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Colorectal CancerDecember 2020Summary reviewed August 2026

A Drug Shrank Colon Polyps in 80% of People with Inherited Cancer Risk

Four people with familial adenomatous polyposis — a condition that causes hundreds of colon polyps and greatly raises cancer risk — took sirolimus for six months. The drug shrank 16 out of 20 tracked polyps and reduced the total number of polyps in all four patients. Everyone had side effects, and one person stopped early because of them.

What the trial was testing

The trial enrolled 4 patients with colorectal cancer. The study was sponsored by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) and tracked outcomes across the full group of patients who matched the trial's eligibility profile.

It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.

What the results showed

The drug shrank 16 out of 20 tracked polyps and reduced the total count in all four patients.

BMJ open gastroenterology · 2020 · NCT03095703

These findings — that shrank after six months of treatment — were published in the BMJ open gastroenterology and represent the headline result of the study.

Researchers tracked outcomes across 4 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.

What this means for patients

For patients with colorectal cancer, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.

What you can do now

This was an early-stage study in just four people, so this treatment is not yet standard care. If you have FAP and continue to develop polyps after surgery, talk to your doctor about whether sirolimus or other options might be right for you. The drug showed promise but caused side effects in everyone who took it.

Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.