Advanced Research in Anticancer Inhibitors and Targeted Therapy

A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Drug Discovery, Development and Delivery".

Deadline for manuscript submissions: 31 October 2025 | Viewed by 488

Special Issue Editors


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Guest Editor
School of Medicine and Health Sciences, The George Washington University, Washington, DC, USA
Interests: histone deacetylases (HDACs); YAP signaling; drug resistance in cancer; protein O-GlcNAcylation

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Guest Editor
Department of Biomedical Sciences, Edward Via College of Osteopathic Medicine, Louisiana Campus, Monroe, LA 71203, USA
Interests: drug discovery; cancer pharmacology; novel molecular targets; herbal medicine
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Special Issue Information

Dear Colleagues,

Rapidly advancing targeted cancer therapy has significantly improved treatment efficacy by selectively inhibiting oncogenic signaling pathways. Among these, histone deacetylase (HDAC) inhibitors, kinase inhibitors, and immune checkpoint inhibitors have demonstrated remarkable potential in both preclinical and clinical studies. However, the emergence of drug resistance, tumor heterogeneity, and adverse side effects pose substantial challenges to their clinical application.

This Special Issue aims to explore novel anticancer inhibitors, their molecular mechanisms, and potential combination strategies to overcome resistance, seeking to provide novel insights into improving the effectiveness and durability of targeted cancer therapies. We welcome original research and review articles focusing on epigenetic therapy, small-molecule inhibitors, immune-based treatments, and new therapeutic targets. Studies employing biochemical, molecular, and cellular approaches to investigate drug efficacy, biomarker discovery, and resistance mechanisms are highly encouraged.

Dr. Changmin Peng
Dr. Hassan Ebrahim
Guest Editors

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Keywords

  • histone deacetylases (HDACs)
  • YAP signaling
  • targeted therapy
  • oncogenic signaling
  • biomarker discovery
  • small-molecule inhibitors
  • cancer drug resistance
  • epigenetic therapy

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Published Papers (1 paper)

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12 pages, 693 KiB  
Article
Efficacy and Safety of the Combination of Durvalumab Plus Gemcitabine and Cisplatin in Patients with Advanced Biliary Tract Cancer: A Real-World Retrospective Cohort Study
by Eishin Kurihara, Satoru Kakizaki, Masashi Ijima, Takeshi Hatanaka, Norio Kubo, Yuhei Suzuki, Hidetoshi Yasuoka, Takashi Hoshino, Atsushi Naganuma, Noriyuki Tani, Yuichi Yamazaki and Toshio Uraoka
Biomedicines 2025, 13(8), 1915; https://doi.org/10.3390/biomedicines13081915 - 6 Aug 2025
Viewed by 310
Abstract
Background/Objectives: The TOPAZ-1 phase III trial reported a survival benefit of using durvalumab, an anti-programmed death ligand 1 (anti-PD-L1) antibody, in combination with gemcitabine and cisplatin (GCD) treatment in patients with advanced biliary tract cancer. This retrospective study investigated the efficacy and [...] Read more.
Background/Objectives: The TOPAZ-1 phase III trial reported a survival benefit of using durvalumab, an anti-programmed death ligand 1 (anti-PD-L1) antibody, in combination with gemcitabine and cisplatin (GCD) treatment in patients with advanced biliary tract cancer. This retrospective study investigated the efficacy and safety of GCD treatment for advanced biliary tract cancer in real-world conditions. Methods: The study subjects were 52 patients with biliary tract cancer who received GCD therapy between January 2023 and May 2024. The observation parameters included the modified Glasgow Prognostic Score (mGPS), neutrophil–lymphocyte ratio (NLR), platelet–lymphocyte ratio (PLR), tumor markers (CEA, CA19-9), overall response rate (ORR), disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and adverse events. Results: The cohort included 36 men and 16 women, with a median age of 73.0 years. There were 36 cases of cholangiocarcinoma (distal: 10, perihilar: 19, intrahepatic: 7), 13 cases of gallbladder cancer, and 3 cases of ampullary carcinoma. The stages were locally advanced in 30 cases and metastatic in 22 cases. Biliary drainage was performed in 30 cases. There were 38 cases receiving first-line therapy and 14 cases receiving second-line or later treatments. The median values at the start of GCD therapy were ALB 3.7 g/dL, CRP 0.39 mg/dL, NLR 2.4, PLR 162.5, CEA 4.8 ng/mL, and CA19-9 255.9 U/mL. The mGPS distribution was 0:23 cases, 1:18 cases, and 2:11 cases. The treatment outcomes were ORR 25.0% (CR 2 cases, PR 11 cases), DCR 78.8% (SD 28 cases, PD 10 cases, NE 1 case), median PFS 8.6 months, and median OS 13.9 months. The PLR was suggested to be useful for predicting PFS. A decrease in CEA at six weeks after the start of treatment was a significant predictor of PFS and OS. Gallbladder cancer had a significantly poorer prognosis compared to other cancers. The immune-related adverse events included hypothyroidism in two cases, cholangitis in one case, and colitis in one case. Conclusions: The ORR, DCR, and PFS were comparable to those in the TOPAZ-1 trial. Although limited by its retrospective design and small sample size, this study suggests that GCD therapy is an effective treatment regimen for unresectable biliary tract cancer in real-world clinical practice. Full article
(This article belongs to the Special Issue Advanced Research in Anticancer Inhibitors and Targeted Therapy)
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