Promising treatment results for blood cancers, colorectal cancer and brain metastases Novel insights into early pancreatic cancer cell progression A new therapeutic target for patients with high blood pressure HOUSTON, JUNE 26, 2025 ― The University of Texas MD Anderson Cancer Center’s Research Highlights showcases the latest breakthroughs in cancer care, research and prevention. These advances are made possible through seamless collaboration between MD Anderson’s world-leading clinicians and scientists, bringing discoveries from the lab to the clinic and back.
Triplet combinations improve outcomes for elderly patients with IDH-mutant AML
Read summary | Read in Journal of Clinical Oncology
New targeted therapies have improved treatment options for elderly patients with IDH-mutant acute myeloid leukemia (AML) who are not eligible for intensive chemotherapy, with two double therapy regimens approved as frontline therapies. However, most patients either do not benefit or will experience a relapse. Therefore, researchers led by Courtney DiNardo, M.D., evaluated new triplet regimens in 60 newly diagnosed, elderly patients with IDH-mutated AML who were ineligible for chemotherapy, including:
Azacitidine, venetoclax and ivosidenib for patients with IDH1 mutations Oral decitabine, venetoclax and ivosidenib for patients with IDH1 mutations Oral decitabine, venetoclax and enasidenib for patients with IDH2 mutations The regimens were well tolerated with similar safety profiles to previous doublet regimens. The therapies achieved complete remissions in 92% of patients and an overall response rate of 95%. After more than two years of follow-up, nearly 70% of patients were still alive, and relapse rates were lower than anticipated, especially in patients who had not received prior therapy. Researchers continue to examine patient outcomes, and this study has informed a large Phase III randomized trial that is currently enrolling patients.
Presurgical radiation therapy may offer alternative approach to treating brain metastases
Read summary | Read in JAMA Oncology
Many patients with brain metastases receive stereotactic radiation therapy (SRT) – a targeted high dose of radiation – after surgery to remove the metastases. Studies have suggested that pre-surgical SRT may be comparable to standard post-surgical SRT, but little is known about both methods from clinical trials. To address this, researchers led by Debra Nana Yeboa, M.D., conducted a Phase III clinical trial examining the logistics and short-term safety of both pre- and post-surgical SRT in 103 patients with resectable brain metastases. Compared to post-surgical SRT, preliminary results show that pre-surgical SRT resulted in similar 30-day post-surgical toxicity outcomes with potentially shorter time between surgery and radiation. These findings suggest that giving SRT before surgery is a promising, potentially safe strategy for patients with brain metastases.
Heated chemotherapy improves overall survival in some patients with colorectal cancer
Read summary | Read in the Journal of the American College of Surgeons
Some patients with advanced colorectal cancer develop metastases in the lining of the abdominal cavity, known as peritoneal metastases. Cytoreductive surgery (CRS) can help remove most tumors, and it is sometimes combined with heated chemotherapy delivered to the abdominal cavity – a treatment approach called hyperthermic intraperitoneal chemotherapy (HIPEC) – to eliminate remaining cancer cells. In a retrospective study, researchers led by Michael White, M.D., and Paula Smith, M.D., examined the benefits of HIPEC in 147 patients with colorectal cancer and peritoneal metastases who received CRS from 2008 to 2023. They compared overall survival (OS) and peritoneal disease-free survival (PDFS) in patients treated with CRS alone or with HIPEC. The researchers also studied each patient’s peritoneal cancer index (PCI), a scoring system to assess the extent of cancer spread. Researchers found that patients with PCI of 11 or higher had significantly improved OS with CRS-HIPEC compared to those who received CRS alone. Conversely, patients with lower PCI scores did not have any added benefit. These findings suggest adding HIPEC to surgery when treating peritoneal metastases benefits certain patients with colorectal cancer, allowing doctors to forgo HIPEC for others.
Researchers identify driver of nervous system overactivity in hypertension
Read summary | Read in Circulation Research
The hypothalamus is an area of the brain that controls the sympathetic nervous system – which regulates the fight or flight response – by increasing heart rate and tightening blood vessels. When this system is overactive, it leads to high blood pressure (hypertension), increasing the risk of cardiovascular and brain diseases. Normally, the calcineurin protein in the hypothalamus regulates this process by calming down specific brain signals. However, in lab models of hypertension, researchers led by Hui-Lin Pan, M.D., Ph.D., identified another protein, RCAN1, that sticks to calcineurin and blocks it from working properly, leading to overactivity. The researchers injected a small piece of protein to stop RCAN1, which lowered sympathetic nerve activity and reduced blood pressure. These findings suggest the promising potential of targeting the RCAN1-calcineurin interaction as a therapeutic strategy for patients with hypertension.
Injectable immunotherapy yields high remission rates in patients with B-cell ALL
Read summary | Read in The Lancet Haematology
Some patients with relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (B-ALL) are unable to receive chemotherapy and have limited alternative treatment options. Blinatumomab is a type of immunotherapy that simultaneously engages T cells and leukemia cells to eliminate the cancer cells. The treatment currently is approved as a continuous infusion over 28 days, though a subcutaneous injection would offer a simpler administration. In an international Phase I/II trial led by Elias Jabbour, M.D., researchers evaluated two injectable doses of blinatumomab in 88 adult patients with relapsed or treatment-resistant B-ALL. Over 75% of patients experienced remission within two cycles using either dose. The most common serious side effects included low white blood cells, immune-related reactions and neurological issues, though no deaths linked to treatment were reported. Based on both safety and effectiveness observed in the trial, the lower dose was recommended for future studies, and researchers continue to evaluate the duration of responses.
Biomarker in pancreatic cysts could predict risk of cancer progression
Read summary | Read in Clinical Cancer Research
Pancreatic cysts are fluid-filled growths that appear on or in the pancreas and have the potential to develop into pancreatic cancer. Currently, few tools are available to reliably predict which cysts are likely to progress. To identify predictive biomarkers, researchers led by Johannes Fahrmann, Ph.D., used advanced techniques to study the metabolites in cystic fluid and to evaluate the spatial distribution of key genes within the most common pancreatic cysts – intraductal papillary mucinous neoplasms (IPMNs) – a known pancreatic cancer precursor. The study included 125 patients with IPMN. The researchers found that IPMNs nearing transformation into cancer secrete more acetylated polyamines, which are compounds highly associated with enhanced cell growth and proliferation. These results suggest that elevated levels of acetylated polyamines in cystic fluid may help identify patients with high-risk IPMN to better guide their clinical management.
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