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First patient receives groundbreaking new cancer therapy
4 February 2019
An 11-year-old boy has become the first patient to receive a ‘revolutionary’ new cancer therapy, which has been developed with the aid of scientists at UCL.
Yuvan, from Watford, has relapsed acute lymphoblastic leukaemia (ALL) and has now received the ground-breaking treatment, known as Kymriah.
Kymriah is the Tisagenlecleucel form of CAR T therapy, which modifies a patient’s immune system cells, known as T cells, to attack the cancer cells.
UCL has a number of growing CAR T-cell programme at both the UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) and the UCL Cancer Institute, which includes expertise, ranging from protein and T-cell engineering through to clinical grade cell manufacture.
While it will be some time before the results of this treatment are known, the first patient has received the therapy and is currently recovering at Great Ormond Street Hospital (GOSH). This treatment has been tested in clinical trials in the US where it has been shown that approximately 50–62% of patients survive without leukaemia for 12 months or more.
Previously, CAR T therapy was only available to patients as part of research trials. In September last year, following a landmark deal between NHS England and the manufacturer, the therapy is being offered to eligible NHS patients with relapsed ALL. Research has shown the treatments are effective for patients with particularly aggressive cancers after standard treatments have failed.
A new treatment for ALL
ALL affects around 600 people per year, most of whom are children. Although treatments have improved steadily, approximately 10% of patients still relapse.
The availability of the treatment follows the success of the CAR T therapy research programme at GOSH and UCL GOS ICH, which is supported by the National Institute for Health Research (NIHR) GOSH Biomedical Research Centre, GOSH Children’s Charity and many other funders.
Dr Sara Ghorashian, Consultant in Paediatric Haematology at GOSH, and Honorary Senior Lecture at UCL GOSH ICH, is actively researching new targets for CAR T-cell therapy as well as investigating what characteristics of CAR T cells support best disease responses.
“As one of Yuvan’s doctors, we are so pleased to be able to offer patients like Yuvan another chance to be cured. While it will be a while before the outcome of this powerful new therapy is known, the treatment has shown very promising results in clinical trials and we are hopeful that it will help,” she said.
Professor Persis Amrolia (UCL GOS ICH), led one of the first European studies in CAR T-cell therapy for children with leukaemia in 2013 and continues to be a leader in the field.
“Immunotherapy with CAR T cells offers has shown remarkable results in patients with otherwise untreatable leukaemia and lymphoma and I’m delighted this revolutionary new personalised treatment is now being made available through the NHS,” he said.
”However, we’re just at the beginning and many challenges remain, particularly in extending this approach to other cancers. UCL has been at the forefront of development of CAR T-cell therapy within Europe with 7 active clinical trials and a pipeline of basic and translational work in the field.”
Yuvan
Yuvan, a keen cricket fan, was diagnosed with leukaemia in 2014. He unfortunately relapsed after standard treatments and underwent a bone marrow transplant last year. But sadly, in October, Yuvan relapsed again.
Yuvan’s parents Sapna and Vinay said: “When Yuvan was diagnosed it was the most heart-breaking news we had ever received. We tried to stay hopeful as they say leukaemia in children has 90% cure rate, but sadly, his illness relapsed. This new therapy is our last hope. It means a rebirth to us if this treatment works and we hope it really does. We are so glad that we at least have this new option now. If he had relapsed a year ago it would have been a different story”.
Yuvan said, “I really hope I get better soon so I can visit Lego House in Denmark. I love Lego and am building a big model Bugatti while I’m in hospital.”
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Credit: GOSH
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