Around eight people are diagnosed with AML every day in the UK. But years of hard work in UK laboratories have led to trials for a revolutionary new treatment.
It’s led to a therapy that works by modifying a patient’s own stable, non-cancerous white blood cells (T-lymphocytes or ‘T-cells’) to attack cancer cells which are created by other, abnormal white blood cells.
“Seeing patients in clinic every week is a stark reminder of how urgent it is to have new treatment options for diseases such as acute myeloid leukaemia,” Dr Emma Morris, UCL Clinical Academic, chief investigator and inventor of the therapy commented.
In AML a patient’s white blood cells become cancerous. The treatment involves modification by gene therapy of the patient’s own blood cells.
T-cells are extracted and genetically engineered so that they bind to a protein called WT1 on the cancer cells. The cells are re-infused into the blood with the aim of triggering an immune response against cancer cells.
It’s hoped the therapy will encourage a similar immunology to chicken pox – once you’ve had it, you don’t get it again.
“The immune cells will stay in your body forever and we hope it will be able to control the leukaemia if it returns,” Dr Morris told The Times newspaper in August 2014.
The commercial divisions of University College London and Imperial College, along with the Cell Therapy Catapult – a Catapult centre funded by Innovate UK – have formed a new joint venture company Catapult Therapy TCR Ltd, to develop the therapy.
The joint venture – supported by an investment of up to £10 million from the Cell Therapy Catapult – has been set up to accelerate clinical trials and work on making the therapy accessible to patients once trials are successfully completed.
“The involvement of the Cell Therapy Catapult has really accelerated project progress and put it on a much stronger commercial footing. It’s an exciting time for us,” Dr Morris commented.
Phase II clinical trials are expected to begin in 2015. Manufacture of the genetically modified cells will be undertaken at the world-leading UCL Institute of Child Health/Great Ormond Street Hospital cell therapy production unit led by Professor Adrian Thrasher and Dr Waseem Qasim.
The Cell Therapy Catapult, based at Guy’s Hospital in London, works with the cell therapy community to develop and commercialise therapies and build the wider UK cell therapy industry.
It’s acting as trial sponsor for this venture and its skills and expertise in manufacturing, process development and commercialisation complement those of Imperial College and UCL.
“We’re helping to develop manufacturing processes with clinical trial applications, as well as being the trial sponsor,” said Keith Thompson, chief executive of the Cell Therapy Catapult.
“Our mandate has many aspects: return to developers and funders; jobs created, via manufacturing and the supply chain; inward investment and exports. Generating drug sales is not our sole purpose. Our aim is to build a healthy, sustainable cell therapy industry in the UK,” he added.
“It’s hard to quantify the amount of acceleration the catapult has brought to this particular project at present since this may only become obvious in the later stages of drug development, but I’d say months if not years,” said Keith.
“On the commercial side, the formation of Catapult Therapy TCR Ltd gives a clear mechanism for future funding and development of a therapy. It’s one we hope that future funders such as venture capital/pharma/biotech firms will be comfortable with,” he concluded.
Imperial College, UCL and the Cell Therapy Catapult, have formed a company to take a new leukaemia-fighting cell therapy to market. Catapult Therapy TCR Ltd will develop a T-cell receptor therapy to treat acute myeloid leukaemia and haematological disorders that affect thousands of patients in the UK each year.