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An experimental drug doubled one-year survival in advanced pancreatic cancer

In a randomized trial of 233 patients with metastatic pancreatic cancer, twice as many who received the experimental drug elraglusib alongside standard chemotherapy were alive after one year — 44 percent, compared with 22 percent among patients who got chemotherapy alone. The drug, developed inside Northwestern University’s own labs, also cut the risk of death by 38 percent, according to the trial led by Dr. Devalingam Mahalingam of Northwestern Medicine.

Median survival reached 10.1 months for patients on elraglusib, against 7.2 months for those on chemotherapy alone. Among patients who lived long enough to actually benefit from treatment, the gap widened further: roughly 13 percent of the elraglusib group was alive at two years, compared with none in the chemotherapy-only arm.

How elraglusib targets pancreatic tumors differently than chemotherapy

Elraglusib works by blocking a protein called GSK-3 beta, which plays a role both in tumor growth and in suppressing the immune system’s response to cancer. That mechanism sets it apart from standard chemotherapy, which is designed to kill cancer cells directly rather than change the environment surrounding them. According to the Northwestern Medicine research team, patients who received elraglusib showed increases in cancer-fighting immune cells inside their tumors, early evidence that the drug helps re-engage an immune system that pancreatic tumors are unusually effective at suppressing.

The trial, published in Nature Medicine, also found that certain immune-related markers present in patients’ blood at the start of treatment were associated with longer survival among those who received elraglusib. Researchers describe those findings as preliminary, but they suggest the drug may work best in patients whose immune systems are already primed to respond — a distinction that could eventually help doctors decide who is likeliest to benefit before treatment even begins.

A trial built to test the drug against a genuinely hard cancer

Pancreatic cancer is the third leading cause of cancer death in the United States, according to the National Cancer Institute’s SEER cancer statistics program, which estimates 67,530 new diagnoses and 52,740 deaths from the disease in 2026 alone. Five-year relative survival across all stages sits at just 13.7 percent, a figure that reflects how often the cancer is caught only after it has already spread.

Against that backdrop, the phase 2 trial enrolled patients at 60 sites across six countries in North America and Europe, randomly assigning each to receive either standard chemotherapy or the same chemotherapy paired with elraglusib. Mahalingam, a professor of medicine in Feinberg’s division of hematology and oncology and associate director of clinical research at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, called the survival benefit encouraging in part because so few randomized trials in the disease have ever shown a benefit applicable to a broad patient population.

The patients who took part, in their families’ account

Donna Husar, 65, of Palos Heights, Illinois, said her late husband Matthew enrolled shortly after his diagnosis, on the advice of a relative who worked in oncology. He stayed in the trial for nearly two years, and the family credits the drug with buying them more time together. His daughter, Madeline Husar, 29, of Chicago, said the trial gave the family something to hold onto beyond fear. “When you read about pancreatic cancer online, it’s terrifying,” she said. “But knowing there was research and a trial gave us something positive to focus on instead of stressing about the worst-case scenarios.”

Maria Lepowsky, 75, of Madison, Wisconsin, described a similar experience with her late husband, Robert Brightman, who also remained in the trial for about two years. “My husband believed in helping future patients,” she said, adding that the drug appeared to improve his quality of life as well as extend it. For much of his treatment, she said, Brightman was able to ride a city bus to his Chicago clinic appointments on his own, a small independence that mattered to him.

The limits of a phase 2 result

Side effects were broadly consistent with standard chemotherapy but somewhat more frequent in patients who received elraglusib, including low white blood cell counts, fatigue, and temporary vision changes that resolved after treatment. Because the U.S. arm of the trial began more than five years before its results were published, most of the American patients who took part, including both Matthew Husar and Robert Brightman, have since died of their disease — a reminder that even a successful trial in pancreatic cancer runs on a timeline the disease itself rarely allows patients to outlast.

The research was funded by Actuate Therapeutics, the company developing elraglusib, and Mahalingam discloses both institutional research funding and consulting compensation from the company; Northwestern University also holds a financial interest in Actuate. None of that changes the trial’s randomized design, but it is the kind of detail that matters before treating a phase 2 result as settled. “These results will need to be confirmed in phase 3 trials,” Mahalingam said, “but observing survival benefit in such a difficult-to-treat cancer is encouraging.” He and his colleagues are now seeking funding and partnerships to run that larger, confirmatory trial.

This article was produced with the assistance of AI and reviewed by Morning Overview editors prior to publication.


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