If you have the KRAS G12D mutation, you already know these drugs are being tested in other cancers. Two big announcements today tell me we’re that much closer to them being tested in appendix cancer too.
What KRAS G12D actually is
KRAS is a gene. In a healthy cell, it works like a switch that turns on to tell the cell to grow, then turns back off. When KRAS is mutated, the switch can get stuck on, and the cell keeps getting a grow signal it can’t shut off. G12D is one specific version of that stuck switch. It’s also the most common KRAS change in mucinous appendix tumors, which is why it’s the one your care team watches for.
Two ways to hit the same target
For a long time, no one could build a drug that hit this switch. That’s changed, and there are now two ways to do it. The first is an inhibitor. It sits on the stuck switch so it can’t send its grow signal. The second is a degrader. Instead of just blocking the switch, it tags the switch so the cell clears it out completely. Both go after the same mutation, just in different ways.
The drugs to watch
A few of these are getting the most attention right now. Two are inhibitors. One is VS-7375, from Verastem. Another is zoldonrasib, from Revolution Medicines. The third is a degrader called setidegrasib, from Astellas. Those three are among the furthest along.
They aren’t the only ones. Other companies are testing their own G12D drugs, some inhibitors and some degraders, with more in early trials. Here’s the wider list worth keeping an eye on.
What these drugs are doing in other cancers
In pancreatic and lung cancer, these drugs are working. In early trials, tumors have shrunk, and some people who had run out of other options have responded. The results were strong enough that two of the three have already moved into large, late-stage trials, and one earned an FDA Fast Track designation, which is the FDA’s way of saying a drug matters enough to push it along faster. One of them had its early results published in the New England Journal of Medicine, one of the most respected journals in medicine. It’s early, but it’s real.
Where KRAS G12D appendix cancer research stands today
So far, all of this has happened in other cancers, mostly pancreatic and lung. For KRAS G12D appendix cancer, the work is still in the lab. Researchers have tested these same ideas in appendix cancer organoids, which are tiny tumors grown from real patient tissue, and in mice. That early work looks promising, but it’s not the same as a person with appendix cancer taking one of these drugs and getting better. Don’t change anything about your treatment based on this. These drugs are not approved or available for appendix cancer yet.
Why this still matters for you
So many appendix tumors carry the exact same KRAS G12D mutation these drugs are built to hit. When a drug keeps working in one cancer, researchers start asking which other cancers it could help. Appendix cancer is near the top of that list, because so many of us carry G12D. Every new result makes the case stronger for testing these drugs in us too.
One more reason I’m hopeful
We aren’t watching this from the outside. I’ve started talking with people inside this work about what it would take to bring appendix cancer into these trials, and those conversations are happening right now. I can’t share details yet, and nothing is promised. But our community is being represented where these decisions get made, and I’ll tell you more the moment I can.
What you can do now
There are two things worth doing right now. First, know your own mutation status. Ask your oncologist whether your tumor has had molecular or genetic testing, and whether KRAS showed up. If it did, ask which exact change it is. Knowing whether you carry G12D puts you a step ahead if a matched trial ever opens. Second, get counted. Appendix cancer is rare, and researchers often can’t find enough patients to study or to fill a trial. The Patient-Led Global Appendix Cancer Registry exists to fix that. When you add your information, you help us show researchers there’s a real group of patients ready and waiting.
You can join the registry here. It takes a few minutes, it’s open to patients and caregivers anywhere in the world, and every entry makes our community easier to find and harder to overlook.
None of this is a promise yet. But after years with no targeted options, having real drugs aimed right at our mutation is something to hold onto. And if you can chip in to help us move faster, you can support Appendicure here. Every dollar goes toward education, research, and finding the patients who still need to be found.
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