UT MD Anderson Shares Latest Research Breakthroughs

At The University of Texas MD Anderson Cancer Center, research breakthroughs are made possible through seamless collaboration between the institution’s world-leading clinicians and scientists, bringing discoveries from the lab to the clinic and back. The studies below showcase the latest advances in cancer care, research and prevention.

First-line targeted therapy shows antitumor activity in patients with advanced lung cancer

Read the full release | Read the study in The New England Journal of Medicine

First-line zongertinib showed antitumor activity in treatment-naïve patients with advanced or metastatic HER2-mutant non-small cell lung cancer (NSCLC), providing a safe and effective oral targeted treatment alternative to chemotherapy. Results from the multi-site Phase Ia/Ib Beamion LUNG-1 trial reported a 76% objective response rate (ORR), indicating tumor shrinkage in 56 of 74 patients. Responses lasted for a median duration of 15.2 months, with no progression of disease for over 14 months. This data supports the recent accelerated approval of zongertinib by the Food and Drug Administration (FDA) for the treatment of unresectable or metastatic HER2-mutant NSCLC. 

“We observed unprecedented response rates for this cancer subtype, and these findings led to zongertinib becoming the first FDA-approved treatment of its kind for these patients,” said principal investigator John Heymach, M.D., Ph.D., chair of Thoracic/Head and Neck Medical Oncology. “What’s particularly exciting is that just a few years ago, patients with this disease had no effective targeted therapies. Now, health care providers have a HER2-targeted treatment option that can make a meaningful difference.”

Novel combination therapy yields high response rates in early-stage classical Hodgkin lymphoma

Read the full release | Read the study in Blood

The combination of abbreviated chemotherapy with brentuximab vedotin and nivolumab achieved high response rates in patients with non-bulky, early-stage classical Hodgkin lymphoma (cHL). By using this combination approach, researchers of the Phase 2 trial, led by Hun Lee, M.D., associate professor of Lymphoma, were able to omit two chemotherapy drugs along with radiation therapy for these patients, a significant de-escalation of treatment.

“We are pleased with these outcomes, which were consistent across both favorable- and unfavorable-risk groups,” Lee said. “Our ability to omit several medications and radiation therapy demonstrates that we can maintain or improve outcomes and reduce treatment toxicity.”

Researchers identify how enzyme affects infertility and cancer progression

Read the full release | Read the study in Nature Communications

Activation of a specific part of the Dicer enzyme can change its shape in a way that affects its critical role in proper cell division, with implications for both cancer biology and fertility.

The study, led by Swathi Arur, Ph.D., professor of Genetics, fills a major gap in understanding how the Dicer enzyme is regulated. The results open new avenues for studying how small epigenetic changes – which turn genes on or off without altering their sequence – and other disruptions in these pathways can affect Dicer’s role in a way that can contribute to both cancer progression and infertility.

“These findings expand the implications of Dicer regulation beyond germline biology by suggesting that epigenetic modifications can tune its shape and the proteins it recruits, creating a potential mechanism for disease,” Arur said. “In cancer, where DICER1 dysfunction is already linked to altered cell identity and tumor progression, this raises the possibility that irregular epigenetic activity could reshape these networks in ways that promote cancer.”

Imaging tool reveals novel insights into DNA replication stress response

Read the full release | Read the study in Nature Communications

Researchers have developed a new imaging method, known as RF-SIRF, that quantitatively detects and maps reversed DNA replication forks with single-cell resolution. The results also demonstrated a unique epigenetic code for DNA replication stress that can be further examined to understand mechanisms of genomic stability, aging and treatment response. The study was led by Katharina Schlacher, Ph.D., associate professor of Cancer Biology.

“By capturing reversed DNA replication forks in their spatiotemporal context, our new assay identifies site-specific epigenetic signatures,” Schlacher said. “This technology provides a unique lens, enabling scientists to decode cancer-specific DNA replication stress dynamics and their crosstalk with inflammation and transcription programs, representing a major step in precision oncology.”

FLAG-based regimen delivers strong outcomes in subtype of acute myeloid leukemia

Read the full release | Read the study in Blood Cancer Discovery

A new analysis demonstrates that combination therapy consisting of fludarabine, cytarabine and G-CSF (FLAG) plus gemtuzumab ozogamicin (GO) or idarubicin (IDA) continues to deliver strong long-term outcomes for patients with core-binding factor acute myeloid leukemia (CBF-AML), a subtype of the disease involving a chromosomal rearrangement. The study was co-led by Gautam Borthakur, M.D., professor of Leukemia, and Jayastu Senapati, M.D., assistant professor of Leukemia. The researchers analyzed long-term outcomes from a cohort of patients treated in a Phase 2 trial. The five-year overall survival rate reached 74%, while the relapse-free survival rate was 67% for patients treated in this study. Among patients who had received GO along with FLAG (FLAG-GO), the five-year overall survival rate was superior at 80% as opposed to FLAG-IDA on non-randomized comparisons.

“This is one of the highest reported five-year, relapse-free overall survival rates we have observed,” Borthakur said. “This regimen is now our standard frontline therapy for adults with core-binding factor AML, and these findings further strengthen the evidence supporting its use.”

Novel tool more accurately predicts risk of Li-Fraumeni Syndrome

Read the full release | Read the study in The American Journal of Human Genetics

In a prospective validation study, researchers demonstrated that a new mathematical model, called LFSPRO, was effective in supporting genetic counselor decision-making and more accurately predicted individuals at higher risk of Li-Fraumeni Syndrome (LFS), a hereditary condition that carries a higher risk of developing multiple cancers. LFSPRO provided risk scores that significantly outperformed current criteria for genetic testing and provided valuable insights and quantifiable data for genetic counselors that more closely aligned with their clinical judgment. The study was led by Wenyi Wang, Ph.D., professor of Bioinformatics and Computational Biology, and Banu Arun, M.D., professor of Breast Medical Oncology.

“Most risk prediction models are validated only in research-based cohorts, but our study demonstrates LFSPRO's performance in a real-world genetic counseling setting, where limited family history information is provided within 30 minutes and most counselees test negative,” Wang said. “Our model demonstrated substantially higher accuracy than current clinical guidelines and had strong concordance with genetic counselors' judgment.”

New simulation framework DKOsim advances CRISPR-based gene to gene interaction research

Read the full release | Read the study in PLOS Computational Biology

Researchers developed Double-CRISPR Knockout Simulation (DKOsim), a novel computational framework designed to address longstanding challenges in profiling gene to gene interactions. The findings may help streamline genetic interactions detection through optimized computational methods and guide the development of next-generation dual-knockout CRISPR screens.

“DKOsim provides a much-needed bridge between experimental and computational biology,” said John Paul Shen, M.D., assistant professor of Gastrointestinal Medical Oncology. “It enables scientists to systematically test a hypothesis, optimize experimental design and benchmark analytical tools under controlled conditions.”

Awards and Honors

• John Weinstein, M.D., Ph.D., professor of Bioinformatics and Computational Biology, was inducted into the 2026 Class of the International Society for Computational Biology (ISCB) Distinguished Fellows

UT MD Anderson at AACR

Read highlights from UT MD Anderson at the American Association for Cancer Research (AACR) Annual Meeting 2026. More information can be found at MDAnderson.org/AACR.

• Novel approach prevents cancer progression, spares surgery for majority of patients with precancerous oral lesions
• New platform uses machine learning to predict responses in patients with lung cancer
• Zedoresertib and lunresertib combination shows promising antitumor activity
• Clinical trial presentations feature advances across cancer care
• UT MD Anderson shares latest breakthroughs in cancer research
• Jennifer Wargo, M.D., elected Fellow of the AACR Academy
• Immunotherapy pioneer James P. Allison, Ph.D., honored with Award for Lifetime Achievement in Cancer Research