What the trial was testing
The trial enrolled 81 patients with bladder cancer. The study was sponsored by Memorial Sloan Kettering Cancer Center 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
68% of patients with DNA repair defects responded to immunotherapy versus 19% without them.
Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2018 · NCT02553642
These findings — that versus 19% in patients without these genetic changes — were published in the Journal of clinical oncology : official journal of the American Society of Clinical Oncology and represent the headline result of the study.
Researchers tracked outcomes across 81 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 bladder 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
If you have advanced bladder cancer, tumor genetic testing may predict whether immunotherapy will work for you. Patients whose tumors had specific DNA repair gene mutations were much more likely to benefit from checkpoint inhibitor drugs. These immunotherapy drugs are FDA-approved for advanced bladder cancer — talk to your oncologist about testing your tumor for these mutations before starting treatment.
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 bladder cancer trials
A Dose Escalation and Dose Expansion Study of Intratumoral ONM-501 Alone and in Combination With Cemiplimab in Patients With Advanced Solid Tumors and Lymphomas.
A phase 1, multicenter, open label, non-randomized dose escalation and dose expansion study to examine the maximum tolerated dose, (MTD), minimum effective dose (MED) and/or recommended dose for expansion (RDE) of intratumoral ONM-501 as monotherapy and in combination with a PD-1 checkpoint inhibitor in patients with advanced solid tumors and lymphomas.
Disitamab Vedotin Plus Intravesical Mitomycin C in HER2-Expressing Intermediate- to High-Risk Non-Muscle-Invasive Bladder Cancer
This study aims to evaluate the efficacy and safety of intravenous administration of vedacitinib combined with intravesical instillation of mitomycin in treating patients with high-risk NMIBC with HER2 expression. The goal is to provide a new treatment option for NMIBC patients, reduce the risk of tumor recurrence, decrease the proportion of radical bladder surgery, and improve the quality of life of patients.