GOSH to be first in UK to offer groundbreaking personalised therapy for international children with cancer

Dr Sara Ghorashian, Consultant in Paediatric Haematology
Dr Sara Ghorashian, Consultant in Paediatric Haematology

Great Ormond Street Hospital for Children (GOSH) in London is to become the first hospital in the UK to offer a new pioneering cancer therapy to patients. Patients with B-cell acute lymphoblastic leukaemia (ALL) will now be able to receive the new personalised treatment, known as CAR-T therapy, with first patients beginning treatment at GOSH this week. This is the first treatment of its kind to become available to UK and international patients in the UK outside of clinical trials.

CAR-T therapies are specifically tailored for individual patients and work by harnessing the patient’s own immune system to fight cancer. In a complex manufacturing process, immune cells are taken from a patient’s blood and reprogrammed to specifically to target and kill cancer cells.  

ALL is a severe form of leukaemia that affects around 600 people per year, most of whom are children between the age of 2 and 5. Although the outlook for children with ALL has dramatically improved over the last decade, 10-15% of patients still do not respond to standard treatments. The new therapy has been shown to be effective in treating patients with particularly aggressive or relapsed cancers where other treatments have failed. 

Dr Peter Steer, chief executive of GOSH said: “I am delighted GOSH will be providing this pioneering treatment. As a leader in the field for developing personalised medicines for children and young people, GOSH has a proven track record in delivering this type of advanced cancer treatment. We aim to help every child we see fulfil their potential; offering CAR-T therapy does exactly that.” 

Dr Sara Ghorashian, Consultant in Paediatric Haematology at GOSH said: CAR-T therapies represent a new era of cancer treatments and it’s fantastic that GOSH will soon be able to provide this cutting-edge therapy to children for whom other treatment has not worked. These groundbreaking treatments offer the real chance of a long-term cure so this is a major step forward for the families affected by this devastating condition.”  

Since 2012, GOSH has treated over 25 patients from all over the UK with CAR-T therapies as part of several different clinical trials. GOSH led one of the first clinical studies of CAR-T cell therapy for paediatric leukaemia in Europe which was launched in 2013. In 2015, we successfully treated the first patient in the world with gene-edited “universal” CAR-T cells. We continue to innovate in the field of personalised medicine and are currently exploring the use of “next generation” CAR-T cells to further reduce the chance of relapse. This underlying research was supported by the National Institute for Health Research (NIHR) GOSH Biomedical Research Centre, GOSH Charity and a range of other funders. 

GOSH’s commission to provide the drug follows a deal between manufacturer Novartis and NHS England, announced by Simon Stevens in September and a site accreditation process by NHS England. Tisagenlecleucel (also known as Kymriah) is licenced to treat patients up to 25 years old with relapsed or refractory B-cell ALL.

For more information, or to refer a patient please click here. 

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