• Allogeneic Stem Cell Transplant Gene Therapy Clinical Trial

    Gene Therapy

    Phone: 617-632-5064  

    Clinical Trial for Haploidentical Stem Cell Transplant Incorporating Cellular Therapy of Donor Lymphocytes to Decrease Risk of Graft-Versus-Host Disease (GVHD)

    Graft-versus-host disease (GVHD) can occur after an allogeneic stem cell transplant (a stem cell transplant in which someone receives stem cells from a donor). The new, transplanted cells regard the recipient's body as foreign and may attack the recipient's body.

    This clinical trial is testing whether gene therapy can be used to help decrease the risk of GVHD in children and young adults who receive a haploidentical stem cell transplant (a transplant from a partially-matched family donor) to treat a malignant or non-malignant blood cell disorder – including acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), non-Hodgkin lymphoma, myelodysplastic syndrome (MDS), or an immunologic deficiency syndrome.

    The study assesses whether immune cells, called T cells, from the family donor – which will be specially grown in the laboratory and given back to the patient along with the stem cell transplant – can help the immune system recover faster after transplant.

    Children who meet the following criteria may be eligible to take part in this trial:

    • Be between ages 3 Months and 21 Years
    • Have life-threatening hematological malignancy deemed eligible for allogeneic stem cell transplantation – OR – a non-malignant disorder that could be cured by an allograft
    • Lack availability of an HLA identical donor
    • Have a life expectancy greater than 10 weeks
    • Have Lansky/Karnofsky score > 50, WHO > 4
    • Must not be receiving immunosuppressive treatment for Graft versus Host Disease (GvHD)
    • Must not be experiencing significant liver dysfunction or severe cardiovascular disease
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