ADRIAN FLORIDO, HOST:
The Food and Drug Administration yesterday approved a breakthrough drug, a pill, as a first-of-its-kind treatment for metastatic pancreatic cancer. It's one of the deadliest cancers and notoriously hard to treat. In clinical trials, this new drug roughly doubled how long patients survived. NPR's Yuki Noguchi joins us to discuss why this drug holds such promise. Hi, Yuki.
YUKI NOGUCHI, BYLINE: Hey.
FLORIDO: Tell us more about this new drug.
NOGUCHI: Well, it's a new class of drug, and it's also something of an engineering feat. It's very clever. But just to back up, one reason pancreatic cancer is so deadly is it exists deep in our abdomens, and it's hard to diagnose. Often, by the time it's caught, it's spread. And at a molecular level, it's also really hard to treat because there's no obvious pocket or binding site for a drug to attack. The key target is a protein in this case, called KRAS, known to drive nearly all pancreatic cancers, and the drug needs to attach to that protein to work.
But Mark Goldsmith, CEO of Revolution Medicines, the company making the drug, says in this case of pancreatic cancer, that's like trying to grab onto a flat surface. And so what this new medicine does, which is known generically as daraxonrasib, is it creates its own binding site, kind of like putting a handhold on that flat surface. So Goldsmith describes it this way.
MARK GOLDSMITH: The drug first binds to another protein in the cell and then make a sandwich with the target. And so the drug binding pocket is not redefined on the target, but it is basically created by this sandwich or glue or what we call a tricomplex binding mechanism.
NOGUCHI: Which is really cool - in clinical trials, Rasonque, which is its brand name, nearly doubled survival to 13.2 months compared to 6.7 months for patients on just chemotherapy. And that was enough to prompt the FDA to expand access to the drug months before it granted approval.
FLORIDO: So this drug's already been in use. What do doctors and patients say about It?
NOGUCHI: Well, it's a rare, new lifeline, and it works better than chemotherapy and has fewer and generally milder side effects, the most common being a skin rash. And that's really critical. Dr. Pashtoon Kasi is a medical oncologist at City of Hope in Orange County, California, and is principal investigator of the expanded access trials. He says this drug worked for just about every patient, and that's very uncommon. Plus, it's just a pill not an infusion and so much easier to tolerate than chemo too.
PASHTOON KASI: One thing that the trials and the news and the numbers don't do justice is the tangible impact that we're seeing in the quality of life for patients. I have patients that they stopped taking their pain meds. You know, that was not something that we saw before with chemotherapy.
NOGUCHI: So that's something actually patients have told me too.
FLORIDO: Are there implications for people with other kinds of cancers, Yuki?
NOGUCHI: Yeah, there are. I mean, the same genetic mechanism that drives almost all these pancreatic cancers also drives some lung and colorectal cancers, a smaller percentage but still significant. So clinical trials are underway to see if patients might benefit from this, as well.
FLORIDO: How much is this drug going to cost?
NOGUCHI: Oh, it's expensive, you know, like most cancer drugs. Revolution Medicines says the wholesale price is nearly $40,000 a month.
FLORIDO: Yowzers (ph).
NOGUCHI: But how much patients actually pay will depend on the particulars of their insurance situation, of course.
FLORIDO: That's NPR's Yuki Noguchi. Thanks.
NOGUCHI: Thanks. Transcript provided by NPR, Copyright NPR.
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