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LCL-385
- Development stage
- Preclinical
- Lead developer
- Icahn School of Medicine
- Modality
- Small Molecules
- Administration
- Intraperitoneal
01
Overview
LCL-385 is a small molecule inhibitor of acid ceramidase (ASAH1), an enzyme responsible for the hydrolysis of ceramide into sphingosine and fatty acids. By inhibiting this enzyme, LCL-385 disrupts the sphingolipid rheostat, leading to an accumulation of pro-apoptotic ceramide and a decrease in pro-survival sphingosine-1-phosphate (S1P). This mechanism effectively lowers the apoptotic threshold of malignant cells, making them more susceptible to programmed cell death. Research, particularly in models of acute myeloid leukemia (AML), has shown that LCL-385 can work synergistically with standard induction chemotherapies such as daunorubicin and cytarabine to inhibit cell proliferation and enhance therapeutic efficacy.
02
Targets
ASAH1 (Acid ceramidase)SMPD1 (Acid sphingomyelinase)
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