ASH 2025 | The PedAL master trial and sub-studies: accelerating drug development in pediatric acute leukemias

This is a master trial effort that we started now five years ago looking to try and make pediatric drug development go faster and also make sure that we actually did pediatric trials that were of the quality and quantity to get therapies approved or labeled for kids. Because in the United States, too many kids are getting therapies off-label, and they’re adult therapies that the pediatricians are forced to use without any data to really tell them how to use them. So we have an international collaboration. What we started with is what we call our screening trial. And what that is in the United States, though we have a parallel effort in Europe, is every child agrees to be on this screening trial provide their sample and then they get free molecular testing, so they get their sequencing done for free. And unfortunately we came to do this because kids were in a catch-22 – they were not able to decide whether to use a FLT3 inhibitor or an IDH inhibitor or any other targeted therapy because many kids didn’t have the sequencing done, and insurance wouldn’t pay for it because there were no therapies approved. And so we went around in circles. And so our goal was to provide the sequencing. And so Blood Cancer United pays for the sequencing for these kids. And over the period of the last three years while we’ve been doing this trial, we have over 500 kids who’ve had full sequencing and that goes back to their docs so that they can make decisions about therapy. Some of those kids go on the PedAL trial, but that isn’t our primary goal. It’s to make sure every kid has as many options for the best possible therapy up front. And we’re kind of prescient in that, you know, you need that information to go on some of the menin inhibitors and some of the other therapies that are now becoming so important for pediatric AML. 

Right now, we have two subtrials. One is actually a large international randomized trial of standard relapse therapy with and without venetoclax. And our hope is to get venetoclax as a therapy that’s approved and paid for all over the world, not just so docs in the U.S. can get it on the shelf because they work at a hospital that also has adult oncology. Our second trial is ziftomenib, and we’re particularly proud of that because Blood Cancer United sponsored the research that actually discovered this drug and this therapeutic pathway. And so we also, when we helped the investigator create a company around this, that company was part of our therapy acceleration program. So we helped it with research, with our therapy acceleration program and now we are having it as a trial within PedAL so it’s really full circle for us and very exciting. We have been talking about a third trial here at ASH and there isn’t ink on paper yet so I can’t talk about it, but it’s a very exciting trial that will be a new type of therapy entirely for kids with leukemia and very close in to the adult trials so that, like venetoclax, we aren’t waiting six years after the adult approval for the pediatric trial to get done. And that’s the goal of PedAL, is to get, not to change, you know, the process, but to have it happen sooner so that people are thinking about pediatrics and we help pharma companies find a more efficient way to get this done.

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