Drug intelligence / Profile preview

ML-SI1

Development stage
Preclinical
Lead developer
University of Michigan
Modality
Small Molecules
01

Overview

ML-SI1 is a potent and selective small molecule inhibitor of the Mucolipin-1 (TRPML1) channel, a lysosomal cation channel encoded by the *MCOLN1* gene. TRPML1 is a member of the transient receptor potential (TRP) superfamily and is primarily localized to the membranes of late endosomes and lysosomes, where it regulates ion homeostasis and vesicular trafficking. ML-SI1 is widely utilized as a pharmacological tool to investigate the physiological roles of TRPML1. In the context of oncology, research has demonstrated that ML-SI1 can attenuate the proliferation and invasion of p53-deficient bladder cancer cells. Its mechanism involves inducing G0/G1 cell cycle arrest and suppressing the production of pro-tumorigenic cytokines such as IL-6 and TNF-alpha, particularly in cells harboring p53 mutations or oncogenic HRAS activation.

Other names
TRPML1 inhibitorTRPML-1 inhibitorTRPML 1 inhibitorMucolipin Synthetic Inhibitor 1
02

Targets

MCOLN1 (Transient receptor potential cation channel mucolipin subfamily member 1)TRPML2 (Transient receptor potential cation channel mucolipin subfamily member 2)TRPML3 (Mucolipin-3)

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