If you live with pseudomyxoma peritonei, or love someone who does, you already know the central strangeness of it. The pathology reports use words like “low grade.” The tumors do not divide quickly. And yet the disease keeps producing mucin, year after year, surgery after surgery, until the abdomen cannot hold any more.
This is the paradox that has shaped how patients describe PMP for decades. It is not aggressive in the way most cancers are aggressive. It is relentless in a different way. It secretes.
A new study from Bina Therapeutics, published in April 2026, takes that paradox seriously and asks a question that has not been asked clearly before: if PMP is not really a growth disease, what kind of disease is it? Their answer reframes the biology in a way that has real implications for how we might treat it.
The work behind the work
Before getting into the science, it matters to know who is doing it and why.
The lead researcher, Ricki Dhal, is not a stranger to this disease in the way most scientists are strangers to the things they study. His wife, Bina, has appendix cancer and PMP. The company is named for her. Every analysis, every line of code, every late night spent reanalyzing single-cell datasets is part of a husband trying to save his wife’s life. That is the engine driving this work.
When you read the study, you can feel that. It is not a survey paper looking for any answer that will publish. It is someone asking the question that actually needs answering, with the urgency that comes from loving someone whose body is doing something the medical system has not yet figured out how to stop.
It’s also a love story….
Looking at PMP one cell at a time
Dhal and his collaborator Archie Svetlov reanalyzed single-cell RNA sequencing data from PMP tumors. Single-cell sequencing is exactly what it sounds like. Instead of grinding up a piece of tumor and measuring the average behavior of all the cells mixed together, you measure each cell individually. You get to see what the malignant cells are actually doing, separate from the immune cells, the blood vessels, the connective tissue, and everything else in the sample.
When they looked at PMP tumor cells this way, they found something consistent across multiple datasets. The malignant cells were not running the standard cancer playbook. The genes that drive cell division, the ones oncologists usually target, were quiet. The genes that were loud were the ones involved in making, folding, and exporting protein.
In other words, PMP tumor cells look less like factories that are reproducing and more like factories that are producing.
The polyamine pathway and why it matters
The specific program the researchers zeroed in on involves a metabolic pathway called polyamine-hypusination. The details are technical, but the practical version is this. MUC2, the gel-forming mucin that defines PMP, is an enormous and structurally awkward protein. It contains long stretches of an amino acid called proline, and proline is notoriously difficult for cells to translate efficiently. The ribosome stalls.
To get past those stalls, cells use a specialized translation factor called eIF5A, which only works after it has been chemically modified through a process that requires polyamines. Polyamines come from a pathway whose first and rate-limiting step is controlled by an enzyme called ODC1.
So if you are a tumor cell whose entire job is to crank out vast quantities of a proline-rich, hard-to-translate protein, you need this pathway running at full volume. Not for growth. For output.
That is exactly what the data show. ODC1 was upregulated in all 19 patient samples analyzed. The gene is naturally expressed at low levels in healthy tissue, and in a normal appendix it shows up in roughly 27 percent of cells. In appendix tissue affected by LAMN and PMP, that figure rises sharply to more than 80 percent of tumor cells, with even higher expression across the peritoneum, omentum, and other sites where the disease spreads. The other enzymes in the same polyamine pathway followed the same pattern. And critically, the proliferation markers stayed low. The pathway was tracking with secretory stress, mucin production, and the protein-folding machinery that underpins PMP, not with cell division.
A different therapeutic logic
This is where the study moves from descriptive science to something patients can actually use.
Most cancer drugs are designed to kill or slow dividing cells. That is why they work better against fast-growing tumors and why PMP has historically been so frustrating to treat with conventional chemotherapy. The cells are not dividing fast enough to be vulnerable to drugs that exploit division.
But if PMP is dependent on a metabolic pathway that supports its secretory phenotype, then a drug targeting that pathway would attack the disease where it actually lives. The most established ODC1 inhibitor is DFMO, also known as eflornithine. It is not a new drug. It was originally developed for African sleeping sickness, and it is now used as maintenance therapy in high-risk neuroblastoma in children. It has also been studied for colorectal adenoma prevention. Its safety profile in long-term use is reasonably well understood.
To be clear, no one is saying DFMO is a cure for PMP. The Bina study is a hypothesis-generating analysis, not a clinical trial. There is still significant work between this finding and any patient receiving treatment based on it. The researchers are explicit that direct testing in PMP-relevant systems is needed.
What the study does is build the rationale. It says: here is a pathway that PMP appears to depend on for the very feature that makes it PMP, here is a drug that already exists and already inhibits that pathway, and here is why that combination is worth pursuing seriously.
How patients can move this work forward
This is where the community comes in, and where what you have in your medical records may matter more than you realize.
Single-cell RNA sequencing is the foundation of the analysis Dhal and Svetlov just completed, and it will be the foundation of the next round too. To validate these findings, refine the model, and move toward something that can actually be tested in trials, the work needs more PMP tumor data. Specifically, single-cell sequencing reports from patients who have had this kind of testing done.
Some of you have. Maybe through a research protocol at MD Anderson, MSK, Stanford, or another major center. Maybe through a clinical trial that included tumor profiling. Maybe a curious oncologist ordered it. The reports may be sitting in your patient portal right now.
If you have had single-cell RNA sequencing on your tumor, please consider submitting your report to the Patient-Led Global Appendix Cancer Registry at Appendicure. The registry is built specifically to channel this kind of data to researchers like Dhal who are trying to answer questions the existing scientific record cannot answer on its own. Every additional sample sharpens the picture. Every additional patient brings the next study closer.
PMP is rare enough that no single institution has the data needed to solve it. The patient community, working together, does.
What this means going forward
The biology of PMP is finally being studied on its own terms. For years, PMP was treated as a footnote to colorectal cancer or a strange cousin of it. Studies like this one start with PMP itself and ask what makes it distinct. That matters, because treatments built on the wrong biology will keep failing.
The secretion-versus-growth framing actually matches what patients have been describing all along. The disease is not behaving like a fast-moving cancer because it is not one. Naming that accurately is a step toward treating it accurately.
The work ahead is real. Functional studies in PMP models. Eventually, if the science holds, clinical trials. But this is one of the first analyses that gives a coherent answer to the question patients have been asking for years: why does my body keep doing this?
The answer, it seems, is that PMP cells are running a program built for output, not for speed. And programs can be interrupted.
Bina is one of those patients. So are the people reading this. The data you carry in your records could be what helps the next analysis go further than this one did. Please click here to share your data with the Appendicure Global Patient-Led Data Repository and contribute to the research needed to find new treatments and cures for Appendix Cancer and PMP.
Source: Dhal R, Svetlov A. The Polyamine Pathway in PMP: Secretion Beyond Growth. Bina Therapeutics, April 15, 2026.
Leave a Reply