Pediatric Cellular Immunotherapy: CAR T-cell Therapy
Immunotherapy is a treatment that mobilizes the body’s own immune system to fight diseases. A new type of personalized immunotherapy — CAR T-cell therapy — is revolutionizing treatment for B-cell acute lymphoblastic leukemia (ALL), the most common pediatric cancer.
Most children with ALL can be cured using conventional treatments: chemotherapy, radiation and stem cell transplant. But in 20 percent of cases, the cancer is refractory (does not respond to treatment) or returns after treatment. Until now, doctors had few options to fight aggressive ALL. Today, the medical community sees great promise in CAR T-cell therapy for these patients.
CAR (Chimeric Antigen Receptor)T-cell therapy reprograms a patient’s disease-fighting white blood cells (T-cells) to seek out, recognize and attack cancer cells without harming healthy cells. After CAR T-cell immunotherapy saved the life of a young ALL patient in Philadelphia five years ago, the experimental treatment was offered to more children through clinical trials. In the majority of cases, signs of cancer disappeared and the patients continue to be in remission.
How Does CAR T-cell Therapy Work?
How does CAR T-cell therapy work?
Clinical trials at UChicago Medicine helped develop CAR T-cell therapy for leukemia and lymphoma patients. By removing, supercharging, then returning white blood cells into the bloodstream with instructions to find and kill cancer cells, this living drug can often result in full remission. The process works like this, CAR T-cell therapy uses genetically engineered versions of the patient cells to find tumor cells and kill them with minimal damage to healthy cells.
In the first part of the process, T-cells, the workhorse of the immune system, are collected from the patient's blood. Then scientists insert instruction that enable those T-cells to find specific cancer cells. While the T-cells multiply in the lab, the patient receives chemotherapy to reduce the number of cancer cells. And finally, the engineer T-cells are returned to the patient's bloodstream, where they seek out and kill remaining cancer cells. For more information about this exciting new treatment, visit UChicagoMedicine.org/defeatcancer or call 844-482-7823.
CAR T-cell Therapy for Pediatric Patients
I'm extremely excited that the University of Chicago Comer Children's Hospital is one of the small number of the initial sites nationwide that is going to provide this new and revolutionary therapy for acute lymphoblastic leukemia.
Previously, we're only curing about 10% of the time. And with this new therapy, we're going to be able to care about 80% of these patients.
What will happen, they will come to see us. Cells from their immune system, T cells, will be collected and then in the laboratory will be enhanced or supercharged, so that way they can target the leukemia cells to kill them. These cells are reinfused.
And our hope is that, after a few weeks, we'll see that these leukemia cells that we previously couldn't get rid of are now gone.
This will hopefully allow some patients who would have gone on to get even more intense chemotherapy, perhaps second or third bone marrow transplants, to receive a therapy that won't carry as many long-term or late effects.
Over 70% of children who receive CAR T cell therapy experience toxicity, including respiratory distress and neurological toxicity.
As a result of that, patients will have troubles with breathing and respiratory insufficiency. They may have some kind of changes in their mental status. We watch all these patients very, very closely, and we have a very robust supportive regimen. So virtually all patients recover from these toxicities.
The advent of a licensed drug CAR T cell therapy for acute lymphoblastic leukemia really is a change where we can really now say for many of these children, who would previously have died unfortunately of their disease, that they're cured. The data so far suggests that, unlike in the past where 10% of these children survived, over 80% of these children survive with CAR T cell therapy.
The very first patient who was treated was treated six years ago, treated for ALL. And she's cured. And she's out in the world. She's being a normal adolescent, and that's what we hope for the rest of our patients.