• Gene Therapy for Children, Teens and Young Adults

    Gene therapy delivers DNA into a patient’s cells to replace faulty or missing genes – or adds new genes – in an attempt to cure diseases or to make changes so the body is better able to fight off disease. The DNA for a gene or genes is carried into a patient’s cells by a delivery vehicle called a vector, typically a specially engineered virus. The vector then inserts the gene(s) into the cells' DNA.

    Although gene therapy is relatively new and often still considered experimental, it can provide a cure for life-threatening diseases that don’t respond well to other therapies (including immunodeficiencies, metabolic disorders, and relapsed cancers) and for acute conditions that currently rely on complex and expensive life-long medication and management (such as sickle cell disease and hemophilia).

    CAR T-Cell Therapy for Relapsed Acute Lymphoblastic Leukemia (ALL)

    Dana-Farber/Boston Children’s is actively preparing to offer the recently-FDA-approved CAR T-cell therapy called Kymriah for relapsed B-cell acute lymphoblastic leukemia (ALL). This treatment provides renewed optimism for some of the most challenging cases we face in pediatric ALL. The therapy entails genetic engineering of the patient’s own T-cells to increase targeting of a specific leukemia protein and then accelerate killing of the target. After modification, they are returned to the patient via IV where they can immediately begin destroying circulating cancer cells.

    For more information about CAR T-cell therapy, contact our gene therapy program

    FDA approves sickle cell gene therapy trial

    Another Dana-Farber/Boston Children’s gene therapy innovation — this one designed to correct sickle cell disease — will soon reach the clinic. This therapy suppresses the gene that normally shuts off production of fetal hemoglobin. As a result, healthy fetal hemoglobin can still be produced, compensating and replacing "adult" hemoglobin that stiffen and take on the hallmark "sickle" shape that can trigger an acute sickle cell crisis. The trial has received FDA approval and is expected to open for recruitment in mid-November.

    Our Gene Therapy Clinical Trials

    Learn more about our gene therapy clinical trials

    Gene Therapy at Dana-Farber/Boston Children’s

    Dana-Farber/Boston Children’s has one the most extensive and long-running pediatric gene therapy programs in the world. Since 2010, we have treated 25 patients from 11 countries through eight gene therapy clinical trials.

    Why choose Dana-Farber/Boston Children’s:

    • Our patients have immediate access to the in- and outpatient services of two world leading academic medical institutions – Boston Children’s Hospital and Dana-Farber Cancer Institute.
    • Our concierge services make gene therapy treatment easier for families. These include:
      • assistance with travel and housing for both national and international patients
      • assistance with the insurance process; we have successfully negotiated insurance approval for 80% of our U.S. patients – more than any other gene therapy program
    • We have one of the most efficient enrollment processes in any pediatric gene therapy program – allowing children to move quickly and smoothly from study enrollment into treatment, without losing precious time.
    • Our unique satellite training module supports referring physicians who care for our patients. This approach allows children to return home more quickly without compromising clinical monitoring for safety.
    • We provide a lifetime commitment to care – and will continue to answer questions and address concerns related to the patient’s gene therapy treatment throughout the patient’s life.

    Learn more