Gene Therapy Program

FDA-Approved Gene Therapies | CAR T-cell Therapy for ALL

The Dana-Farber/Boston Children's Cancer and Blood Disorders Center offers the first FDA-approved CAR T-cell therapy called KYMRIAH™ for patients up to 25 years of age with relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (ALL).  

What is CAR T-cell therapy?

CAR (chimeric antigen receptor) T-cell therapy is a promising treatment for pediatric ALL that works by altering the body's T-cells — immune system cells — to detect and destroy infected ‭or cancerous cells.

To genetically engineer these cells to fight cancer, a sample of a patient's T-cells is collected intravenously, then modified in the lab to produce special structures called chimeric antigen receptors (CARs) on their surface. Once reinfused into the patient, the new receptors enable the T-cells to latch onto a specific antigen on the patient's tumor cells and destroy them.

Who is eligible for KYMRIAH™?

KYMRIAH™ is indicated for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. You and your care team will decide if this treatment is right for your child.

CAR T-cell therapy for lymphoma

Dana-Farber/Boston Children’s is also a certified treatment center for providing KYMRIAH™ to patients who are 18 and older with relapsed or refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

For more information about whether KYMRIAH™ is right for you or your child, or to refer a patient, contact:

Colleen Dansereau, RN
Gene Therapy Director of Clinical Operations
We are proud to serve a broad and diverse patient population that often comes to us for answers they can't find anywhere else. This top ranking is a credit and a tribute to our extraordinary and deeply committed clinicians, researchers and so many others who dedicate every day to improving and advancing the health and well-being of all children.

Sandra L. Fenwick, CEO

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