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.
Open colorectal cancer trials
A Study of BL-B01D1 in Patients With Locally Advanced or Metastatic Gastrointestinal Tumor and Other Solid Tumor
In phase Ia study, the safety and tolerability of BL-B01D1 in patients with locally advanced or metastatic gastrointestinal tumor and other solid tumor will be investigated to determine the dose-limiting toxicity (DLT), maximum tolerated dose (MTD) of BL-B01D1. In phase Ib study, the safety and tolerability of BL-B01D1 at the phase Ia recommended dose will be further investigated, and recommended phase II dose (RP2D) for phase II clinical studies will be determined. In addition, the preliminary efficacy, pharmacokinetic characteristics, and immunogenicity of BL-B01D1 in patients with locally advanced or metastatic gastrointestinal tumor and other solid tumor will be evaluated.
New bioMarkers tO straTIfy cOlorectal caNcer Referrals
The goal of this observational study is to evaluate if a blood test for circulating progastrin (hPG80) and transposable elements (TEs) can accurately predict colorectal cancer (CRC) or polyps in adult patients referred to the 2-week wait (2WW) or Straight to Test (STT) pathways for suspected lower gastrointestinal cancer. The main questions it aims to answer are: Can plasma hPG80 levels accurately predict a diagnosis of CRC or polyps in patients undergoing standard 2WW investigations? Can transposable elements (TEs) in the plasma serve as predictive biomarkers for CRC diagnosis in these patients? What are the patient preferences for different diagnostic tests for CRC, particularly a blood-based test compared to more invasive methods? Participants will: Provide a 20ml blood sample during a routine hospital visit for their 2WW diagnostic test (e.g., colonoscopy, CT Colon). Undergo standard clinical investigations as determined by their treating clinicians. Have their final diagnosis (cancer, polyp, or normal) correlated with their plasma hPG80 levels. For a subset of 100 participants (25 with confirmed CRC, 75 non-cancer), have their plasma analyzed for circulating signatures using RNAseq and DNAseq. Complete an electronic post-study questionnaire to explore their preferences and experiences with different CRC diagnostic tests used within the 2WW pathway.