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@ARTICLE{Jennemann:296197,
author = {R. Jennemann$^*$ and M. Volz$^*$ and R. C. Frias-Soler$^*$
and A. Schulze$^*$ and K. Richter$^*$ and S. Kaden$^*$ and
R. Sandhoff$^*$},
title = {{G}lucosylceramide {S}ynthase {I}nhibition in {C}ombination
with {A}ripiprazole {S}ensitizes {H}epatocellular {C}ancer
{C}ells to {S}orafenib and {D}oxorubicin.},
journal = {International journal of molecular sciences},
volume = {26},
number = {1},
issn = {1422-0067},
address = {Basel},
publisher = {Molecular Diversity Preservation International},
reportid = {DKFZ-2025-00110},
pages = {304},
year = {2025},
note = {DKFZ-ZMBH Alliance / #EA:A411#LA:A411#},
abstract = {Hepatocellular carcinoma (HCC) is one of the leading causes
of cancer deaths due to its late diagnosis and restricted
therapeutic options. Therefore, the search for appropriate
alternatives to commonly applied therapies remains an area
of high clinical need. Here we investigated the therapeutic
potential of the glucosylceramide synthase (GCS) inhibitor
Genz-123346 and the cationic amphiphilic drug aripiprazole
on the inhibition of Huh7 and Hepa 1-6 hepatocellular cancer
cell and tumor microsphere growth. Single and combinatorial
treatments with both drugs at 5 µM concentration led to
efficient cell cycle arrest, reduced expression of cyclins A
and E, increased lipid storage in lysosomal compartments,
accompanied by increased uptake of lysotracker, and elevated
expression of the autophagy marker Lc3 II. Both drugs
affected mitochondrial function, indicated by altered
mitotracker uptake and impaired mitochondrial respiration.
Aripiprazole in monotherapy, or even more pronounced in
combination with Genz, also potentiated the effect of the
cytostatic drugs sorafenib and doxorubicin on tumor cell-
and tumor spheroid-growth inhibition. Targeting GCS with
Genz with the parallel application of cationic amphiphilic
drugs such as aripiprazole in combination with cytostatic
drugs may thus represent a potent therapeutic approach in
the treatment of HCC and potentially other cancer types.},
keywords = {Humans / Carcinoma, Hepatocellular: drug therapy /
Carcinoma, Hepatocellular: pathology / Carcinoma,
Hepatocellular: metabolism / Sorafenib: pharmacology / Liver
Neoplasms: drug therapy / Liver Neoplasms: pathology / Liver
Neoplasms: metabolism / Glucosyltransferases: antagonists
$\&$ inhibitors / Glucosyltransferases: metabolism /
Doxorubicin: pharmacology / Aripiprazole: pharmacology /
Cell Line, Tumor / Drug Synergism / Cell Proliferation: drug
effects / Mitochondria: drug effects / Mitochondria:
metabolism / Antineoplastic Agents: pharmacology / Genz
(Other) / anti-psychotic drugs (Other) / aripiprazole
(Other) / cationic amphiphilic drugs (CAD) (Other) /
doxorubicin (Other) / glucosylceramide synthase (Other) /
hepatocellular tumor spheroids (Other) / lysosomal function
(Other) / mitochondrial function (Other) / sorafenib (Other)
/ Sorafenib (NLM Chemicals) / Glucosyltransferases (NLM
Chemicals) / Doxorubicin (NLM Chemicals) / Aripiprazole (NLM
Chemicals) / ceramide glucosyltransferase (NLM Chemicals) /
Antineoplastic Agents (NLM Chemicals)},
cin = {A411 / A410 / W230},
ddc = {540},
cid = {I:(DE-He78)A411-20160331 / I:(DE-He78)A410-20160331 /
I:(DE-He78)W230-20160331},
pnm = {311 - Zellbiologie und Tumorbiologie (POF4-311)},
pid = {G:(DE-HGF)POF4-311},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39796160},
pmc = {pmc:PMC11720485},
doi = {10.3390/ijms26010304},
url = {https://inrepo02.dkfz.de/record/296197},
}