Clinical trial of an immuno-oncology treatment with UK Universities

Breaking down industry barriers: progressing an immuno-oncology treatment through clinical trial.

The challenge

A novel approach to the treatment of acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) was being developed by scientists at Imperial College and University College London (UCL), initially funded by the charity Leukaemia & Lymphoma Research. Patients with these diseases often have limited treatment options and a poor prognosis, meaning new treatment options are much needed. A new multiparty approach was needed in order to accelerate the development through clinical trials of the treatment, a T-cell therapy targeting overexpression of the WT1 antigen.

Working in collaboration to find a solution

The Cell and Gene Therapy Catapult, UCLB and Imperial Innovations worked together to form a new joint venture company, Catapult Therapy TCR Ltd, to manage the development of the WT1 cell therapy. Having a spin out also allows everyone to share in its eventual success.

Our expertise

The Cell Therapy Catapult is using its expertise in many aspects of this project, including clinical trial operations, regulatory affairs, manufacturing, analytical and process development.

The collaboration between my research team and the Cell and Gene Therapy Catapult has enabled us to begin opening additional clinical trial sites across the UK and Europe for the testing of novel immunotherapies in patients with cancer.

Prof Emma C Morris — UCL Institute of Immunity and Transplantation, Chief Investigator and co-inventor as well as Director of the Infection, Immunity and Inflammation Programme at the National Institute for Health Research University College London Hospitals Biomedical Research Centre

Outcomes so far

CGT Catapult was transferred sponsorship of the existing UK trial focused on AML, opening new sites and accelerating recruitment. Successful interim safety results were achieved in this trial in early 2016. In addition we set up a new trial targeting MDS with sites both in the UK and selected other European countries. This both demonstrated and expanded the experience and expertise of the regulatory and clinical operations teams as we progressed the trial through the approval process for Gene Modified Organisms and Clinical Trial Application and clinical trial setup in the UK, Belgium, Germany and France.

We established the set up and delivery of seamless logistics from procurement of patient sample through centralised manufacture to timely delivery of product for patient.

On the industrialisation front, we have managed to reduce the future Cost of Goods by 60 percent and cut the number of process steps from 978 to 188. We also achieved successful transfer of manufacture to a UK CMO and RT-PCR and flow cytometry assays to a UK CRO. Work is ongoing on further process and analytical improvements.

Generating health and wealth

This T-cell therapy project is addressing the clear unmet medical needs of acute myeloid leukaemia and myelodysplastic syndrome, for patient benefit, as well as showing the UK to be a leader in the development of this novel, growing and important area of medicine.