But the field of CAR-T cell therapy and immune cell therapy, genetically engineered immune cell therapy, has benefited from these early approvals of the CAR-T cells and blood cancers, and there's been a lot of investment not just by granting agencies but importantly (by) commercial investors creating companies to develop these therapies.Īnd it's also important to note this is worldwide. It will take some time to move those forward into latter-stage clinical trials and regulatory approval and commercial development. There are preclinical studies in other cancers, in some solid cancers, neuroblastoma and high-grade glioma or brain cancers. The first approved CAR-T cell therapy was actually approved in a pediatric indication, acute lymphoid leukemia (ALL), and that is Kymriah (tisagenlecleucel). Some things that may differ are in some of the preconditioning chemotherapy that the patients receive (or) some of the treatment that they receive after to manage side effects. Levine: So, the treatment, the conceptual part of the treatment, and the execution is more or less the same. And you could think of it like a remote, genetically engineered immune cell transplant.īiese: And then when you're dealing with pediatric patients with cancer specifically, does the approach to CAR-T differ at all in treatment course or intensity or anything like that? Going back to the preclinical work, it's a complex process to manufacture and execute this therapy. So we're removing immune cells, we're collecting them, and they go to a laboratory where that gene encoding the chimeric receptor can be delivered to the T cells, the T cells grow up in the lab for a short period of time and then they're frozen and they are tested according to criteria for safety, purity, potency and identity and then can be released back to the patient. And then, this molecule continues inside the cell and inside the cell are signaling domains that tell the T cell, "Hey, this is something you want to pay attention to and start to react."Īnd we're doing this outside of the body. Now, some people may know that the chimera is a mythical beast composed of the head of a goat, the body of a lion and the tail of a serpent and in our case, we're creating a molecular chimera with parts of an antibody domain to recognize antigen or cancer antigens. So, what we do is to teach immune cells, specifically T cells, to recognize cancer by inserting a chimeric receptor. But in large part, the immune system is unaware and unable to react to cancer. So how could the immune system recognize cancer if it's derived from your own cells? Now, there are some rare immune cells that can recognize some cancer mutations, particularly for some virally induced cancers. Now, we know that cancer is derived from our own cells, the immune system evolved to tell self from non-self. Levine: Well, at the most basic level, CAR-T cell therapy is a way to teach your immune system to recognize cancer. But for anyone who isn't familiar, what is CAR-T cell therapy and on a fundamental level, how does it work? So, CAR-T therapy, I think anyone who observes or works in the oncology space has been hearing a fair bit about CAR-T lately. And thank you so much for highlighting the Emily Whitehead Foundation and their tremendous work.īiese: Absolutely, it is our It is our pleasure. Levine, thank you so much for taking some time to talk with us about this topic today.īruce Levine: Well, it's a pleasure to be with you. Bruce Levine of the University of Pennsylvania Perelman School of Medicine about CAR-T therapy. In this edition of "Speaking Out" video series on behalf of the Emily Whitehead Foundation. I'm the assistant managing editor of CURE magazine.
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