Experimental Pill Nearly Doubles Survival Rates

• A new medication performed remarkably well in a phase 3 clinical trial for treating pancreatic cancer.
• The drug daraxonrasib reduced the risk of death by nearly half, as well as shrank tumors in people who were given the drug.
• Pancreatic cancer has the highest mortality rate of all major cancers, and early detection is the key to treating the disease.

A new type of medication is showing promise in the treatment of pancreatic cancer, one of the deadliest forms of cancer.

In a phase 3 clinical trial, the drug daraxonrasib significantly improved survival rates of participants with pancreatic cancer who had previously been treated with chemotherapy.

The researchers found that daraxonrasib reduced the overall risk of death by 60% compared with people with advanced pancreatic cancer who were treated with chemotherapy.

The medication also helped shrink or eliminate tumors among participants in the trial.

Officials at Revolution Medicines, the manufacturer of daraxonrasib, said the clinical trial results are a major breakthrough in pancreatic cancer treatment.

“These results represent a potentially transformative advance for patients and underscore daraxonrasib’s potential to redefine the treatment landscape,” said Mark Goldsmith, MD, the chief executive officer and chairman of Revolution Medicines, in a statement.

Participants were given doses of between 10 milligrams (mg) and 400 mg of daraxonrasib orally once a day, with 300 mg selected as the phase 3 dose.

The researchers focused on 168 participants who had been previously treated with chemotherapy.

The researchers reported that participants using daraxonrasib had an average survival rate of 13 months from diagnosis to death compared to 6 months for participants treated with standard chemotherapy.

In people with a known RAS mutation called G12, tumors remained ⁠under control for a median of about 7 months among participants on daraxonrasib compared to about 3 months for those treated with chemotherapy.

The percentage of patients whose cancer shrank or disappeared was about 33% in patients with the G12 mutation compared to about 12% for people given chemotherapy. Overall, about 31% of trial participants saw their tumors shrink or disappear compared with 11% who received chemotherapy.

Experts not involved in the trial are very encouraged by the findings.

“This is an extraordinarily hopeful moment for the pancreatic cancer field,” said Diane Simeone, MD, the director of the UC San Diego Moores Cancer Center, as well as the founder and chief scientific advisor of the Pancreatic Cancer Early Detection Consortium (PRECEDE), in a statement shared with Healthline.

“Pancreatic cancer patients have had far too few effective treatment options,” she added. “The progress around daraxonrasib shows what is possible when long-term scientific investment, academic discovery, and industry innovation come together,” Simeone continued.

Christina Annunziata, MD, senior vice president of Extramural Discovery Science at the American Cancer Society (ACS), was also buoyed by the research.

“These results are extremely promising since this drug doubled overall survival for patients with relapsed pancreatic cancer,” she told Healthline.

Daraxonrasib is the first in a new class of drugs called RAS(ON) inhibitors that target variants of the RAS gene that drive cancer growth.

Side effects were reported in 96% of participants who received doses of 300 mg or less. Those included:

Side effects were grade 3 or higher in about a third of participants.

Annunziata, however, noted that “the side effects were lower compared to standard chemotherapy typically used in this second-line treatment setting.”

However, she had concerns about access to the new medication. “The only concern will be the availability of this drug to people who need it. This will depend on the cost and level of insurance coverage,” Annunziata said.

The American Cancer Society (ACS) reports that 68,000 Americans will be diagnosed with pancreatic cancer this year, and roughly 53,000 will die from the disease.

Pancreatic cancer affects the pancreas, an organ that plays an essential role in digestion by producing enzymes that the body needs to digest fats, carbohydrates, and proteins. The pancreas is located behind the stomach, which can make detection and diagnosis of cancer difficult.

SEER data from the National Cancer Institute (NCI) show that around 3% of people whose cancer has spread from the pancreas to distant organs have a 5-year survival rate. About 80% of patients are diagnosed in the advanced or metastatic stage.

The average lifetime risk for pancreatic cancer is about 1 in 56 in males and about 1 in 60 in females.

Pancreatic cancer often doesn’t produce symptoms until it reaches an advanced stage. Symptoms at later stages may include:

• loss of appetite
• unintentional weight loss
• abdominal pain that may radiate to the back
• fatigue, weakness
• jaundice
• nausea and vomiting

The American Cancer Society (ACS) reports that there are risk factors for pancreatic cancer that you can lower by adopting certain lifestyle behaviors. These include:

• tobacco use
• excess body weight
• exposure to workplace chemicals

They also note there are risk factors that cannot be modified.

One of them is age. The ACS states that almost all pancreatic cancer patients are older than 45. About two-thirds are at least 65 years old. The average age at diagnosis is 70.

Males are slightly more likely than females to develop pancreatic cancer, partly due to higher smoking rates and other factors.

African-Americans are also slightly more likely to be diagnosed with pancreatic cancer than other groups. The ACS states this may be due to factors such as diabetes, smoking, and excess body weight.

There are various treatments that are used to fight pancreatic cancer, including:

• surgery in which portions of the pancreas are removed
• radiation therapy
• chemotherapy
• targeted therapy
• immunotherapy

“New treatments can help patients live longer, but we have the greatest opportunity to change the trajectory of this disease when pancreatic cancer is found earlier, before it has spread,” Simeone said.

Annunziata listed various reasons why pancreatic cancer is so difficult to treat:

• it’s often discovered in its later stages
• dense connective tissue around the cancer decreases the effectiveness of chemotherapy and immunotherapy
• pancreatic cancer cells have gene mutations that make them grow quickly and evade chemotherapy

Early detection of pancreatic cancer can help improve survival rates.

There are a variety of tests used for pancreatic cancer screening, such as:

• CT or MRI scans of the pancreas
• an endoscopic ultrasound
• a biopsy of tissue from the pancreas
• blood tests to check for biomarkers

Annunziata said what makes screening for pancreatic cancer problematic is the lack of early stage symptoms.

“There is currently no screening method for pancreatic cancer in the general population,” she said. “People with a strong family history of pancreatic cancer, or known hereditary syndromes like BRCA [breast cancer gene] or Lynch [syndrome], should talk with their doctor about regular screening with endoscopic ultrasound or MRI.”

Annunziata said the best defense is to adopt a lifestyle that decreases the possibility of pancreatic cancer developing.

“People can reduce their risk by avoiding factors known to be associated with pancreatic cancer: don’t smoke or use tobacco products, maintain a healthy weight and diet, pursue regular exercise, and limit alcohol intake,” she said.