Boy, 11, becomes first NHS patient to receive revolutionary new cancer drug
Yuvan Thakkar has a form of leukaemia that has failed to respond to treatment.
An 11-year-old boy has become the first NHS patient to receive a pioneering new cancer therapy that uses the body’s own immune system to fight the disease.
Yuvan Thakkar, from Watford, received the drug Kymriah, a type of immunotherapy called CAR-T cell therapy, at Great Ormond Street Hospital (GOSH) in London last week.
Yuvan has acute lymphoblastic leukaemia (ALL), which can be cured in around 90% of children who undergo conventional chemotherapy.
However, Yuvan’s cancer has failed to respond to two rounds of chemotherapy and a bone marrow transplant, meaning he has few options left.
Now, experts at GOSH have given him Kymriah (also known as (tisagenlecleucel), which was approved for use on the NHS after a deal was struck between NHS England and the drugs firm Novartis to offer it at a reduced price.
Previously, CAR-T cell therapy was only available to patients as part of research trials.
The treatment involves taking the patient’s own T cells (a type of immune cell) from their blood and genetically engineering them in the laboratory so they recognise and fight cancer cells.
Millions of these genetically engineered CAR-T cells are grown in the laboratory and then given back to the patient via an infusion into their bloodstream.
Each dose of Kymriah is a customised treatment created using these own T-cells.
In Yuvan’s case, his T cells were first extracted in November last year, and shipped to laboratories in Rotterdam and Texas, where they underwent the complex editing procedure.
Research has shown that CAR-T can lead to a cure or extended survival for a high number of patients, although not everyone benefits.
In clinical trials in the US, around 50% to 62% of patients survive without leukaemia for 12 months or more.
Yuvan, a keen cricket fan, was diagnosed with leukaemia in 2014.
Despite treatment and a bone marrow transplant last year, he was still found to still have leukaemia.
Yuvan’s parents, his mother Sapna, and father Vinay, said in a statement: “When Yuvan was diagnosed it was the most heartbreaking 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.”
Professor Charles Swanton, Cancer Research UK’s chief clinician, said: “It’s fantastic news that children and young people like Yuvan can receive CAR-T cell therapy on the NHS, giving another option when their cancer returns.
“This is an incredibly complex treatment to give, Yuvan’s cells were processed in both Europe and the US, and needed collaboration across borders to get the T-cell infusion back to London so he could be treated.
“The UK was one of the first countries in the world to approve CAR-T cell therapy, showing that the NHS remains at the forefront of innovation when it comes to new cancer treatments.
“More research is underway so that we can identify who’s most likely to benefit from CAR-T cell therapy, as well as how to further refine the technique, so as many as possible can benefit.”
Side-effects from CAR-T can occur and patients need to be kept in isolation following treatment.
CAR-T can cost around £280,000 per patient, although NHS England has brought down this price during negotiations.
As well as GOSH, the Royal Manchester Children’s Hospital and Newcastle-upon-Tyne Hospitals NHS Foundation Trust will offer the treatment to young people with leukaemia.
Another type of CAR-T cell therapy, called Yescarta, has been approved on the NHS for adults with aggressive types of lymphoma.
Dr Sara Ghorashian, consultant in paediatric haematology at GOSH and Yuvan’s doctor, said: “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.”