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N-acylsphingosine amidohydrolase 1 (acid ceramidase, ASAH1) (ASAH1)
- Target
- ASAH1
- Molecular classification
- Enzyme, Lipid hydrolase, Lysosomal hydrolase
01
Overview
Acid ceramidase (ASAH1) is a lysosomal lipid hydrolase responsible for cleaving ceramide into sphingosine and free fatty acids at acidic pH. This catalytic activity is central to sphingolipid metabolism, with downstream effects on bioactive lipids (sphingosine, sphingosine-1-phosphate) critical for regulating cellular proliferation, apoptosis, and differentiation. Mutations in ASAH1 cause Farber disease and spinal muscular atrophy with progressive myoclonic epilepsy, while aberrant expression or activity has been linked to cancer progression and treatment resistance. As such, ASAH1 is considered a potential therapeutic target for cancer, rare genetic, and neurodegenerative diseases.
- Other names
- Acid ceramidaseAcid ceramidase subunit alphaAcid ceramidase subunit betaASAH1ASAHACDaseACPHP32PHPSMAPMEN-acylsphingosine amidohydrolase (acid ceramidase) 1FLJ21558acylsphingosine deacylaseglycosylceramide deacylaseN-acylethanolamine hydrolase ASAH1putative 32 kDa heart proteinASAH1_HUMANFLJ22079
02
Mechanism of action
Inhibition of acid ceramidase leads to ceramide accumulation and induces cell death in cancer cells (pro-apoptotic effect through sphingolipid metabolism perturbation)
03
Biological functions
Sphingolipid metabolism (hydrolysis of ceramide to sphingosine and fatty acids)Regulation of cell proliferationInduction and regulation of apoptosisCell differentiation (notably in epidermal keratinocytes)Potential regulation of steroidogenesis via nuclear receptor interaction
04
Disease associations
Lysosomal storage disorder (Farber lipogranulomatosis / Farber disease)Neurodegenerative disease (Spinal muscular atrophy with progressive myoclonic epilepsy)Cancer (glioblastoma, breast cancer, colorectal cancer, other human cancers via overexpression)
05
Safety considerations
Systemic inhibition may disrupt normal lipid metabolism, with risk for adverse cellular effects, as ceramide metabolism is fundamental to cell signaling, immune response, and neuronal survival. Genetic deficiency leads to severe disorders, cautioning therapeutic modulation
06
Interacting drugs
Carmofur (an approved colorectal cancer drug, proposed repurposing for glioblastoma)
07
Biomarkers
Elevated ASAH1 expression can serve as a biomarker for cancer progression, radioresistance in glioblastoma, and may assist in diagnosis or prognosis in lysosomal storage disorders
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