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@ARTICLE{Schmitz:302004,
author = {S. B. M. Schmitz and J. Gülden and M. Niederreiter and C.
Eichner and J. Werner$^*$ and B. Mayer$^*$},
title = {{S}trong {H}sp90α/β {P}rotein {E}xpression in {A}dvanced
{P}rimary {CRC} {I}ndicates {S}hort {S}urvival and
{P}redicts {R}esponse to the {H}sp90α/β-{S}pecific
{I}nhibitor {P}imitespib.},
journal = {Cells},
volume = {14},
number = {11},
issn = {2073-4409},
address = {Basel},
publisher = {MDPI},
reportid = {DKFZ-2025-01203},
pages = {836},
year = {2025},
abstract = {The prognosis of advanced (UICC IIb-IV) primary colorectal
cancer (pCRC) remains poor. More effective targeted
therapies are needed. Heat shock protein 90 alpha/beta
(Hsp90α/β) expression was immunohistologically quantified
in 89 pCRCs and multivariately correlated with survival.
Pimitespib (Pim, TAS-116), a Hsp90α/β-specific inhibitor,
was tested in pCRC cell lines and patient-derived cancer
spheroids (PDCS) and referenced to the pan-Hsp90 inhibitor
ganetespib (Gan, STA-9090) and standard-of-care therapies. A
total of $26.97\%$ pCRCs showed strong tumoral Hsp90α/β
expression (Hsp90α/β > $40\%),$ which correlated with
reduced PFS (HR: 3.785, $95\%CI:$ 1.578-9.078, p = 0.003)
and OS (HR: 3.502, $95\%CI:$ 1.292-9.494, p = 0.014).
Co-expression of Hsp90α/β > $40\%$ with its clients
BRAF-V600E and Her2/neu aggravated the prognosis (BRAF-V600E
mutated: PFS, p = 0.002; OS, p = 0.012; Her2/neu score3:
PFS, p = 0.029). The prognostic cut-off Hsp90α/β > $40\%$
was also a predictor for response to Pim-based therapy. Pim
efficacy was increased in combination with 5-FU, 5-FU +
oxaliplatin, and 5-FU + irinotecan (all p < 0.001). Pim
induced sensitization to all chemotherapies in HT-29 (p <
0.001), Caco-2 (p < 0.01), and HCT116 (p < 0.05) cells. Pim
combined with encorafenib in HT-29 and with trastuzumab in
Caco-2 cells was most effective in dual-target inhibition
approaches (HT-29: p < 0.005; Caco-2: p < 0.05). The
anti-cancer effect and chemosensitization of Pim-based
therapy were prospectively confirmed in PDCS directly
generated from Hsp90α/β > $40\%$ pCRCs. Protein profiling
combined with functional drug testing stratifies Hsp90α/β
> $40\%$ pCRC patients diagnosed with UICC IIb-IV for
effective Pim-based therapy.},
keywords = {Humans / HSP90 Heat-Shock Proteins: metabolism / HSP90
Heat-Shock Proteins: antagonists $\&$ inhibitors /
Colorectal Neoplasms: drug therapy / Colorectal Neoplasms:
metabolism / Colorectal Neoplasms: pathology / Female / Male
/ Aged / Middle Aged / Cell Line, Tumor / Prognosis /
Triazoles: pharmacology / Triazoles: therapeutic use /
Spheroids, Cellular: drug effects / Spheroids, Cellular:
metabolism / CRC (Other) / Hsp90α/β (Other) / TAS-116
(Other) / chemosensitization (Other) / ganetespib (Other) /
patient stratification (Other) / patient-derived cancer
spheroid model (Other) / personalized therapy (Other) /
pimitespib (Other) / prognosis (Other) / HSP90 Heat-Shock
Proteins (NLM Chemicals) / STA 9090 (NLM Chemicals) /
Triazoles (NLM Chemicals) / HSP90AB1 protein, human (NLM
Chemicals)},
cin = {MU01},
ddc = {570},
cid = {I:(DE-He78)MU01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40498011},
pmc = {pmc:PMC12154481},
doi = {10.3390/cells14110836},
url = {https://inrepo02.dkfz.de/record/302004},
}