Great Ormond Street Hospital Children’s Charity funding helps develop new CAR T-cell therapy for leukaemia with less dangerous side effects

Great Ormond Street Hospital Children’s Charity (GOSH Charity) has helped to fund a clinical trial to investigate a novel CAR T-cell therapy, designed to target cancer cells more quickly and cause less side effects, that has shown very promising results for children with previously incurable acute lymphoblastic leukaemia (ALL).In CAR-T therapy immune cells called T-cells are modified in the lab to hunt down and kill cancer cells. The CAR T therapy used in this study was designed to more specifically target cancer cells with the hope that this would lead to fewer dangerous side effects.

The CARPALL trial, which was led by Professor Persis Amrolia and Dr Sara Ghorashian at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health received funding from GOSH Charity in 2015 and began testing this new kind of CAR T-cell therapy in June 2016 in children and young adults with relapsed ALL. This new CAR T therapy was developed in collaboration with the UCL Cancer Institute.

This particular novel therapy was used to treat 14 patients with relapsed ALL at GOSH, Manchester Children’s Hospital and University College Hospital, London.

Austin, 10, from South Shields in the North East of England was diagnosed with acute lymphoblastic leukaemia at the age of two. By the age of eight he had dealt with three relapses and exhausted all traditional treatment options including radiotherapy, chemotherapy and two bone marrow transplants.

Austin’s dad, Scott, says: “When we had our meeting with Persis Amrolia and found out about this trial it was life changing. I remember that day so clearly. He was talking about potential hope, and suddenly we could see a future both for Austin and also for medicine.

“We owe so much to Persis and his team, but we are also acutely aware that without the early funding that he and his team received from GOSH Charity, Children with Cancer and the JP Moulton Foundation, to go into this research, this might not have been possible.
“It is difficult to express our gratitude to Persis and the research team; what they were able to offer was something that we never imagined possible during the darkest of times. Hopefully in the future more families can be given hope and a chance at life using similar ground-breaking approaches to treatment.”
Published in Nature Medicine today, the results show that after receiving the CAR T treatment, 12 out of 14 patients with otherwise incurable ALL were free of the disease for at least three months and five patients remain leukaemia-free. Patients also experienced less of a harmful side effect known as cytokine release syndrome (CRS).

ALL affects around 400 children a year in the UK and, while most patients are curable with standard treatments like chemotherapy and a bone marrow transplant, in some patients the disease comes back (relapses) despite maximal treatment. CAR T-cells have shown great promise for relapsed ALL - the most common cause of cancer death in children in the UK

The study enabled UK patients to have access to this ground-breaking new treatment before a commercial CAR-T cell therapy, Kymriah, became available on the NHS.

Kiki Syrad, Director of Grants and Impact at Great Ormond Street Hospital Children’s Charity says: “It’s amazing to the difference GOSH Charity’s investments in childhood cancer can make. Seeing research come to fruition in this way, to benefit seriously ill children from across the UK is amazing.

“It’s an exciting time for GOSH Charity as we continue to invest in pioneering cancer research and help GOSH provide the very best environment to treat children with cancer. We couldn’t do any of this without the generosity of our supporters.”

Professor Amrolia, study Chief Investigator and National Institute for Health Research (NIHR) Research Professor at UCL GOS ICH, said: “CAR T therapy is a fantastic example of using the power of the immune system to specifically target cancer cells. While it doesn’t work for everyone, it can offer hope for those children who have run out of all other options. The side effects of CAR T therapies can be severe, so we hope that this new technology can reduce the risk for patients.”

The CARPALL study was also funded by Children with Cancer, the JP Moulton Foundation and the work was underpinned by the NIHR Biomedical Research Centres at GOSH and University College London Hospitals NHS Foundation Trust (UCLH). The study is part of UCL’s pioneering CAR T-cell programme which currently has nine Phase I studies, including a related study looking at the use of the same therapy to treat adult patients. Other research in the centre aims to extend CAR T cell therapy to other cancers.

For further information, interviews or additional quotes and images please contact the GOSH Press Office:

Ruth Maurice, Senior Press Officer at Ruth.Maurice@gosh.org or call 02038413125.

Notes to editors

1. Nature Medicine paper: Enhanced CAR-T cell expansion and prolonged persistence in pediatric patients with all treated with a low-affinity CD19 CAR https://www.nature.com/articles/s41591-019-0549-5  

About Great Ormond Street Hospital for Children NHS Foundation Trust 

Founded in 1852, Great Ormond Street Hospital is one of the world’s leading children’s hospitals with the broadest range of dedicated, children’s healthcare specialists under one roof in the UK. With more than 252,000 outpatient and 43,000 inpatient visits every year, the hospital’s pioneering research and treatment gives hope to children from across the UK with the rarest, most complex and often life-threatening conditions. As an international centre of excellence in child healthcare, our patients and families are central to everything we do – from the moment they come through the door and for as long as they need us. Visit www.gosh.nhs.uk

About Great Ormond Street Hospital Children’s Charity

Great Ormond Street Hospital Children’s Charity needs to raise money to support the hospital to give seriously ill children, the best chance for life. The charity funds research into pioneering new treatments for children, provides the most up to date medical equipment, funds support services for children and their families and supports the essential rebuilding and refurbishment of the hospital. You can help us to provide world class care for our patients and families. For more information visit www.gosh.org  

About the UCL Great Ormond Street Institute of Child Health (ICH) 

The UCL Great Ormond Street Institute of Child Health (ICH) is part of the Faculty of Population Health Sciences within the School of Life and Medical Sciences at University College London. Together with its clinical partner Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH), it forms the UK's only paediatric National Institute for Health Research (NIHR) Biomedical Research Centre and has the largest concentration of children's health research in Europe. For more information visit www.ucl.ac.uk/child-health

About UCL

UCL was founded in 1826. We were the first English university established after Oxford and Cambridge, the first to open up university education to those previously excluded from it, and the first to provide systematic teaching of law, architecture and medicine. We are among the world's top universities, as reflected by performance in a range of international rankings and tables, and are committed to changing the world for the better. Our community of over 41,500 students from 150 countries and over 12,500 staff pursues academic excellence, breaks boundaries and makes a positive impact on real world problems.

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About the JP Moulton Foundation

The Foundation has funded over 100 clinical trials including four in cancer immunotherapy. Address c/o J P Moulton Charitable Foundation, 10 Buckingham Street, London WC2N 6DF.

About the NIHR

This work was supported by the NIHR Biomedical Research Centres (BRCs) at GOSH and UCLH.

The National Institute for Health Research (NIHR) is the nation's largest funder of health and care research. The NIHR:

• Funds, supports and delivers high quality research that benefits the NHS, public health and social care
• Engages and involves patients, carers and the public in order to improve the reach, quality and impact of research
• Attracts, trains and supports the best researchers to tackle the complex health and care challenges of the future
• Invests in world-class infrastructure and a skilled delivery workforce to translate discoveries into improved treatments and services
• Partners with other public funders, charities and industry to maximise the value of research to patients and the economy

The NIHR was established in 2006 to improve the health and wealth of the nation through research, and is funded by the Department of Health and Social Care. In addition to its national role, the NIHR supports applied health research for the direct and primary benefit of people in low- and middle-income countries, using UK aid from the UK government.