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Pharmaceuticals
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Advances in Targeted Therapy for Gastrointestinal Cancers
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Advances in Targeted Therapy for Gastrointestinal Cancers

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmacology".

Deadline for manuscript submissions: 31 July 2026 | Viewed by 755

Special Issue Editors

Dr. Juraj Prejac

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Guest Editor
1. Department of Oncology, University Hospital Centre Zagreb, Kišpatićeva 12, 10000 Zagreb, Croatia
2. School of Dental Medicine, University of Zagreb, Gundulićeva 5, 10000 Zagreb, Croatia
Interests: hepatobiliary cancers; colorectal cancer; gastric cancer; immunotherapy; targeted therapy
Dr. Marin Golčić

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Guest Editor
1. Tumor Clinic, Clinical Hospital Center Rijeka, 51000 Rijeka, Croatia
2. School of Medicine, University of Rijeka, 51000 Rijeka, Croatia
Interests: gastrontestinal cancers; skin cancers; palliative care
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

This Special Issue, “Advances in Targeted Therapy for Gastrointestinal Cancers”, aims to highlight recent progress and emerging strategies in the development of targeted therapies for gastrointestinal (GI) malignancies. GI cancers, including those of the esophagus, stomach, pancreas, liver, bile ducts, small intestines, colorectum, and anus, represent a significant global health burden. In recent years, we have witnessed the development of targeted therapies and immunotherapies, which dramatically improved the outcomes for many patients. However, many challenges remain, as only a small subset of GI cancer patients benefits from currently approved drugs. Furthermore, due to complex tumor biology, both primary and acquired resistance frequently occur. Along with new medications, there is also a need for better prognostic and predictive biomarkers. 

This Issue welcomes original research articles, reviews, and short communications that address novel targets, mechanisms of drug resistance, biomarker discovery, clinical trial results, and translational approaches in the field of GI cancer therapeutics. By gathering cutting-edge findings and expert perspectives, this Special Issue seeks to foster scientific exchange and accelerate the development of more effective, personalized treatments for patients with gastrointestinal cancers.

Dr. Juraj Prejac
Dr. Marin Golčić
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 short 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. Pharmaceuticals is an international peer-reviewed open access monthly 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

  • targeted therapy
  • gastrointestinal cancers
  • molecular targets
  • drug resistance
  • biomarkers
  • immunotherapy
  • precision medicine
  • translational research
  • clinical trials
  • tumor microenvironment

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Published Papers (1 paper)

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Review

19 pages, 735 KB  
Review
Targeting the Unmet Need in Gastrointestinal Stromal Tumors: A Contemporary Review of Investigational Clinical Trials and Therapeutic Landscape
by Andrej Belančić, Juraj Prejac, Marin Golčić, Gordan Adžić, Andrija Katić, Lidija Kocić, Anamarija Kovač Peić, Nikša Librenjak, Borislav Belev, Ivana Mikolašević and Stjepko Pleština
Pharmaceuticals 2026, 19(4), 548; https://doi.org/10.3390/ph19040548 - 29 Mar 2026
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Abstract
Background: Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal neoplasms of the gastrointestinal tract and are primarily driven by activating mutations in KIT or PDGFRA. Although tyrosine kinase inhibitors (TKIs), particularly imatinib, have substantially improved outcomes, most patients with advanced disease [...] Read more.
Background: Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal neoplasms of the gastrointestinal tract and are primarily driven by activating mutations in KIT or PDGFRA. Although tyrosine kinase inhibitors (TKIs), particularly imatinib, have substantially improved outcomes, most patients with advanced disease eventually develop resistance, resulting in disease progression. Methods: We performed a narrative review with scoping approach of interventional clinical trials registered on ClinicalTrials.gov between January 2020 and July 2025 to characterize the contemporary investigational therapeutic landscape in GIST. Eligible studies included clinical trials evaluating novel agents, combinations, or alternative strategies beyond current regulatory approvals. Trial characteristics, therapeutic classes, endpoints, enrollment, and funding sources were analyzed. Results: A total of 27 ongoing trials were identified. Most studies were phase I/II and focused on metastatic or unresectable disease, predominantly in the second-line or later settings. TKIs remained the dominant therapeutic class, included in over 70% of trials, either as monotherapy or in combination. Emerging strategies comprised antibody–drug conjugates, immune checkpoint inhibitors, HIF inhibitors, FGFR inhibitors, and epigenetic modulators. Only four phase III trials were identified, reflecting the difficulty of conducting large, randomized studies in GIST. No trial used overall survival or quality of life as a primary endpoint. Conclusions: The current investigational landscape in GIST is largely focused on overcoming TKI resistance in advanced disease. Molecular stratification and personalized approaches dominate ongoing research, but evidence generation remains limited by small sample sizes and slow recruitment. Future trials integrating innovative therapeutic platforms and patient-centered outcomes are essential to improve long-term disease control and quality of life. Full article
(This article belongs to the Special Issue Advances in Targeted Therapy for Gastrointestinal Cancers)
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