Hundreds of people with aggressive forms of blood cancer are set to benefit after two personalised immunotherapy (CAR-T) treatments were today (Thursday, 27 April) recommended by NICE for the Cancer Drugs Fund (CDF).
CAR-T therapy is a treatment in which a person’s own immune system cells (T-cells) are altered in a laboratory to attach to and kill cancer cells. The two treatments are both given as a one-off treatment straight into the blood stream.
The treatments could benefit up to 600 people in total each year in England.
Axicabtagene ciloleucel
Axicabtagene ciloleucel (Yescarta, Kite) is recommended in final draft guidance as a CAR-T (chimeric antigen receptor) therapy for adults with diffuse large B‑cell lymphoma (DLBCL) that returns within a year of, or is resistant to, first-line chemoimmunotherapy.
In some cases, it can be treated with a stem cell transplant, where healthy stem cells are collected from the blood or bone marrow before treatment, stored, and then given back to the patient after treatment.
Diffuse large B-cell lymphoma (DLBCL) is an aggressive type of cancer of the lymphatic system. Around 5,500 people are diagnosed with DLBCL each year in England.
The new recommendation makes the CAR-T therapy available after just one therapy, providing quicker access for patients. It is currently available as standard care after two or more systemic therapies.
Clinical trial evidence suggests axicabtagene ciloleucel increases how long people live compared with standard care, extending life by more than three months.
It is estimated that just over 500 people a year in England would be eligible to receive this treatment.
Brexucabtagene autoleucel
Brexucabtagene autoleucel (Tecartus, Kite) is also recommended to go into the CDF in final draft guidance for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people aged 26 and over.
B-cell acute lymphoblastic leukaemia is an aggressive type of blood cancer in which too many immature white blood cells are found in the bone marrow and blood.
Standard treatment includes chemotherapy and immunotherapies. Brexucabtagene autoleucel would be offered as an additional treatment.
Clinical evidence suggests that people having the treatment may live longer and have more time before their disease relapses.
It is estimated that just under 90 people a year in England would be eligible to receive this treatment.
Both treatments are recommended for use in the Cancer Drugs Fund to allow more data to be collected to address uncertainties on how well the treatments work over a longer period and whether the results can be applied more generally to NHS practice.
NICE has now made 55 Cancer Drugs Fund recommendations since 2016 and more than 55,000 patients have already benefited from access to the most promising new cancer drugs through managed access.
Helen Knight, director of medicines evaluation at NICE, said: “We know the devastating impact lymphoma and leukaemia have on people. These innovative new treatment options will help people live longer and improve their quality of life.
“Around 1,000 people in total could benefit from a range of different CAR-T therapies, including these latest treatments, which have all been recommended by NICE in recent months.
“We are committed to constantly learning from data and implementation, so patients can benefit from ground-breaking treatments while more information is gathered which will hopefully lead to them being offered routinely on the NHS in the future.”
Evidence on the two treatments will be collected over the next four years, after which NICE will update its guidance.
The companies have a managed access agreement, including confidential commercial arrangements, which make axicabtagene ciloleucel and brexucabtagene autoleucel available to the NHS with a discount.
Read the final draft guidance for axicabtagene ciloleucel for treating relapsed or refractory diffuse large B-cell lymphoma after one systemic treatment here.
The final draft guidance for brexucabtagene autoleucel for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people 26 years and over is also on the NICE website.