Leukapheresis of white cells from patients with CLL for gene therapy
Research type
Research Study
Full title
Leukapheresis of white cells from patients with Chronic Lymphocytic Leukaemia to develop immunotherapy approaches using genetically modified T cells
IRAS ID
158864
Contact name
Amit C Nathwani
Contact email
Sponsor organisation
University College London
Duration of Study in the UK
0 years, 11 months, 30 days
Research summary
Chronic Lymphocytic Leukaemia (CLL) remains incurable for large numbers of patients despite recent advances in available treatments. Novel therapeutic strategies are urgently needed for these patients which forms the basis of our research focus: To redirect patients own normal immune cells to target and kill leukaemia cells following genetic modification.
T-Cells are potent immune cells and can orchestrate an attack on invading pathogens when they recognise specific proteins via their T cell receptor. We have developed a Chimeric Antigen Receptor (CARs) against proteins expressed on CLL and can insert CARs into patient T-cells using gene therapy. When these CAR T-cells recognise the target protein on the surface of the CLL they become activated and kill the leukaemia cells.
We have already shown that using the above approach CAR T-Cells are effective at killing CLL in vitro but before we can take this forward to benefit patients we need to be able to generate CAR-T cells from patient T cells and show that these cells are effective and safe.
Our Aims are:
1. Develop and optimise a protocol through which large numbers of CAR-T cells can be generated in sufficient numbers to allow for a future clinical trial.
2. Demonstrate safety and efficacy of the CAR-T cells generated using this newly developed protocol. We would need to ensure that they comply with guidelines from the Medicines and Healthcare products Regulatory Agency & Gene Therapy Advisory Committee.This proposal seeks approval to obtain sufficient number of cells from patients with CLL via a leukapheresis procedure to allow us to fulfil these aims. Leukapheresis is a specialised procedure where blood is taken from patients in a circuit and centrifuged to remove only the component of interest (in this case the white cells) before the plasma, red cells and platelets are returned back to the patient.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
14/EM/1304
Date of REC Opinion
19 Dec 2014
REC opinion
Favourable Opinion