- Oral combination therapy demonstrates high response rates for patients with hard-to-treat acute myeloid leukemia
- Research brings new biological understanding of cancer cell origin and evolution, age-related inflammation
- Studies provide insights into the tumor immune microenvironments of brain metastases treated with radiation therapy and of muscle-invasive bladder cancer
- New gene therapy successfully delivers large proteins and restores muscle function in models of muscular dystrophy
HOUSTON, JUNE 18, 2026 ― 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.
EHA: Oral combination shows high response rates in patients with hard-to-treat acute myeloid leukemia
Read the full release | Read the study in the Journal of Clinical Oncology
In patients with advanced acute myeloid leukemia (AML) harboring menin-dependent genetic alterations, 71% achieved a composite complete remission when treated with an all-oral combination therapy of revumenib, decitabine/cedazuridine and venetoclax. The Phase 1/2 investigator-initiated SAVE clinical trial enrolled adolescent and adult patients with relapsed or refractory AML with KMT2A rearrangements, NPM1 mutations or NUP98 rearrangements. The combination therapy achieved an 88% overall response rate and a complete remission or complete remission with partial hematologic recovery (CR/CRh) rate of 60%, with 80% of responding patients achieving measurable residual disease negativity by flow cytometry. The median duration of CR/CRh was 10.5 months overall, and the median had not yet been reached among patients with KMT2A rearrangements, a signal of durable responses.
“These results highlight the potential of combining menin inhibition with BCL2 inhibition and a hypomethylating agent for genetically defined AML subtypes that historically have been very difficult to treat,” said principal investigator Ghayas Issa, M.D., associate professor of Leukemia. “Seeing durable remissions in patients who had already received multiple prior therapies is encouraging and supports continued evaluation of this combination strategy.”
Many cancers evolve through early bursts of chromosome changes and originate from a single cancer cell
Read the full release | Read the study in Cancer Discovery
Cancer cells within tumors are genetically diverse, yet all carry the same core genetic changes that can be traced back to a common ancestral cell, providing a single-cell view of how tumors adapt, survive and diversify. Understanding this helps explain why some cancer cells manage to survive treatments, paving the way for more tailored diagnostic and therapeutic strategies. The study, led by Nicholas Navin, Ph.D., chair of Systems Biology, shows that cancer cells do not evolve slowly over time but, rather, grow through sudden bursts of rapid genetic changes that include copy number alterations (CNAs) – gains or losses of entire sections of DNA. This creates a family tree of distinct new subpopulations that can influence tumor aggressiveness, metastasis and treatment response.
“Our findings provide the clearest views to date of how cancers originate and evolve at the single-cell level,” Navin said. “By revealing both the shared early genetic events and the bursts that drive ongoing diversity, we now have a roadmap for developing smarter clinical diagnostic and treatment strategies to improve patient outcomes.”
Spatial map of bladder cancer reveals hidden tumor environments and new paths toward precision therapy
Read the full release | Read the study in Cancer Discovery
Researchers have developed a spatial map of muscle-invasive bladder cancer, revealing how tumor cell states, immune environments and therapeutic vulnerabilities are organized within tumors. The study provides a new framework for understanding why patients with bladder cancer may respond differently to treatment. The research was led by Linghua Wang, M.D., Ph.D., professor of Genomic Medicine, executive director and head of the Center for Cellular Language Intelligence, associate member of the James P. Allison Institute™, and focus area co-lead with the Institute for Data Science in Oncology; together with Jianjun Gao, M.D., Ph.D., professor of Genitourinary Medical Oncology, and co-first authors Kai Yu, Ph.D., postdoctoral fellow in the Wang laboratory, and Jianfeng Chen, M.D., Ph.D., instructor of Genitourinary Medical Oncology.
“Traditional molecular subtyping often classifies bladder cancers as either luminal or basal, but our spatial analyses show that this binary view is incomplete,” Wang said. “Within a single patient’s tumor, luminal and basal-like programs can coexist in highly organized spatial patterns, and those patterns are closely tied to immune activity, lineage-specific treatment vulnerabilities, and how different tumor regions may respond to treatment.”
Novel gene therapy platform restores muscle function in models of Duchenne muscular dystrophy
Read the full release | Read the study in Nature Biomedical Engineering
A new treatment platform was able to deliver messenger RNA (mRNA) of the full-length DMD gene into preclinical models of Duchenne muscular dystrophy, successfully restoring the production of an important muscle protein, dystrophin, and dramatically improving muscle strength, endurance and function in vivo. The study was co-led by Betty Kim, M.D., Ph.D., professor of Neurosurgery and core member of the James P. Allison Institute™, and Wen Jiang, M.D., Ph.D., associate professor of CNS Radiation Oncology. The approach uses engineered extracellular vesicles (EVs) — natural nanoscale delivery particles — which offer distinct benefits over current viral-based gene therapies, including reduced side effects and the ability to transfer the entire DMD gene. The researchers engineered the EVs with special tags that directly target skeletal muscles after being injected into the bloodstream.
“Our new platform overcomes the limitations of current viral-based gene therapies, allowing for the delivery of full-length mRNA, restoring wild-type translation of dystrophin and significantly improving muscle function,” Kim said. “We are highly encouraged by these results, which provide a blueprint for mRNA-loaded EVs as a next-generation therapeutic strategy.”
Study identifies a new cause of age-related inflammation, suggesting promising treatment pathway
Read the full release | Read the study in Nature Aging
A new study has uncovered a previously unknown connection between nucleic acid structures called R-loops and age-related inflammation – or inflammaging – that could herald new intervention options for chronic inflammation and the subsequent health conditions. The study, led by Rugang Zhang, Ph.D., chair of Experimental Therapeutics, identified two specific proteins involved in the export of R-loops, a process that leads to inflammaging and related health issues. In preclinical models, the administration of KPT-330 (selinexor) prevented the R-loops from being exported and led to significant improvement in inflammation, liver damage, fat gain, muscle loss and overall lifespan.
“Chronic, widespread inflammation is a driving factor in many age-related diseases, including cancer, and our research has discovered one reason why this happens,” Zhang said. “Understanding the cause is the first step toward developing treatments. We saw encouraging results using a drug that has already been tested in humans, paving the way for potential clinical use to alleviate age-related conditions.”
Radiation therapy enhances immune environment in brain metastases, improving treatment response
Read the full release | Read the study in Clinical Cancer Research
A new study led by researchers at The University of Texas MD Anderson Cancer Center demonstrated that pre-operative radiation therapy for brain metastases not only targets tumor cells directly but also can activate immune pathways that can make tumors more receptive to immunotherapy. The study showed that radiation therapy is effective in both eliminating cells directly and also acting to reshape the surrounding immune landscape by recruiting and activating T cells, suggesting that radiation-immunotherapy combination strategies could potentially improve patient outcomes. The results also highlighted T cell receptor diversity in the tumor microenvironment as a potential prognostic biomarker for predicting treatment response. The research was co-led by Jason Huse, M.D., Ph.D., professor of Anatomic Pathology; Nuhad Ibrahim, M.D., professor of Breast Medical Oncology; and Alexandre Reuben, Ph.D., assistant professor of Thoracic/Head & Neck Medical Oncology.
“Brain metastases are highly complex, and effective treatment requires addressing both the tumor and its microenvironment in order to engage the immune system,” Huse said. “By enhancing T cell diversity and antigen presentation within tumors, radiation ultimately transforms the immunosuppressed tumor microenvironment into a more responsive one, providing a strong biological rationale for radiation-immunotherapy combination strategies to improve patient outcomes."
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