sirolimus

Overview

Macrolide mTORC1 inhibitor (rapamycin).

Evidence in the corpus

• Referenced among matched targeted therapy options in the Make-an-IMPACT rare-cancer program where matched therapy produced clinical benefit in 17/18 histiocytosis patients (mean duration 21.7 months) PMID:36862133.
• In the UCLA PDTO sarcoma screen, PDTOs from patients with progressive disease at follow-up were more resistant to sirolimus/rapamycin (p=0.014) compared to non-progressors. This inverse correlation — reduced mTOR sensitivity in the most aggressive sarcomas — contrasts with the sorafenib/ruxolitinib pattern seen in the same progressive subset. PMID:39305899

Resistance mechanisms

• Not reported in corpus.

Cancer types (linked)

• LCH, ECD

Sources

• PMID:36862133
• PMID:39305899 — Duminuco et al. 2024, UCLA PDTO sarcoma functional screen; sirolimus resistance in progressive-disease sarcomas.

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