AUTO1 in relapsed or refractory B-ALL
Research type
Research Study
Full title
An open-label, multi-centre, phase Ib/II study evaluating the safety and efficacy of Auto1, a CAR T cell treatment targeting CD19, in adult patients with relapsed or refractory B cell acute lymphoblastic leukaemia
IRAS ID
274671
Contact name
Claire Roddie
Contact email
Sponsor organisation
Autolus Ltd.
Eudract number
2019-001937-16
Duration of Study in the UK
4 years, 0 months, 1 days
Research summary
B-Cell Acute lymphoblastic leukaemia (ALL) is a blood cancer that is common in both adults and children. In Europe, there is an estimated 5,649 new cases and 1,700 deaths per year. The overall prevalence of the disease in Europe is estimated to be 51,099. Patients are predominantly children; approximately 60% of cases occur at age < 20 years. The incidence of ALL peaks between ages 2 and 5 years and another peak occurs in patients older than 50 years of age. It is a serious and life-threatening disease and will progress rapidly if left untreated. There are several treatments available, including chemotherapy, immunotherapy and stem cell or bone marrow transplant. However, despite this, approx. 50% of adult patients relapse and have a very low chance of cure with conventional treatments and chances of long-term survival remain low. Since most of these patients have frequently had the maximal tolerable dose of chemotherapy, it would be highly desirable to develop safe and effective new therapies such as a CAR T cell therapy that could also avoid the mortality, and morbidity associated with current therapy.
T-cells are white blood cells which are part of our immune system. Their function is to move around our bodies on a "seek-and-destroy mission" against cells in our body infected with a virus. Medicine has long sought to harness T-cells to fight cancer, however because cancer cells develop from our own cells, T-cells do not readily target them. This study tests a way of "re-programming" T-cells so they recognise ALL cells.
The study involves harvesting T-cells which are present in blood; to harvest enough T-cells, patients in this study will have a medical procedure called leukapheresis. This involves passing blood from the patient through a machine which separates out white blood cells and returns the rest of the blood to the patient. The patient's own T-cells are taken to a specialised laboratory. Here, a new gene is inserted into the T-cells. This gene instructs the T-cells to make a new protein called a "chimeric antigen receptor" (CAR). This CAR (known as AUTO1) allows the T-cells to recognise and kill leukaemic (ALL) cells.
The AUTO1 CAR T-cells are given back to the patient via an intravenous drip. Patients in this study will have ALL which has come back (relapsed) and no longer responds to standard treatment (refractory). The first phase of this study will test the safety profile and dosing of AUTO1. The second phase of the study will test how effective AUTO1 is in ALL.REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/1949
Date of REC Opinion
17 Feb 2020
REC opinion
Further Information Favourable Opinion