A clinical trial shows a pill almost doubled survival for pancreatic cancer patients. Now what?

A clinical trial shows a pill almost doubled survival for pancreatic cancer patients. Now what?

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This undated microscope image from USC via the NIH shows pancreatic cancer cells, nuclei in blue, growing as a sphere encased in membranes, red. (AP)

If Your Time is short

An experimental pill to treat metastatic pancreatic cancer almost doubled patients’ survival times compared with chemotherapy.

The drug isn’t Food and Drug Administration approved, so it’s not readily available for all patients. Some eligible patients might be able to get the medication before it is FDA approved using a special program for people with life-threatening diseases.

Doctors who treat pancreatic cancer said they expect full FDA approval for the pill within months.

Good news is rare when it comes to pancreatic cancer.

Early detection is difficult. Post-diagnosis survival times are relatively  short . Existing treatments are mostly available in the disease’s early stages. Even though just 3.2% of newly diagnosed cancers are pancreatic, it is the nation’s third-leading cause of cancer deaths.

So when Dr. Rachna Shroff, who has been treating pancreatic cancer patients for 16 years, saw promising findings from a recently published clinical trial, she felt what she described as a monumental shift.

"The data brought tears to my eyes," said Shroff, the University of Arizona Cancer Center’s chief of hematology and oncology.

The study showed that an experimental pill from the company Revolution Medicines almost doubled metastatic pancreatic cancer patients’ survival times compared with chemotherapy.

What happens next will determine whether and when patients may be able to access the drug, daraxonrasib (de-RAK-son-ra-sib). Here’s what these findings could mean for patients in the near future.

Q: What did the study find?

A: The study , a phase 3 clinical trial, involved 500 metastatic, or stage four, pancreatic cancer patients who had already received a first-line treatment such as chemotherapy.

After the initial treatment stopped working or proved unsuccessful, the patients were randomly assigned to receive either the daraxonrasib pill once daily, or a second-line chemotherapy treatment.

Among patients who took daraxonrasib, the median overall survival time was 13.2 months — almost twice chemotherapy’s median overall survival time of 6.7 months.

Shroff said the study showed a trifecta of promising results: It extended survival times, was linked to increased tumor shrinkage and patients reported it positively changed their quality of life.

"It really checks all the boxes," Shroff said.

Chemotherapy is administered to a cancer patient via intravenous drip in Durham, N.C. on Sept. 5, 2013. (AP)

Q: Who is a good candidate for daraxonrasib?

A: Right now, the drug is an option for pancreatic cancer patients who have already received some kind of treatment. But researchers are also studying its use as a potential first-line treatment for newly diagnosed people.

The genetic factors of a patient’s cancer might also affect whether daraxonrasib is a suitable treatment option.

The National Comprehensive Cancer Network’s guidelines urge people to get tested for genetic mutations specific to their pancreatic cancer. This helps doctors determine the best treatment options and clinical trial eligibility as treatments are tested and clinical guidelines evolve, Shroff said.

"There are so many drugs beyond daraxonrasib" being tested, Shroff said, including those that target the cancer’s specific genetic mutations. Plus, with pancreatic cancers that have certain genetic mutations, there might be existing FDA-approved drugs that are the best treatment option.

Q: Is daraxonrasib available for patients now?

A: The drug isn’t Food and Drug Administration approved, so it’s not readily available for all patients. But phase 3 clinical trials are one of the final steps before the FDA reviews and potentially approves a drug.

That said, the FDA on April 30 granted Revolution Medicines’ request for expanded treatment access, sometimes called "compassionate use."

Under this program, patients who have life-threatening diseases can get investigational drugs if they are ineligible for — or cannot access — clinical trials and have no other comparable treatments available to them. Eligibility also requires patients to be over 18 years old and have previously treated metastatic pancreatic cancer.

A doctor who believes a patient would be a good candidate for "compassionate use" of daraxonrasib can submit paperwork to Revolution Medicines, the FDA and an ethics committee or institutional review board requesting approval. Sometimes patients must cover the cost of institutional review boards evaluations.

It’s unclear how many patients are seeking access to daraxonrasib through the "compassionate use" program. Revolution has not said how many people are expected to receive daraxonrasib via expan…