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Cancers
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Preclinical and Clinical Research on the Efficacy of Anticancer Drugs
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Preclinical and Clinical Research on the Efficacy of Anticancer Drugs

A special issue of Cancers (ISSN 2072-6694). This special issue belongs to the section "Clinical Research of Cancer".

Deadline for manuscript submissions: 7 August 2026 | Viewed by 11812

Special Issue Editors

Prof. Dr. Andrzej Hellmann

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Guest Editor
Faculty of Medicine, Medical University of Gdańsk, Gdansk, Poland
Interests: leukemia; cell transplantation; cancer therapy; hematologic malignancies
Prof. Dr. Joanna Jakobkiewicz-Banecka

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Guest Editor
Department Medical Biology and Genetics, Faculty of Biology, University of Gdansk, Gdansk, Poland
Interests: metabolic disorders; epigenetics; cancer genetics; gene expression
Dr. Jan Lica

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Guest Editor
Department Health Science; Powiśle University, 80-214 Gdańsk, Poland
Interests: cancer stem cell; molecular mechanisms; cell biology

Special Issue Information

Dear Colleagues,

“Preclinical and Clinical Research on the Efficacy of Anticancer Drugs" explores the comprehensive evaluation processes involved in the development of cancer therapies. This field encompasses both preclinical studies, which involve laboratory and animal testing to determine the safety and biological activity of new drugs, and clinical trials, which assess their therapeutic effectiveness and safety in human patients. The integration of these studies is crucial for identifying promising anticancer agents, optimizing their formulations, and ensuring they meet regulatory standards before reaching the market.

Authors are encouraged to present findings that contribute to understanding drug mechanisms, improving trial methodologies, and developing novel therapeutic strategies. Original research articles and review articles are welcome.

Prof. Dr. Andrzej Hellmann
Prof. Dr. Joanna Jakobkiewicz-Banecka
Dr. Jan Lica
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Cancers is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • clinical trials
  • preclinical research
  • anticancer drugs
  • drug efficacy
  • drug development
  • cancer therapy
  • therapeutic strategies
  • safety assessment
  • pharmacodynamics
  • pharmacokinetics

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Published Papers (4 papers)

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Research

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15 pages, 962 KB  
Article
Pharmacologic Targeting of miR29b with Bortezomib and Sorafenib to Improve Decitabine Sensitivity in Patients with Acute Myeloid Leukemia: Results from a Phase 1 Dose-Escalation Trial
by Shivani Handa, Kristin Koenig, Qiuhong Zhao, Alice S. Mims, Sumithira Vasu, Ramiro Garzon, Tamanna Haque, Don Benson, Rebecca B. Klisovic, Guido Marcucci, Alison R. Walker and Bhavana Bhatnagar
Cancers 2026, 18(1), 45; https://doi.org/10.3390/cancers18010045 - 23 Dec 2025
Viewed by 615
Abstract
Background: Decitabine efficacy in acute myeloid leukemia (AML) may be enhanced by the pharmacologic upregulation of microRNA miR-29b, a regulator of DNA methyltransferase (DNMT) expression. Bortezomib and sorafenib have been shown preclinically to increase miR-29b levels, providing a biologically informed strategy to sensitize [...] Read more.
Background: Decitabine efficacy in acute myeloid leukemia (AML) may be enhanced by the pharmacologic upregulation of microRNA miR-29b, a regulator of DNA methyltransferase (DNMT) expression. Bortezomib and sorafenib have been shown preclinically to increase miR-29b levels, providing a biologically informed strategy to sensitize leukemic blasts to DNMT inhibition. Objectives: To evaluate the safety, tolerability, biological activity, and preliminary efficacy of combining bortezomib and sorafenib followed by decitabine in patients with newly diagnosed or relapsed/refractory AML. Methods: This phase I, dose-escalation study enrolled 15 patients (11 untreated, 4 relapsed/refractory) who received fixed-dose bortezomib and sorafenib across three dose levels prior to decitabine. Dose escalation was guided by dose-limiting toxicities (DLTs) and an increase in miR-29b expression. Results: The regimen was generally well tolerated with the most frequent grade ≥3 adverse events of hypertension and febrile neutropenia. At the highest dose level, a ≥2-fold increase in miR-29b expression was observed in two of the six evaluable patients. The overall response rate was 33.3%, with clinical responses observed in both newly diagnosed and relapsed/refractory patients. However, changes in miR-29b expression did not consistently correlate with clinical response. Conclusions: Sequential treatment with bortezomib and sorafenib followed by decitabine is feasible and demonstrates acceptable safety in AML. Although the biologic modulation of miR-29b was variable, this trial provides a proof of concept for pharmacodynamic-guided dose finding in epigenetic therapy combinations. Full article
(This article belongs to the Special Issue Preclinical and Clinical Research on the Efficacy of Anticancer Drugs)
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10 pages, 1688 KB  
Communication
Assessment of Objective Response Rate by Investigator vs. Blinded Independent Central Review in Pivotal Trials of Oncology Drugs for Solid Tumor Indications
by Marjorie E. Zettler
Cancers 2025, 17(7), 1096; https://doi.org/10.3390/cancers17071096 - 25 Mar 2025
Cited by 3 | Viewed by 4851
Abstract
Background/Objective: Objective response rate (ORR) is a surrogate endpoint frequently employed in early-phase clinical trials of anticancer agents for the treatment of solid tumors. Assessments of ORR by local investigators tend to be influenced by subjective factors, and blinded independent central review (BICR) [...] Read more.
Background/Objective: Objective response rate (ORR) is a surrogate endpoint frequently employed in early-phase clinical trials of anticancer agents for the treatment of solid tumors. Assessments of ORR by local investigators tend to be influenced by subjective factors, and blinded independent central review (BICR) is recommended by regulatory agencies in order to detect evaluation bias. The objective of this analysis was to compare BICR-assessed vs. investigator-assessed ORRs in pivotal trials of cancer drugs recently approved by the United States Food and Drug Administration (FDA) for solid tumor indications. Methods: The FDA’s Novel Drug Approvals reports were reviewed to identify cancer therapies approved for solid tumor indications between 1 January 2020 and 30 June 2024. Among therapies with ORR as a primary endpoint in pivotal trials, and for which both BICR- and investigator-assessed ORRs were available, a pooled analysis was conducted to compare these ORRs (using the Mantel–Haenszel method). A correlation analysis was also performed to evaluate the concordance between ORR assessments. Results: A total of 20 anticancer agents met the criteria for inclusion in this analysis, each supported by a single pivotal trial. Comparing BICR- and investigator-assessed ORRs in a pooled analysis did not identify any significant difference between the two assessments overall: OR = 0.98 (95% CI: 0.87–1.11), p = 0.75, and I2 = 0%. The correlation analysis also revealed a high level of concordance between BICR- and investigator-assessed ORRs, with r = 0.96 (p < 0.05). Conclusions: This study found no evidence of evaluation bias in the assessment of ORR among registrational trials supporting recent FDA approvals of anticancer agents for solid tumor indications. Full article
(This article belongs to the Special Issue Preclinical and Clinical Research on the Efficacy of Anticancer Drugs)
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20 pages, 500 KB  
Article
Bevacizumab-Based Therapies in Malignant Tumors—Real-World Data on Effectiveness, Safety, and Cost
by Elena Chitoran, Vlad Rotaru, Sinziana-Octavia Ionescu, Aisa Gelal, Cristina-Mirela Capsa, Roxana-Elena Bohiltea, Madalina-Nicoleta Mitroiu, Dragos Serban, Giuseppe Gullo, Daniela-Cristina Stefan and Laurentiu Simion
Cancers 2024, 16(14), 2590; https://doi.org/10.3390/cancers16142590 - 19 Jul 2024
Cited by 13 | Viewed by 4237
Abstract
Overall, it is estimated that more than 3,500,000 patients have received Bevacizumab as part of systemic oncologic treatment. Bevacizumab and its biosimilars are currently marketed in over 130 countries. Given the wide usage of Bevacizumab in current oncological practice, it is very important [...] Read more.
Overall, it is estimated that more than 3,500,000 patients have received Bevacizumab as part of systemic oncologic treatment. Bevacizumab and its biosimilars are currently marketed in over 130 countries. Given the wide usage of Bevacizumab in current oncological practice, it is very important to compare the “real-world” results to those obtained in controlled clinical trials. This study aims to describe the clinical experience of using Bevacizumab in a large cohort of cancer patients in “non-controlled real-world” conditions with regard to effectiveness, safety, and cost of therapy. Methods: For this purpose, we conducted an open, observational, retrospective study involving all patients treated for solid malignant tumors in the Bucharest Institute of Oncology with “Prof. Dr. Al. Trestioreanu” with Bevacizumab-based systemic therapy, between 2017 and 2021. Results: The study consisted of 657 treatment episodes in 625 patients (F/B = 1.62/1, with a median age of 57.6 years) which were treated for malignant tumors (majority colorectal, non-small cell lung, ovarian, and breast cancer). First-line treatment was administered in 229 patients, and the rest received Bevacizumab as second or subsequent lines of treatment. The overall response rate to Bevacizumab-based therapies was around 60–65% across all indication except for subsequent treatment lines in colorectal and ovarian cancers, where lower values were recorded (27.1%, and 31.5% respectively). Median PFS for the entire cohort was 8.2 months (95% CI 6.8–9.6), and the median OS was 13.2 months (95% CI 11.5–14.9). Usual bevacizumab-related toxicities were observed, including bleeding, hypertension, wound-healing complications, gastrointestinal perforation, other types of fistulas, septic complications, and thromboembolic events. Although the clinical benefits are undeniable, the addition of Bevacizumab to standard chemotherapy increased the overall treatment cost by 213%. Conclusions: Bevacizumab remains a high-cost therapy, but it can add to clinical benefits (like overall survival, progression-free survival, and response rate) when used in conjunction with standard chemotherapy. Similar results as those presented in various controlled trials are observable even on unselected cohorts of patients in the uncontrolled conditions of “real-world” oncological practice. Off-label usage is encountered in clinical practice, and this aspect should be monitored given the potential adverse effects of the therapy. Full article
(This article belongs to the Special Issue Preclinical and Clinical Research on the Efficacy of Anticancer Drugs)
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Review

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25 pages, 2428 KB  
Review
Triptolide: A Narrative Review of Its Traditional Use, Derivatives, Pharmacology, Antitumor Effect, and Clinical Applications
by Yibo Geng, Bettina Kritzer and Javad Nazarian
Cancers 2026, 18(8), 1196; https://doi.org/10.3390/cancers18081196 - 9 Apr 2026
Viewed by 1085
Abstract
Triptolide (TPL), a diterpenoid derived from the Chinese medicinal plant Tripterygium wilfordii, exhibits broad-spectrum biological and pharmacological activities, although its clinical translation is limited by systemic toxicity. Recent advances in the development of TPL derivatives have created new therapeutic opportunities. This review [...] Read more.
Triptolide (TPL), a diterpenoid derived from the Chinese medicinal plant Tripterygium wilfordii, exhibits broad-spectrum biological and pharmacological activities, although its clinical translation is limited by systemic toxicity. Recent advances in the development of TPL derivatives have created new therapeutic opportunities. This review summarizes current knowledge of triptolide, with a focus on TPL’s toxicity profile, derivative strategies, and antitumor mechanisms across different tumor types, including glioma, pancreatic tumor, leukemia, lung cancer, gastric cancer and others. We also summarize the plant’s origin and traditional uses, TPL’s pharmacokinetics (PKs), and relevant clinical trials against tumors. The main mechanism of the TPL antitumor effect is to interfere with ATPase of XPB by covalently binding to it, as well as inducing the rapid depletion of RPB1 via hyperphosphorylation and ubiquitination. We also reviewed systemic toxicity including neuro-, cardio-, oto-, nephron-, hepato-, and hemato-toxicity, as well as digestive and reproductive toxicity. Finally, we searched clinical trial databases across three platforms for tumors and concluded that Minnelide has strong clinical potential for solid tumors. By critically evaluating TPL from multiple dimensions, specifically its traditional use, chemical derivatization, pharmacokinetics, antitumor mechanisms, toxicity, and clinical trials, this review aims to inform future strategies that maximize therapeutic efficacy while minimizing adverse effects. Full article
(This article belongs to the Special Issue Preclinical and Clinical Research on the Efficacy of Anticancer Drugs)
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