Children and young adults with certain types of leukemia may be eligible for a groundbreaking new treatment option through University of Minnesota Health starting this fall.
University of Minnesota Masonic Children’s Hospital is one of only 30 centers in the nation certified to offer this treatment, the first Food and Drug Administration-approved CAR T-cell therapy, to patients with treatment-resistant or relapsed acute B-cell lymphoblastic leukemia (ALL).
CAR T-cell therapy is an innovative form of immunotherapy that harnesses a patient’s own immune system to fight cancer. The new treatment received FDA-approval on Aug. 30, following a multi-site study to test the effectiveness of the drug. University of Minnesota Masonic Children’s Hospital was one of the sites that participated in the study. The overall cancer remission rate within three months was 83 percent for patients involved in the study.
“This has the potential to transform cancer care,” said Pediatric Blood and Marrow Transplant Physician Heather Stefanski, MD, PhD, who treats pediatric blood cancer patients. “Before, the only option for patients with blood cancer was a bone marrow transplant or chemotherapy, which is very toxic to the entire body.”
We asked Stefanski and Hematologist/Blood and Marrow Transplant Physician Veronika Bachanova, MD, PhD, who treats adult patients, to answer some helpful questions about CAR T-cell therapy and eligibility for the treatment.
How does CAR T-cell therapy work?
During CAR T-cell therapy, specialists extract a large number of T-cells from a patient’s own blood. T-cells are a vital part of a person’s immune system. Typically, they travel through a person’s body to hunt down and destroy infected or abnormal cells. Following extraction, the T-cells are “reprogrammed” outside the body to target CD19, a protein found on the surface of acute lymphoblastic leukemia cells. Those modified T-cells are then infused back into the patient’s blood, where they go to work attacking cancer cells. Prior to the infusion, patients receive four days of chemotherapy to prepare their bodies to receive the new T-cells. CAR T-cell therapy works with just one dose and also acts rapidly—within days the modified T-cells are at full strength.
“CAR-T therapy represents a revolutionary step in fight against cancer,” Bachanova said. “Administered in a single infusion, CAR-T therapy is a viable and effective option for children and young adults who meet eligibility criteria for the therapy.”
Who is eligible for this new treatment?
Right now, children and young adults up to 25 years of age with refractory or relapsed acute B-cell lymphoblastic leukemia (ALL) are eligible for this treatment. However, a number of new studies testing the effectiveness of immunotherapy drugs against other forms of cancer—such as diffuse large B-cell lymphoma—are already underway. New therapies may be available soon, Stefanski said. Prior to beginning the CAR T-cell therapy, each patient will be evaluated for eligibility by a multi-disciplinary team at University of Minnesota Masonic Children’s Hospital to ensure that the therapy is appropriate for the patient. Adult patients can be referred to University of Minnesota Health for an evaluation. “CAR-T therapy is the first of its kind to be FDA approved,” Stefanski said. “This is a huge step for all cellular therapies and will change the landscape of how we treat cancer in the future.”
Are there any side effects?
Yes. Kymriah, the therapeutic drug that received FDA approval
, carries its own unique set of side effects. Possible complications include flu-like symptoms with high fevers, muscle aches, low blood pressure and headaches related to cytokine release syndrome (CRS). During studies, some patients also experienced neurological problems. If you are eligible to receive this treatment, our doctors will discuss the benefits and the potential side effects with you in detail during an appointment.
How do I learn more?
If you are interested in learning more about CAR T-cell immunotherapy treatment through University of Minnesota Health, including details regarding cost and insurance coverage, we encourage you to call 612-273-2800 to speak with our team.