinases including FGFRs, RET, AKT, and ERK1/2.
- These targeted kinases are implicated in the development and progression of several other cancers.
Purpose of the Study:
- To review recent preclinical and clinical studies of ponatinib in cancers beyond CML.
- To provide a comprehensive overview of ponatinib's efficacy and potential in diverse oncological indications.
- To explore the therapeutic potential of ponatinib in various malignancies driven by specific kinase mutations.
Main Methods:
- Literature review of preclinical studies investigating ponatinib's efficacy in different cancer models.
- Analysis of ongoing and completed clinical trials evaluating ponatinib's safety and effectiveness in various cancers.
- Summarization of ponatinib's kinase inhibition profile and its relevance to non-CML cancers.
Main Results:
- Ponatinib has demonstrated activity in preclinical models of thyroid, breast, ovarian, and lung cancers, as well as neuroblastoma, rhabdoid tumors, and myeloproliferative disorders.
- Clinical trials are evaluating ponatinib for FLT3-ITD positive acute myelogenous leukemia, head and neck cancers, specific lung cancers, and gastrointestinal stromal tumors.
- The multitargeted nature of ponatinib contributes to its broad spectrum of activity across different cancer types.
Conclusions:
- Ponatinib shows significant therapeutic potential in a range of cancers beyond CML due to its broad kinase inhibition profile.
- Further clinical investigation is warranted to establish ponatinib's role in treating various hematological and solid tumors.
- Ponatinib represents a promising targeted therapy option for malignancies driven by the kinases it inhibits.
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