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Great Ormond Street Hospital offers personalised therapy for children with cancer

Dr Sara Ghorashian, Consultant Paediatric Haematologist at GOSH.

Great Ormond Street Hospital for Children (GOSH) in London became one of the first hospitals in the UK to offer a new pioneering cancer therapy to paediatric patients in 2019. International patients with B-cell acute lymphoblastic leukaemia (ALL) can now receive a new personalised treatment, known as CAR-T therapy.

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.

Tamim, from Saudi Arabia, was one of the first international patients at GOSH to be treated with a CAR-T therapy called Kymriah for his relapsed ALL. “I chose GOSH because Tamim’s condition needed urgent treatment,” explains Tamim’s mum. “This treatment is only available in a limited number of countries and hospitals … the plan was to go to the United States, but we decided to come to GOSH because it would be quicker.”

Dr Sara Ghorashian, Consultant Paediatric Haematologist at GOSH, explains: “Rather than being a pharmaceutical drug, this is a cellular product created by taking a patient’s immune cells and then genetically- engineering them to recognise the patient’s leukaemia.”

When asked if she would recommend this form of treatment, Tamim’s mother responded: “Of course, it was excellent. There are other forms of gene therapy available that we have tried before, and I noticed they all had some effect on Tamim. With this one, he didn’t feel as affected and tired as with the other forms of gene therapy.”

Dr Ghorashian says: “The CAR-T cell service at GOSH is a joint service between haematology and the bone marrow transplantation services and contributed to by consultants from each of these teams.

“We have a team of clinical nurse specialists who help coordinate a patient’s care, and ward nurses. We also link in with the neurology, endocrine and intensive care departments who regularly review our patients when needed and provide specialist support. Finally, we have a team of therapists, including physiotherapists and play therapists, who support patients and their parents throughout the treatment.

Tamim, from Saudi Arabia, was one of the first international patients at GOSH to be treated with a CAR-T therapy called Kymriah.

“Tamim has a very resistant disease and he faced a number of complications,” explains Dr Ghorashian. “He is a remarkable little boy and recently returned home.”

Tamim will return to GOSH for check-ups and the family are hopeful that the treatment is successful.

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.

Want to know more about Great Ormond Street Hospital (GOSH) in London?

We’ve been helping children overcome rare and complex conditions ever since we opened our doors in 1852. Stronger than ever, our team is made up of 300 exceptional and dedicated consultants across 60 specialties. We’re a driving force in medical technology and research so we can provide much needed treatment for children across the world.
Our International and Private Care Service supports over 5,000 children from
80 different countries every year. We have a compassionate and multi-lingual
team to help all our international patients and their families feel at home.

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