GU Cancers 2026 | Immune checkpoint therapy hyper-progression in renal medullary carcinoma

So this is exactly an example of how a relatively novel at the time, clinical trial design, a so-called Bayesian phase two trial design developed in our group at our institution originally, helped us in a very fast and efficient way activate, conduct, complete, accomplish it, and get the maximum information for a rare and deadly kidney cancer. So renal medullary carcinoma, when not treated, is the deadliest of the kidney cancers. And it is the only cancer that I’m aware of that so specifically afflicts a minority, young individuals of African descent. That particular trial was the first ever clinical trial specifically designed for that kidney cancer subtype and we were able to activate it and finish it and learn from it thanks to the novel trial designs that we used. And also by thinking that no matter what happens with the trial when it is conducted correctly, it is a win-win situation. When it shows the efficacy that you hoped for, then it’s a win. But also when the trial is so-called negative, you can learn even more. When a trial is negative despite your hopes for it, that refutes your original hypothesis, so that gives you new information. It is exactly that new type of information that taught us, specifically from this that that type of combination immune checkpoint therapy is not appropriate for most or possibly all of these patients which allowed us to then change our treatment strategies in ways that have actually improved the survival outcomes in this rare disease. Our hope is now to use this paradigm to also accelerate discoveries across other rare and common kidney cancer types.

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