New therapy shrinks tumors to treat lung cancer

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by Stony Brook University

edited by Gaby Clark, reviewed by Andrew Zinin

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Graphical abstract. Credit: Molecular Therapy Nucleic Acids (2026). DOI: 10.1016/j.omtn.2026.102997

The need for more effective lung cancer treatments is paramount, as the disease remains a leading cause of cancer deaths in the United States. New findings by a research team from the Stony Brook Cancer Center highlight a novel therapeutic shown to be highly effective in reducing non-small cell lung cancer (NSCLC) tumors and appearing to halt drug resistance in a laboratory model.

Led by Jingfang Ju, Ph.D., professor in the Department of Pathology at the Renaissance School of Medicine (RSOM) at Stony Brook University and a researcher at the Stony Brook Cancer Center, the team designed a multitargeted, multimodal tumor-suppressor miRNA-129-based therapy to combat drug resistance in NSCLC by improving the agent's stability, deliverability and efficacy. The molecule miRNA-129 is a noncoding RNA molecule that regulates gene expression and often acts as a tumor suppressor in the human body.

The team tested this approach in an NSCLC murine model. Essentially, they combined the natural cancer-fighting molecule (miR-129) with a commonly used chemotherapy drug for lung cancer (gemcitabine). Their drug, Gem-miR-129, can enter cancer cells on its own without the need for a delivery vehicle. Unlike single-targeted therapies for NSCLC, it simultaneously shuts down three different oncogenic driver proteins that help lung cancer grow and resist therapy. The driver proteins are HMGB1, YAP1 and PBX3.

"The results in the model were remarkable, with tumor shrinkage reaching more than 95%," Ju says. "Additionally, the survival time for the mice treated with Gem-miR-129 increased by many weeks, which may translate to approximately 5 to 15 more years in terms of human life."

The paper is published in the journal Molecular Therapy: Nucleic Acids.

The approach and action of the new agent

Currently, gemcitabine is a standard first-line chemotherapy treatment for humans with NSCLC. Recent advancements in targeted therapies have improved survival for a subset of patients with NSCLC harboring significant oncogenic driver mutations, namely those with mutations in the epidermal growth factor receptor (EGFR). These patients respond to drugs called tyrosine kinase inhibitors (TKIs), which result in an EGFR blockade. Despite a good initial response to TKIs, about half of cancers acquire resistance to TKIs.

Gem-miR-129 is designed for the subset of NSCLC tumors that develop TKI resistance. It prevents resistance because the new agent suppresses several oncogenes associated with both intrinsic and acquired resistance to chemotherapy such as gemcitabine and EGFR inhibitors.

Ju explains that once the gemcitabine-modified (Gem) drug is released from Gem-miR-129, low-dose Gem will inhibit tumor-infiltrating T regulatory cells (Ti-Tregs), which block cytotoxic T cells (CD4 and CD8) from eliminating tumor cells. By reducing Ti-Tregs, the agent can remove the inhibitory effect on CD4 and CD8 T cells so they can reduce the tumor burden.

"Resistance is the primary reason why current lung cancer therapies stop working for cancer patients, and for that reason, this approach is highly promising," Ju says. "In the model, we also did not see the agent causing any noticeable toxic side effects. We believe this new approach provides a proof of concept for future studies that could lead to clinical trials in NSCLC patients."

Ju says the next step in the team's research will be to pursue Investigational New Drug (IND) studies for Gem-miR-129. The IND process is designed to establish a drug's safety profile with the hope of gaining regulatory approval for human clinical trials.

More information

Erick M. Intriago et al, Development of gemcitabine-modified multimodal miR-129 mimic as a novel therapeutic in non-small cell lung cancer, Molecular Therapy Nucleic Acids (2026). DOI: 10.1016/j.omtn.2026.102997

Key medical concepts

Carcinoma, Non-Small-Cell LungGemcitabine

Clinical categories

OncologyPulmonary medicine Provided by Stony Brook University Who's behind this story?

Gaby Clark

MA in English, copy editor since 2021 with experience in higher education and health content. Dedicated to trustworthy science news. Full profile →

Andrew Zinin

Master's in physics with research experience. Long-time science news enthusiast. Plays key role in Science X's editorial success. Full profile →

Citation: New therapy shrinks tumors to treat lung cancer (2026, August 4) retrieved 5 August 2026 from https://medicalxpress.com/news/2026-08-therapy-tumors-lung-cancer.html This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.