RO4929097 is a gamma-secretase inhibitor that has been primarily investigated for its potential in cancer therapy. It works by blocking the Notch signaling pathway, which plays a critical role in cell differentiation, proliferation, and survival—processes often dysregulated in tumors. This compound has shown activity in preclinical models of various cancers, including breast, brain, and hematological malignancies such as T-cell acute lymphoblastic leukemia.

Its application focuses on disrupting tumor growth and enhancing the effectiveness of other anticancer agents, especially in cancers dependent on Notch pathway activation. RO4929097 has been evaluated in clinical trials to assess its safety and efficacy, either as a monotherapy or in combination with chemotherapy or targeted drugs. Despite promising mechanisms, challenges such as limited bioavailability and variable patient response have affected its development progress. Nonetheless, it remains a tool for studying Notch pathway inhibition and its therapeutic implications in oncology.