AUTO3, which is part of a new generation of personalised ‘CAR-T’ cancer therapies, works by genetically modifying a patient’s own immune cells to recognise and destroy cancer cells. Immune cells are taken from the patient’s blood and modified to target a specific molecule found on the surface of cancer cells.
Great Ormond Street Hospital (GOSH) has been at the forefront of research in to CAR-T cell therapy for children with leukaemia with three active clinical trials. CD19 CAR-T cells have shown remarkable results in children with otherwise incurable ALL and have recently been licensed by NHS England. However, approximately 50% of patients relapse because the leukaemia evolves to down-regulate the CD19 target on leukaemic cells. AUTO3 is the first CAR-T therapy to simultaneously target two different molecules – CD19 and CD22 – which could reduce the chance of relapse after CAR T cell therapy.
The AMELIA trial found that AUTO3 was well-tolerated and safe for use in children with the rare form of blood cancer, acute lymphoblastic leukaemia (ALL).
“AUTO3 appears to have a manageable safety profile, with the potential to overcome target-negative relapse, a major limitation of current CD19-targeted therapies," said Professor Persis Amrolia, lead investigator and Consultant in Bone Marrow Transplant at Great Ormond Street Hospital (GOSH) and NIHR Research Professor of Transplantation Immunology at UCL Great Ormond Street Institute of Child Health (ICH).
AMELIA, which is a multicentre phase I/II study led by GOSH in the UK, aims to treat 54 children in total and will evaluate the safety and optimal dose of AUTO3 for patients. 10 children with relapsed ALL have so far been treated and eight of these patients entered remission.The interim results for both AUTO3 trials were presented at the American Society of Haematology (ASH) annual meeting earlier this month.