• Relapsed Acute Lymphoblastic Leukemia (ALL) Gene Therapy Clinical Trial

    Gene Therapy

    Phone: 617-632-5064  

    Chimeric Antigen Receptor (CAR) T-cell Trial for Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia

    Note: This study has reached the target number of patients to be treated, and accrual for the trial is currently closed.

    Relapsed acute lymphoblastic leukemia, or relapsed ALL, refers to the return of acute lymphoblastic leukemia (ALL) in patients who have already undergone treatment for the disease. Between 15 and 20 percent of children who are treated for ALL and achieve an initial complete remission will have the disease return (relapse) at some point

    Our gene therapy clinical trial for relapsed B-cell ALL is testing the safety of giving patients immune-cancer-fighting cells that are created from their own blood; these are called “modified T-cells.” The goal is to find a safe dose of modified T-cells for patients whose leukemia has returned.

    CAR T-cell therapy is a promising option for patients with leukemia for several reasons:

    • Targeted T-cells are capable of both seeking out and eradicating tumor cells
    • They augment anti-tumor immunity by secreting cytokines thereby recruiting other effector cells to the tumor site
    • They can persist as memory cells, potentially conferring long-lasting disease control

    Patients with recurrent leukemia in their bone marrow may have defective T-cells that are less able to kill leukemia cells. By genetically modifying T-cells so that they target the B-cell-specific antigen CD 19 (which is almost always expressed by B-ALL cells), they may be able to better recognize and kill leukemia cells. The concept is to utilize genetic engineering to “re-direct” T-cells to this antigen and also provide additional genetic information to the cell that allows it to better respond to the tumor itself. The genetic engineering is accomplished using a DNA shuttle (or vehicle) to insert new DNA information into the T-cells in a highly specialized laboratory (called a GMP laboratory).

    Children who meet the following criteria may be eligible to take part in this relapsed ALL gene therapy clinical trial:

    • Aged under 26 years
    • Have a history of relapsed/refractory CD19+ B-cell ALL involving the bone marrow
    • Patient would not benefit from additional chemotherapy as determined by the treating physician

    Patients also must NOT have the following:

    • Active HIV, hepatitis B or hepatitis C infection
    • Any concurrent active malignancies requiring any therapy other than expectant observation
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