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She gave the first child a pioneering therapy for CAR-T



Eleven years old Yuvan, with his parents Vinay and SapnaImage rights
SAKRA

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Yuvan, 11, with his parents, Vinay and Sapna

Eleven-year-old became the first NHS patient to receive therapy that uses her own body cells to fight cancer.

Yuvan Thakkar, who has the form of leukemia, received personalized treatment at Great Ormond Street Hospital (GOSH) in London after a failure of conventional cancer treatment.

CAR-T involves the removal of immune cells and their treatment in the laboratory to identify tumor cells.

Previously, it was only available as part of a clinical research experiment.

CAR-T, called Kymriah, costs £ 282,000 per patient, but the NHS has negotiated a lower price with Novartis.

And money comes from the Cancer Drugs Fund, which is preparing for quick access to the most promising treatments.

Kymriah is approved for the treatment of patients over 25 years of age with acute lymphoblastic leukemia (ALL) in B-cells who have failed other treatments.

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Novartis

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Kymriah is a personal immunotherapy against cancer

NHS England announced in September that it would fund treatment less than 10 days after being granted a European license.

Yuvan, of Watford, was diagnosed with leukemia in 2014. He received chemotherapy and then underwent bone marrow transplantation, but relapse after both drugs.

Last Hope & # 39;

Juana's parents, Sapna, and Vinay, said, "When Yuvan was diagnosed, it was the most heartfelt news we ever received.

"We have tried to remain hopeful, because, according to them, leukemia in children has 90% of the treatment rate, but unfortunately his illness is relapsed.

"This new therapy is our last hope."

Yuvan said, "I really hope to improve soon enough to visit Lego House in Denmark.

"I love Lego and build a big Bugatti model when I'm in the hospital."

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SAKRA

Image caption

Yuvan gives time in the hospital to make Lego models

Acute lymphoblastic leukemia affects approximately 600 people per year, mostly children. Most are treated with conventional treatments but approximately 10% relapse.

In November it was announced that GOSH, together with Royal Manchester Children's Hospital and Newcastle upon Tyne NHS Foundation Trust, will treat children with this rare leukemia.

It is expected that up to 30 patients will be treated per year.

CAR-T is a truly personalized form of cancer treatment.

First, the patient has the blood removed and the white blood cells are separated from each other, the rest of the blood is returned to the patient.

T cells, a particular type of immune cells, are then sent to laboratories in the United States where a harmless virus is inserted into them.

These genes cause T-cells to add to their surface a hook, known as the chimeric antigen receptor (CAR).

These developed CAR-T cells – programmed to recognize and destroy the cancer cells of the patient – multiply in huge amounts and then infiltrate back into the patient.

Possible side effects

Tuvan removed the immune cells in November, and created T-cells were infused last week.

Dr. Sara Ghorashian, a consultant for child hematology at GOSH and Dr. Yuvan, said: "We are very pleased to be able to offer patients such as Yuvan another chance for cure.

"Though it will be a while before the outcome of this powerful new therapy is known, treatment has shown very promising results in clinical trials and we hope it will help."

Children's Hospital Royal Manchester has also begun treating its first patient with personalized Kymriah immunotherapy.

An 11-year-old girl from Liverpool expects her modified CAR-T cells to infuse within a few weeks.

In a clinical trial, 75 patients who failed all other treatments were halved in one full year after a year.

However, some serious side effects have been reported; some patients require intensive care due to cytokine release syndrome.

This can cause fever, low blood pressure and difficulty in breathing, but it can be treated in most patients.

CAR-T for adults

NHS England also agreed to fund additional CAR-T therapy for adults with lymphoma.

Treatment, Yescarta, would cost nearly £ 300,000 per patient, but Gilead Sciences has agreed to a lower price.

Up to 200 patients per year will be eligible.

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