How do clinical trials contribute to advance ALL treatments, and how can patients find clinical trial opportunities?
Acute lymphoblastic leukemia in children is one of the greatest success stories in all of hematology oncology. The way that we have reached the point where more than 90% of children are cured of their illness is through clinical trials and through cooperative groups. I often refer to progress through collaboration. There are a number of cooperative groups, both in pediatric ALL as well as adult ALL, that have allowed us to have multiple different centers working together on the same study.
When you have a relatively rare disease like ALL, where you only have, let's say, 7,000 patients per year in the United States, you want to try to learn from every patient in order to advance the standard of care. The way that we've gotten to where we are now is by building on what we know already through clinical trials. Most frontline clinical trials in ALL use drugs that we already know are active, but maybe use them in a slightly different way, like a different dose or schedule, or might include a new drug on top of drugs that we know already work.
Sometimes when patients hear about clinical trials in the upfront setting, they think this is something that maybe has never been investigated before or is a first-in-human study. But when we talk about using clinical trials upfront in patients with ALL, we're talking about therapies that we know have activity and that we know can be effective, but trying to improve on the standard of care so that we can have more patients achieving long-term cure.
The way that we learned, for example, that there's a benefit to adding the bispecific T-cell engager blinatumomab in adults with B-cell acute lymphoblastic leukemia, even in those who've already achieved minimal residual disease negativity by flow cytometry, was through a large cooperative group study through the Eastern Cooperative Oncology Group, or ECOG.
There are clinical trials that are also important for patients whose leukemia has not responded as optimally to their initial treatment regimen. It's important to talk with your hematologist-oncologist about the phase of that particular trial, as well as the trial design, so that patients know what they would be receiving if they enrolled in that trial.
There are several resources available for finding clinical trials. One is ClinicalTrials.gov, where nearly every trial in the United States is required to register. A number of societies, such as the Leukemia & Lymphoma Society, also help connect patients with clinical trials through patient navigators. The HealthTree Foundation also has resources available to help patients find appropriate clinical trials for their condition. You can certainly reach out there. And of course, you can always talk with your hematologist-oncologist about what clinical trials might be available in your area and whether they would be right or particularly appropriate for you.