Two breakthroughs in CAR-T therapy

It's been a historic week for blood cancer treatments with two significant developments in the use of CAR-T therapies. Headlines were made yesterday when it was announced that the first child with ALL had successfully received CAR-T therapy on the NHS.

CAR-T therapies are a ground-breaking treatment that take samples of immune cells from the patient and genetically modify them in a laboratory to recognise, seek out and kill cancer cells, before putting them back into the patient’s blood system.

11-year-old Yuvan Thakkar, from Watford, was diagnosed with leukaemia in 2014 but relapsed after standard chemotherapy and then again after a stem cell transplant.

Yuvan, who is being treated at Great Ormond Street Hospital in London had his engineered T-cells infused last week.

Yuvan's mum and dad, Sapna and Vinay, said in a statement: "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.”

NHS England initially approved the use of Kymriah in September 2018 for young people with ALL who do not respond to chemotherapy or a stem cell transplant. Kymriah is available through the Cancer Drugs Fund (CDF), which enables the most promising treatments to be made available to patients while further evidence on their effectiveness is gathered.  Read about more about NHS England’s approval of Kymriah for childhood ALL.

Our thoughts are with the Thakkar family and we wish Yuvan all the best in his treatment.

The National Institute for Health and Care Excellence (NICE) announced today that adults with diffuse large B-cell lymphoma (DLBCL) will now also be able to access Kymriah through the CDF. 

Clinical trials have shown that Kymriah could provide a long-term cure for four out of 10 people with DLBCL who don’t respond to chemotherapy. The outlook for patients treated with further intensive chemotherapy is extremely poor.     

NICE estimates that around 140 patients with DLBCL could be eligible for Kymriah each year. Bloodwise was one of the patient organisations consulted in NICE’s appraisal process on the treatment.

Dr Alasdair Rankin, Director of Research and Patient Experience at Bloodwise, said: “CAR-T therapy is the most promising breakthrough in blood cancer treatment of the past decade, with the potential to be used much more widely in the future.

"Ensuring access to CAR-T therapies gives these patients the real chance of long-term survival when all other treatments have failed."

NICE approved the use of another CAR-T therapy, called Yescarta, for the treatment of DBCL in December 2018. Read more about NICE’s approval of Yescarta.

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