Experimental drug promises new hope for deadly pancreatic cancerExBulletin

This undated microscopic image provided by USC via the NIH shows pancreatic cancer cells with blue nuclei growing as spheres surrounded by a red membrane.

Min Yu/AP/USC (via NIH)

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Min Yu/AP/USC (via NIH)

WASHINGTON — A new pill helped extend the lives of patients with advanced pancreatic cancer, researchers reported Sunday, raising hopes for a long-needed better treatment for one of the deadliest types of cancer.

“It doesn’t cure cancer, but it’s a huge step forward,” said study leader Dr. Zev Weinberg of the University of California, Los Angeles.

The drug, called daraxone lasib, blocks a mutated protein that promotes tumor growth in more than 90% of pancreatic cancer cases. This protein has been untargeted for treatment for decades.

In a study that randomly assigned 500 patients whose metastatic or metastatic cancer had stopped responding to previous treatment to an drug or additional chemotherapy, the daily pill reduced severe side effects and nearly doubled survival. The study results were published in the New England Journal of Medicine and presented Sunday at the American Society of Clinical Oncology meeting in Chicago.

Median survival for patients who received dalaxone lasib was 13.2 months, compared with 6.7 months for patients who received chemotherapy. Although this may seem like a small improvement, Weinberg said this is the first drug to show a significant benefit over chemotherapy.

“I’ve been treating pancreatic cancer for 16 years, and when I saw the study results for the first time, I actually started crying,” Dr. Rachna Shroff of the University of Arizona Cancer Center, who was not involved in the study, told the ASCO conference. She was struck by how “patients continued with this treatment because it was providing lasting and meaningful benefits.”

Although the effects of the pills eventually wear off, recipients used the pills significantly longer than the comparison group who continued chemotherapy, reporting less pain and improved quality of life as their tumors shrank. After analyzing the data, many people were still using the drug, Weinberg said, meaning the difference in survival rates could widen if researchers continued to follow them.

Dr. Brian Wolpin of the Dana-Farber Cancer Institute announced the study results on Sunday. He said the drug should become a “new standard of care” for previously treated metastatic pancreatic cancer, adding that researchers would also consider its use in earlier stages of the disease, including to see if shrinking tumors would allow more patients to undergo surgery.

The side effects most likely to affect the use of the tablets were rash and mouth sores, which can be serious.

The manufacturer, Revolution Medicine, is funding the study, and the Food and Drug Administration will expedite its review of the drug. Meanwhile, government agencies are granting so-called “expanded access” to the drug to patients who meet certain criteria. The drug gained public attention when former U.S. Sen. Ben Sasse explained on “60 Minutes” how his pain decreased while taking the drug. Oncologists have been inundated with requests since the special access program was launched.

Pancreatic cancer is one of the deadliest cancers because it is difficult to detect before it spreads to other organs. The American Cancer Society estimates that about 67,000 new cases will be diagnosed in the United States this year and more than 52,000 will die from the disease. The 5-year overall survival rate is 13%.

Unlike other cancers that have benefited from a variety of chemotherapy options, pancreatic cancer is more difficult to tackle.

Cancer experts not involved in the new study expressed optimism that dozens of drugs are in development and that this could be a turning point in the search for new options.

The new drug targets mutations in the RAS gene family, which normally controls cell growth. So-called KRAS mutations are particularly important in promoting the development of pancreatic cancer. However, because the mutated protein has a structure that makes it difficult for drugs to attach to it, this cancer-promoting factor was long thought to be “undruggable.”

Revolution Medicines’ drug essentially uses molecular glue to bind to multiple KRAS subtypes. Weinberg said the researchers will next investigate whether the drug works better at identifying these subtypes.

Dr. Andrew Kaveler of Fred Hutchinson Cancer Center, who was not involved in the study, said the drug would change pancreatic cancer treatment.

“This works much differently,” he said.

Weinberg said other drugs in development also target specific KRAS subtypes. Other approaches in the early stages of testing include vaccines designed to prevent recurrence after pancreatic cancer surgery by teaching the immune system to recognize mutated proteins.