1. Investigational drugs: Gene modified WT1 (Wilms’ Tumor 1) TCR therapy - Patient's white blood cells (T cells) will be modified by transferring a gene which enables them to make a new T cell receptor (TCR) that can recognize fragments of a protein called WT1 which is present at abnormally high levels on the surface of myelodysplastic and leukaemic cells.
  2. For what level of MDS severity risk: Intermediate – 2 or High Risk
  3. What subtype of MDS: High (> 10% bone marrow blasts); also Acute Myeloid Leukaemia
  4. Aims and benefits: 
    • Safety and signs of efficacy of the WT1 TCR therapy in a carefully defined group of patients (those who have failed to respond to Azacitidine but do not have rapid disease progression).
    • Aims to assess blood count improvement, overall remission rate, marrow remission, cytogenetic response, reduction in size of molecular clone, AML transformation, progression free, event free and overall survival.
  5. Basic inclusion criteria:
    • Aged ≥18 yrs
    • ECOG performance status 0, 1 or 2
    • Diagnosis of MDS (Intermediate – 2 or High Risk) or AML
  6. Basic exclusion criteria:
    • Patients who are in complete remission, partial remission, marrow complete remission or have cytogenetic response, or haematological improvement or molecular response following Azacitidine treatment
    • CMML patients who have a white blood cell count > 13 x 109/L
      Acute promyelocytic leukaemia (FAB M3 Classification)
    • Active malignancy, except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix or breast
    • Known history of Human Immunodeficiency Virus (HIV), Hepatitis C virus (HCV) or Hepatitis B virus (HBV) or positive for Human T-Lymphocyte Virus (HTLV) or Syphilis.
    • Active auto-immune disease
    • Kidney impairment requiring dialysis
  7. Trial sites/locations and name of physician in charge of trial: 
    Aberdeen Royal Infirmary, Aberdeen; University College London Hospitals NHS Trust, London.
    University Hospitals Bristol NHS Foundation Trust, Bristol;
    Western General Hospital, Edinburgh;
    The Leeds Teaching Hospitals NHS Trust, Leeds;
    University College London Hospitals NHS Trust, London
  8. More information:

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