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Large clinical trial finds targeted therapy helped patients live longer than chemotherapy

• An experimental targeted drug nearly doubled median survival for people with previously treated metastatic pancreatic cancer.

• The phase three clinical trial compared the daily pill with standard chemotherapy in nearly 500 patients worldwide.

• Researchers say the findings could lead to a new treatment option for patients with few effective alternatives.

Pancreatic cancer is one of the most difficult cancers to treat, especially after it has spread to other parts of the body and stopped responding to initial therapy. For many patients in that situation, treatment options are limited and survival has historically remained short.

Now, a large international clinical trial is offering new hope with an experimental targeted drug that significantly outperformed standard chemotherapy.

The medication, called daraxonrasib, works differently from traditional chemotherapy. Instead of attacking rapidly growing cells, it targets the RAS signaling pathway, which drives the growth of more than 90% of pancreatic cancers.

According to researchers, the study marks the first time a phase three trial has shown such a substantial survival benefit over chemotherapy for patients with previously treated metastatic pancreatic cancer.

For years weve made incremental gains in treating pancreatic cancer, researcher Dr. Zev Wainberg said in a news release.

Now, for the first time, we have demonstrated that targeted inhibition of RAS using an oral inhibitor is changing the landscape of this terrible disease. Seeing this magnitude of benefit in a randomized phase three study is very encouraging for all patients with advanced pancreatic cancer and is a paradigm shift in this deadly disease.

The study

The phase three trial enrolled 479 adults with metastatic pancreatic ductal adenocarcinoma whose cancer had continued growing after one previous course of chemotherapy.

Participants were randomly assigned to receive either a 300-milligram daily dose of daraxonrasib or one of several commonly used chemotherapy treatments selected by their physician.

Researchers followed patients to compare how long they lived overall, how long their cancer remained under control, how their tumors responded to treatment, and how the therapies affected quality of life.

They also monitored side effects throughout the study. Because the trial randomly assigned participants to one treatment or the other, researchers were able to directly compare outcomes between the two groups.

The results

The results were striking. Patients who received daraxonrasib had a median overall survival of 13.2 months, compared with 6.7 months for those treated with chemotherapy.

The drug also delayed disease progression for longer and produced higher tumor response rates.

Patients taking daraxonrasib generally maintained their quality of life for a longer period, although side effects were common. The most frequently reported side effects included rash, diarrhea, and nausea, while serious treatment-related side effects occurred less often than with chemotherapy.

For consumers, these findings represent an encouraging step forward rather than an immediate change in care. Daraxonrasib remains an experimental treatment, but the results suggest it could become a valuable option for patients whose metastatic pancreatic cancer has progressed after initial chemotherapy.

While additional regulatory review is still needed before the drug becomes widely available, the study offers new optimism for a disease that has long had few effective treatment options.

Were this drug to be approved by the FDA, it would mark a dramatic shift in how pancreatic cancer is treated, researcher Brian Wolpin said in a news release.