NIH R01 · 2025
Evaluating and Optimizing Novel nSMase2 Inhibitors in the Treatment of Cancers
PROJECT ABSTRACT / SUMMARY: Immune checkpoint inhibitors (ICIs) have revolutionized cancer treatment, but many patients fail to respond. Expanding the response to ICIs is a major goal in immuno-oncology. We and others recently discovered that blocking exosome biogenesis through the genetic depletion of nSMase2 can overcome resistance to ICIs in multiple mouse models leading to robust antitumor immune response and inhibition of tumor growth. Therefore, we hypothesize that small molecule inhibition of nSMase2 could be a novel therapeutic strategy to promote antitumor immunity. There are no clinically available nSMase2 inhibitors. Current inhibitors have low potency, unknown selectivity, and…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.