patient-derived

TH287 is a selective MTH1 inhibitor(IC50 : 0.8 nM). It causees incorporation of oxidized dNTPs in cancer cells, leading to DNA damage, cytotoxicity and therapeutic responses in patient-derived mouse xenografts.

HR68, a derivative of the peroxisome proliferator-activated receptor (PPAR) agonist fenofibric acid, serves as an anticancer agent. It effectively reduces the viability of LN-229 glioblastoma cells, with an IC50 value of 1.17 µM. Notably, HR68 is capable of crossing the blood-brain barrier in temozolomide-resistant orthotopic patient-derived xenograft (PDX) mouse models of glioblastoma.

Selective peptide-based β-catenin Degrader. Comprises a β-catenin-targeted stapled peptide, xStAx, linked to a VHL-binding peptide. Reduces β-catenin levels in HEK293T cells and colorectal cancer cell lines. Selectively degrades β-catenin over other Wnt signaling pathway components. Inhibits tumor growth in APCmin/+ mice, with constitutively active Wnt signaling, and reduces survival of patient-derived colorectal cancer cell organoids.

CAY10722 is an inhibitor of sirtuin 3 (SIRT3), a class III HDAC (71% inhibition at 200 μM). SIRT3 is involved in modulating metabolic homeostasis as a NAD+-dependent protein deacetylase in the mitochondria. SIRT3 functions as either an oncogene or tumor suppressor, depending on cancer cell type. High SIRT3 expression in patient-derived esophageal cancer tissues is associated with shorter survival and, in mice, downregulation leads to a lower tumor load. In contrast, low SIRT3 expression in patient-derived breast cancer cells is correlated with shorter survival.

6-(1-Hydroxyethyl)-5,6-dihydrochelerythrine, an alkaloid, significantly alters the characteristics of early endosomes, mitochondria, and autophagosomes in Parkinson's Disease patient-derived olfactory cells [1].

W1131, a potent STAT3 inhibitor, effectively triggers ferroptosis and suppresses cancer progression across various models including gastric cancer cell subcutaneous xenograft, organoids, and patient-derived xenograft (PDX). Additionally, W1131 overcomes chemical resistance in cancer cells to 5-FU by regulating cell cycle, DNA damage response, and oxidative phosphorylation pathways, notably the IL6-JAK-STAT3 and ferroptosis pathways [1].

DS89002333 is a potent, orally active PRKACA inhibitor (IC50: 0.3 nM).DS89002333 has shown good antitumor effects in a patient-derived xenograft model of FL-HCC expressing a DNAJB1-PRKACA fusion gene.DS89002333 can be used to study fibrotic hepatocellular carcinoma ( FL-HCC).

KSL-128114, a peptide inhibitor of the membrane regulator syntenin (Ki = 300 nM), notably diminishes the viability of A2058 melanoma and patient-derived glioblastoma multiforme (GBM) cells in a concentration-dependent manner. Furthermore, when administered at 50 µM, KSL-128114 elevates survival times in a patient-derived xenograft (PDX) mouse model of GBM, where mice were implanted intracranially with patient-derived GBM cells pre-cultured with KSL-128114.

Promotes proteasomal degradation of Miro1 (mitochondrial Rho GTPase 1). Reduces Miro1 levels in fibroblasts from Parkinson's disease (PD) patients (IC50 = 7.8 μM). Exhibits no significant effect on related outer mitochondrial membrane protein Mitofusin. Reduces stress-induced degeneration of dopaminergic neurons derived from PD patient iPSCs. Rescues age-dependent neuronal loss and prolongs lifespan in fly PD models.

FGTI-2734 mesylate, a RAS C-terminal mimetic compound with dual farnesyl transferase (FT) and geranylgeranyl transferase-1 (GGT) inhibitory activities (IC50s of 250 nM and 520 nM for FT and GGT, respectively), mitigates KRAS resistance by preventing its membrane localization, effectively impeding mutant KRAS patient-derived pancreatic tumors.

5-Chlorouracil is a chlorinated derivative of the pyrimidine nucleoside base uracil . In vivo, it is converted into chlorodeoxyuridine, which is mutagenic and genotoxic.1 Uracil is chlorinated at the 5 position in a cell-free myeloperoxidase, peroxide, and chloride system in which hypochlorous acid is formed.2 5-Chlorouracil has been found in human neutrophils stimulated with phorbol 12-myristate 13-acetate in vitro and in inflammatory human exudate isolated from sites of superficial infection. Levels of 5-chlorouracil are increased in exudate isolated from the site of inflammation in a rat model of carrageenan-induced inflammation and in patient-derived human atherosclerotic aortic tissue.3,4References 5-Chlorouracil is a chlorinated derivative of the pyrimidine nucleoside base uracil . In vivo, it is converted into chlorodeoxyuridine, which is mutagenic and genotoxic.1 Uracil is chlorinated at the 5 position in a cell-free myeloperoxidase, peroxide, and chloride system in which hypochlorous acid is formed.2 5-Chlorouracil has been found in human neutrophils stimulated with phorbol 12-myristate 13-acetate in vitro and in inflammatory human exudate isolated from sites of superficial infection. Levels of 5-chlorouracil are increased in exudate isolated from the site of inflammation in a rat model of carrageenan-induced inflammation and in patient-derived human atherosclerotic aortic tissue.3,4 References

Potent and selective BTK Degrader (IC50 = 5.1 nM); degrades BTK in a proteasome- and CRBN-dependent manner. Suppresses BTK signaling and proliferation in mantle cell lymphoma (MCL) cells by degrading BTK, IKFZ1, and IKFZ3 (3 validated targets in B-cell malignancies). Also degrades Ibrutinib (Cat. No. 6813) -resistant C481S-BTK mutant cancer cells. Exhibits no binding against a panel of 468 kinases at 1 μM. Reduces tumor burden and extends survival in lymphoma patient-derived xenograft models.

VT02956 is a LATS inhibitor ( IC 50 : 0.76 nM for LATS1, 0.52 nM for LATS2). VT02956 targets the Hippo pathway. VT02956 inhibits ESR1 expression and growth of ER+ breast cancer cell lines and patient-derived tumor organoids .

1,2-Dipalmitoyl-sn-glycero-3-PE-N-(cap biotin) is a biotinylated phospholipid. It has been used in PEGylated polyamidoamine-dendrimer-conjugated supported lipid bilayers (SLB) to isolate circulating tumor cells and tumor cell microembolis from patient-derived blood by antibody-coated microfluidics. [1] It has also been used as a component of SLBs to detect protein-ligand binding with ortho-conjugated Texas Red DHPE. [2] In addition, 1,2-dipalmitoyl-sn-glycero-3-PE-N-(cap biotin) has been used in SLBs partitioned into nanowells to create DNA curtains, which can be used as a high-throughput tool for detection of protein-DNA interactions at the single molecule level.[3]

FGTI-2734 is a dual farnesyl transferase (FT) and geranylgeranyl transferase-1 (GGT-1) inhibitor, exhibiting IC50 values of 250 nM and 520 nM for FT and GGT-1, respectively. It effectively prevents the membrane localization of KRAS, addressing the issue of KRAS resistance and inhibiting the growth of mutant KRAS patient-derived pancreatic tumors.

SJ 11646, a potent lymphocyte-specific protein tyrosine kinase (LCK) Degrader (PROTAC) with a DC50 of 0.00838 pM, utilizes Dasatinib as the LCK ligand and incorporates a phenyl glutarimide-based cereblon binder. Demonstrating cytotoxicity in both LCK-activated T cell acute lymphoblastic leukemia (T-ALL) cell lines and primary leukemia samples in vitro, SJ 11646 also exhibits anti-leukemic efficacy in vivo within patient-derived xenograft models of T-ALL.

L-778123 is an inhibitor of FPTase and GGPTase-I, which was developed in part because it can completely inhibit Ki-Ras prenylation. The combination of L-778,123 and radiotherapy at dose level 1 showed acceptable toxicity in patients with locally advanced pancreatic cancer. Radiosensitization of a patient-derived pancreatic cancer cell line was observed.

YK-029A, an orally active EGFR mutant inhibitor, targets both EGFR T790M and EGFRex20ins mutations. It demonstrates notable antitumor efficacy, inducing tumor regression in EGFRex20ins-driven patient-derived xenograft (PDX) models [1].

Seribantumab (MM 121), a fully human IgG2 monoclonal antibody, specifically targets HER3, inhibiting the activation of the epidermal growth factor receptor (ErbB) family and its subsequent signaling. This action effectively blocks neuregulin 1 (NRG1) fusion-dependent tumorigenesis within in vitro and in vivo studies of patient-derived cancer models in breast, lung, and ovarian cancers [1].

Flotetuzumab (MGD006; S80880), an investigational CD123/CD3 bispecific dual-affinity retargeting antibody (DART) molecule, reactivates T cells through concurrent engagement with CD123 on target cells and CD3 on effector T cells, facilitating T-cell-mediated cytotoxicity in target cells. Demonstrating an inhibitory effect in a mouse model of patient-derived xenograft (PDX) for acute myeloid leukemia (AML) [1] [2], Flotetuzumab offers potential therapeutic prospects for AML treatment.

PF-9363 (CTX-3648) is a potent and high selective KAT6A KAT6B inhibitor. PF-9363 can be used for the research of cancer.

MTX-241F, a selective small molecule inhibitor, targets members of the EGFR and PI3 kinase families. It penetrates the blood-brain barrier, providing long-term tumor growth control and exhibits radiosensitizing activity in patient-derived DIPG neurospheres, potentially beneficial for studying diffuse intrinsic pontine glioma (DIPG) [1].

PARP1-IN-15 (Compound 6) is a PARP1 inhibitor that also inhibits tankyrase (TNKS) and promotes DNA double-strand breaks, leading to tumor cell apoptosis. It exhibits anti-cancer activity in triple-negative breast cancer (TNBC) cells and patient-derived organoids, making it useful for TNBC research, including cases with or without BRCA1 mutations [1].

ALDH3A1-IN-1 (Compound 18) is a potent ALDH3A1 inhibitor with an IC 50 of 1.61 μM. It is more effective than DEAB against patient-derived primary prostate tumor epithelial cells, both as a single agent and in combination with docetaxel [1].

TG693, an orally available inhibitor of CLK1, promotes the skipping of the endogenous mutated exon 31 in Duchenne muscular dystrophy (DMD) patient-derived cells and increased the production of the functional exon 31-skipped dystrophin protein.