Targeting Notch as a Therapeutic Approach for Human Malignancies
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Updated: Mar 14, 2026
Integration of Bioinformatics Approaches and Experimental Validations to Understand the Role of Notch Signaling in Ovarian Cancer
Published on: January 12, 2020
Natalia Platonova1, Elena Lesma2,3, Andrea Basile2,3
1Department of Oncology and Hemato-Oncology, Universita degli Studi di Milano,via Festa del Perdono 7, I-20122, Milano,Italy.
Background:
Notch is a multifaceted protein that plays a fundamental role in fetal development and tissue homeostasis by directing many cellular functions, including cell growth and differentiation, cell fate determination and regulation of stem cells maintenance. The Notch family consists of four receptors (Notch 1-4) and five ligands (Jagged1-2 and Delta-like 1-3-4) widely expressed in human tissues. Given the crucial contribution of Notch signaling in many physiological processes, it is not surprising that a variety of human malignancies is characterized by a dysregulation of one or more components of this pathway.
Methods:
In this review, we are going to provide a broad overview on the role of Notch pathway in solid and hematological malignancies and a survey on possible Notch-directed therapeutic strategies.
Results:
We present the most recent findings indicating that Notch signaling dysregulation in human cancers may be due to genetic and epigenetic alterations or to the interactions with other oncogenic pathways. Furthermore, Notch activity may have an oncogenic or a tumor suppressor effect. Finally, we describe the latest preclinical and clinical studies concerning the different pharmacological approaches targeting Notch.
Conclusion:
The provided evidence confirms the importance of Notch pathway in human malignancies indicating that a strong rationale exists for the development of a Notch-tailored therapy.
Insights
The Notch pathway is crucial for development and homeostasis, but its dysregulation contributes to various cancers. Targeting Notch signaling offers a promising therapeutic strategy for malignancies.
Area of Science:
- Molecular Biology
- Cell Biology
- Oncology
Background:
- The Notch signaling pathway is essential for fetal development and tissue homeostasis.
- It regulates critical cellular functions like growth, differentiation, and stem cell maintenance.
- Dysregulation of Notch components is implicated in numerous human malignancies.
Purpose of the Study:
- To provide a comprehensive review of the Notch pathway's role in solid and hematological cancers.
- To survey potential therapeutic strategies targeting Notch signaling in malignancies.
Main Methods:
- Literature review of recent findings on Notch signaling in cancer.
- Analysis of genetic and epigenetic alterations affecting Notch pathway components.
- Survey of preclinical and clinical studies on Notch-targeted therapies.
Main Results:
- Notch signaling dysregulation in cancer can arise from genetic/epigenetic changes or interactions with other oncogenic pathways.
- Notch pathway activity can exert either oncogenic or tumor-suppressive effects depending on the context.
- Various pharmacological approaches targeting Notch are under investigation.
Conclusions:
- The Notch pathway plays a significant role in human malignancies.
- Evidence supports the development of Notch-tailored therapies for cancer treatment.
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