ridaforolimus

Ridaforolimus, also known as deforolimus, is a macrolide cancer drug that acts as an inhibitor of the mechanistic target of rapamycin (mTOR) pathway. It is a rapalog, an analog of rapamycin, and functions by forming a complex with the intracellular protein FKBP12 to selectively inhibit mTOR complex 1 (mTORC1). Inhibition of mTORC1 reduces phosphorylation of downstream targets such as S6 kinase and 4E-BP1, leading to decreased protein synthesis, cell-cycle arrest in G1, and reduced tumor cell growth.

Ridaforolimus has been studied in a range of cancers, including soft tissue sarcomas, neuroendocrine tumors, breast cancer, and renal cell carcinoma, across phase II and phase III clinical trials. It has been evaluated in various formulations, including oral dosing regimens and intravenous administration, frequently in combination with other anticancer agents. However, ridaforolimus has not been approved by the United States Food and Drug Administration for any indication, and its development has shown variable results across trials.

Common adverse effects observed with ridaforolimus and other mTOR inhibitors include mucositis or stomatitis, rash, fatigue, hyperglycemia, hyperlipidemia, anemia, thrombocytopenia, and edema. There is also an increased risk of infections, and non-infectious pneumonitis has been reported with mTOR inhibitors. As with other agents in this class, safety monitoring and management of metabolic and inflammatory side effects are important considerations in research and clinical use.

Ridaforolimus is part of the broader class of mTOR inhibitors that target the PI3K-AKT-mTOR signaling axis to hinder tumor growth.