There is new hope for patients with an incurable type of leukaemia – thanks to scientists at the Royal Free Hospital who have helped develop a new type of treatment.
The treatment - known as CAR T-cell therapy – is intended for patients with relapsed B cell acute lymphoblastic leukaemia and researchers at University College London Hospital (UCLH) have just completed a successful phase 1 clinical trial, the first step in human testing.
The CAR T-cell products used in the trial, known as ALLCAR19, were produced at the Centre for Cell Gene and Tissue Therapeutics (CCGTT), based at the Royal Free Hospital.
The therapy works by programming the body’s immune cells (T cells) to recognise and destroy cancerous cells.
The phase 1 clinical trial is a significant step forward for patients, as there is currently no curative treatment available for people who have this type of leukaemia.
Dr Owen Bain, co-director of the CCGTT, says the latest positive trial results show just what the Royal Free London and the NHS is capable of.
He said: “The latest announcement about the ALLCAR19 trial demonstrates the fundamental role of the CCGTT in the development of advanced therapies and bringing these to the clinic.”
Trial investigator, Dr Claire Roddie, associate professor at UCL Cancer Institute and consultant haematologist at UCLH, said “As both scientists and doctors, we are delighted with the results; they are important because they show the new C19 CAR design allows for the safe treatment of CAR T-cell therapy to adult patients with relapsed B-ALL. Moreover, this treatment may allow patients to have long-term remissions with no other treatment.”
Comprised of just 16 members of staff, the CCGTT has accomplished a great deal since it began manufacturing CAR-T cells in 2017, providing personalised treatments for patients who have lymphomas, leukaemia and multiple myelomas. Currently these experimental treatments are given when all other therapies have been exhausted.
Dr Bain explains: “The CAR T-cells are known as ‘vein to vein products’. Patients have a sense of empowerment as they are a fundamental part of the process. The original cell material comes from the patient, is processed at our state of the art manufacturing facility, and is given back to the patient. We modify the white blood cells (T-cells) from the patient to generate an army of cells that are specifically designed to attack and destroy the patient’s cancer.”
In addition to the work on CAR T-cell therapy, the CCGTT is also partnering with other trusts to develop new treatments for a range of conditions. Clinical trials are currently underway at Moorfields Eye Hospital for a product produced at the CCGTT which is designed to treat age-related macular degeneration. This product has already restored sight in a couple of patients.
The CCGTT team are also celebrating reaching the milestone of their 100th product since they began manufacturing - the ‘century’ product was part of the ALLCAR19 trial.
Dr Bain added: “As well as creating life-saving and life-changing products for patients, we also want to bring costs down by engineering better manufacturing solutions. We are a feather in the cap for the UK industry as a leader on the world stage of cell and gene therapy manufacture, acting as a catalyst to take these novel therapies from research to the clinic. We demonstrate how innovative and powerful a small unit in the NHS can be as part of the development of a new paradigm of medicinal products. One of the products developed within our facility will soon be available ‘on the market’, if approved by the regulators, after the pivotal clinical trial next year.”
Farhan Naim, director of research and development at the Royal Free London, said: “The team has worked incredibly hard to deliver products that have huge potential. We are proud to be a research-led trust and our ambition is to continue to innovate in this area.”
Caroline Clarke, group chief executive of the Royal Free London, said: “This ground-breaking work taking place at the trust gives real hope to patients currently being treated and patients of the future. We are proud to be playing such a significant role in cell manufacturing.”
First picture: Juliana Dias Alves Pinto, head of GMP CAR-T production at the Royal Free Hospital.
Second picture: Dr Owen Bain, co-director of the CCGTT.