New Drug Offers Hope for Pancreatic Cancer Patients in Trials

A promising new drug, daraxonrasib, is currently being tested in clinical trials at various locations, including Penn Medicine’s Abramson Cancer Center, offering hope to patients suffering from stage 4 pancreatic cancer. One such patient, Irene Blair, was given an estimated six to eight months to live in June after her cancer rapidly advanced. This drug, a KRAS inhibitor, aims to block a protein associated with this particularly aggressive cancer type, which has a five-year survival rate of only 13%.

Breakthrough Treatment in Clinical Trials

Blair, a 59-year-old grandmother from Newark, Delaware, has joined a phase 3 trial for daraxonrasib, which is part of a class of drugs that researchers have long sought to develop. In recent months, the federal government has expedited the review process for this drug, produced by Revolution Medicines, Inc., due to encouraging preliminary results from trials. In a phase 1 trial involving 38 patients, the drug appeared to double the median survival time for at least half of the participants, extending it from approximately seven months to 15.6 months.

Mark O’Hara, Blair’s oncologist and a leader in KRAS inhibitor research at Penn, noted, “In pancreatic cancer, for too long, we haven’t had effective therapies beyond just chemotherapy.” Blair began her treatment in July, and by August, she reported a significant reduction in pain associated with her cancer. Scans in October revealed that her tumors were stable or decreasing, and her most recent scan in December confirmed that her cancer had not progressed.

Human Impact and Ongoing Research

Before starting the new therapy, Blair experienced severe side effects from traditional chemotherapy, including a loss of 35 pounds and debilitating weakness. Now, she is eager to make the most of her time, having retired from her real estate job in May and planning trips to visit family in California and Florida. “You just wonder, ‘Will I be here next year?’” she expressed, highlighting the emotional toll of her diagnosis.

The development of drugs targeting the KRAS protein has been a long pursuit in cancer research, dating back to its discovery in 1982. Mutations in this protein act like a gas pedal for cancer growth, and they are found in approximately 25% of human cancers, particularly aggressive forms such as pancreatic, lung, and colon cancers. In 2021, the FDA approved the first drugs capable of blocking KRAS for lung cancer, paving the way for more research.

Currently, daraxonrasib is one of the first KRAS inhibitors tested for pancreatic cancer, where nearly 90% of cases involve these mutations. This drug, also known as a “pan-RAS inhibitor,” targets not only KRAS but also related proteins HRAS and NRAS. In the phase 1 trial, over 90% of the 83 patients saw their cancer stall during treatment, with about 30% experiencing tumor shrinkage.

The treatment is administered in the form of three daily pills taken at home, with manageable side effects. The most common is a facial rash, experienced by 91% of trial participants, with 8% reporting severe cases. Other side effects include diarrhea, nausea, vomiting, and mouth sores, but O’Hara maintains that these symptoms are often manageable and lead to a better quality of life compared to chemotherapy.

As research progresses, the medical community remains hopeful that therapies like daraxonrasib will continue to provide effective options for patients battling pancreatic cancer, a disease that has long been associated with bleak prognoses. Blair’s journey is a testament to the potential of new treatments in transforming patient outcomes.