New drugs raise hopes of pancreatic cancer breakthrough

For decades, pancreatic cancer has stood as one of the most formidable and unbeatable foes in modern oncology, a deadly diagnosis with almost no major treatment advances to offer patients and devastatingly low survival rates. But after 40 years of stagnant progress, a wave of cutting-edge new therapies from researchers across the globe have delivered unprecedented, promising results that are being hailed as a turning point in the fight against this aggressive disease.

Pancreatic cancer has long had a grim reputation: global health data shows only around 10 percent of diagnosed patients survive beyond the five-year mark after their initial diagnosis. Worse, incidence rates have been climbing steadily across the world, with particularly sharp growth among younger adult populations. Projections indicate that within the next decade, pancreatic cancer will overtake all other cancers except lung cancer to become the second leading cause of cancer-related death in developed nations. Until recently, this growing public health crisis had seen almost no medical innovation, according to Patrick Mehlen, a senior researcher at France’s Leon Berard Cancer Centre. Mehlen told AFP that for nearly half a century, the field saw virtually no meaningful progress. Over the past decade, however, increased research funding and growing public attention have finally shifted the landscape, turning what was once a dead end into a space of rapid innovation.

While a universal cure remains out of reach for most patients, these new therapies are already delivering tangible, life-extending benefits that were unthinkable just a generation ago. The most high-profile breakthrough came last week from American biopharmaceutical company Revolution Medicines, which released positive late-stage trial data for its experimental targeted therapy daraxonrasib. The drug works by targeting the KRAS protein, a genetic mutation long known to drive uncontrolled tumor growth in a large share of pancreatic cancer cases.

In the company’s phase three trial, half of all patients receiving the daily pill survived more than 13 months after starting treatment — double the survival time of the control group that received standard chemotherapy. While doubling survival to 13 months may seem like a modest gain to outsiders, for a cancer that typically claims patients within months of diagnosis, this result is unprecedented.

One high-profile patient, former U.S. Senator Ben Sasse of Nebraska, has shared his firsthand experience of the drug’s impact after being diagnosed with late-stage, metastatic pancreatic cancer in late 2023. Sasse, 54, told the New York Times that when he received his diagnosis, doctors gave him just three to four months of life. After starting treatment with daraxonrasib, he said he is far healthier and more active than he was just before Christmas last year. He did acknowledge the drug comes with harsh side effects, noting it is “a nasty drug” that caused severe skin reactions including peeling and bloody skin on his face. Revolution Medicines announced it plans to submit a formal application for U.S. regulatory approval in the near future, and full detailed trial results will be presented at the annual American Society of Clinical Oncology (ASCO) conference in Chicago next month.

Daraxonrasib is not the only promising advance to emerge in recent weeks. Earlier this week, a separate research team led by Mehlen published early-stage trial results in the journal Nature for a novel therapy that takes a completely different approach to treating the disease. Unlike traditional treatments that aim to kill tumor cells directly, this new therapy targets the ability of cancer cells to develop resistance to existing treatments, including chemotherapy.

The experimental drug, called the NP137 antibody, was tested in a phase 1b trial on 43 patients with locally advanced pancreatic cancer that had not yet spread to distant organs. All patients received the antibody alongside standard chemotherapy. Compared to historical survival rates for similar patients, study participants gained an average of several additional months of life. “We’re giving people an average of six months more — which is significant for this disease,” Mehlen explained of the results. His team plans to launch a larger, controlled follow-up trial later this year to confirm these early findings, and Mehlen said he eventually hopes the antibody will be able to boost the effectiveness of not just chemotherapy, but also new targeted therapies like daraxonrasib.

The third major advance announced over the weekend brings mRNA technology, which gained global fame for its role in COVID-19 vaccines, into the fight against pancreatic cancer. The experimental therapeutic cancer vaccine was co-developed by pharmaceutical firms BioNTech and Genentech, and it uses mRNA to train the body’s immune system to recognize and attack pancreatic cancer cells.

Early phase one trial results for the vaccine show that among 16 treated patients with pre-existing pancreatic cancer, the vaccine triggered a targeted immune response against cancer cells in eight participants. Seven of those eight patients who responded to the vaccine were still alive six years after receiving treatment. By comparison, just two of the eight patients whose immune systems did not respond to the vaccine survived for the same six-year period. Researchers note that phase one trials are primarily designed to test treatment safety rather than confirm effectiveness, so large-scale follow-up studies will be needed to verify these encouraging early results.

Taken together, the string of positive trial announcements marks the most significant leap forward for pancreatic cancer treatment in decades, turning a once untreatable disease into a condition that researchers believe can be managed with more effective therapies in the coming years.