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Today, the Scottish Medicines Consortium (SMC) has approved the use of Tisagenlecleucel-T (brand name Kymriah) to be funded for use on the NHS in Scotland. It will be available for B-cell acute lymphoblastic leukaemia (B-ALL) patients aged 25 or less, who have refractory disease or have relapsed following a transplant or two or more other therapies. Kymriah is a brand of chimeric antigen receptor (CAR) T-cell therapy. It is manufactured by Novartis and was the first CAR-T therapy to be approved in the UK when it was approved for use in England in November 2018.
CAR-T therapy, also called adoptive T cell therapy, is a type of immunotherapy, meaning parts of the immune system are used as a treatment. Immunotherapy usually uses parts of the immune system made in other animals or in a laboratory, such as antibodies. CAR-T cell therapy is brand new and innovative, involving the harvesting of patient’s own immune cells from their body to be used in the treatment. The cells are taken from the body, edited to be more effective at fighting the cancerous cells, then returned to the patient’s blood to start fighting the cells. The process tailors the treatment to each patient and therefore the cost can be high.
ALL is a type of leukaemia that affects your lymphocytes, which is the collective name for the blood cells called B and T cells. These cells go through many stages of development before they become cells that can work properly in your immune system. ALL happens because the cells have not developed properly, so your bone marrow and blood fills with immature cells that cannot protect you. ALL can affect either B or T cells, but B-ALL is more common in adults. CAR-T cell therapy, as mentioned above, requires working T cells from the patient’s body, which is why it has been approved for B-ALL patients only (i.e. only their B cells are developing incorrectly).
There can be some severe side effects, but the treatment is approved here for use where B-ALL patients have tried other therapies, which may have less severe side effects, but they have not worked. Despite the severe side effects, the SMC approved Kymriah due to the high success rate of the treatment. In the clinical trial data that was submitted to the SMC, 81% of B-ALL patients achieved remission of their disease three months after treatment with Kymriah.
At the time of approval in England, the Head of NHS England described Kymriah as a “true game changer”. Our Patient Advocacy Director, Zack Pemberton-Whiteley, said of today’s SMC decision: “CAR-T therapy is promising, with curative potential for acute lymphoblastic leukaemia patients who have exhausted most alternative treatments. We are glad that SMC has chosen to fund Kymriah so patients are able to access this important new treatment as soon as possible and wherever they live in the UK.”