CAR T-cell therapy is a type of immunotherapy. You might also hear it called a type of adoptive cell transfer.
CAR T-cell therapy is a very complex and specialist treatment. With this treatment, a specialist collects and makes a small change to your T cells. These then target the cancer cells.
It is available as a possible treatment for some children with leukaemia and some adults with lymphoma. People with other types of cancer might have it as part of a clinical trial.
To understand CAR T-cell therapy more, it helps to understand what T cells do.
White blood cells called lymphocytes play an important part in fighting infection and diseases, including cancer. There are different types of lymphocytes. T cells are one type.
T cells move around the body to find and destroy defective cells. When you come into contact with a new infection or disease, the body makes T cells to fight that specific infection or disease. It then keeps some in reserve so that if you come across the infection again your body can recognise it and attack it immediately.
CAR T-cell therapy
T cells are good at fighting infection. But it can be difficult for them to tell the difference between a cancer cell and a normal cell. So the cancer cells can hide away and not be recognised.
Scientists are trying to find ways to get T cells to recognise cancer cells. One possible way to do this might be CAR T-cell therapy.
With this treatment, you have a sample of T cells taken from your blood. Your medical team do this through a process called apheresis.
First you have a tube put into a vein in each arm. One tube removes the blood and passes it into an apheresis machine. The machine separates the different parts of the blood. For CAR T-cell therapy, the machine takes out your T cells. The rest of your blood cells and normal blood fluid go back into your body through the tube in your other arm.
In the lab, they change the T cells. You might hear this called genetically engineering the T cell. The T cell is now a CAR T-cell. CAR stands for chimeric antigen receptor. These CAR T-cells are designed to recognise and target a specific protein on the cancer cells.
These changed T cells grow and multiply in the lab. Once there are enough cells you have a drip containing these cells back into your bloodstream. The aim is for the CAR T-cells to then recognise and attack the cancer cells.
The changes they make in the lab mean that they can stay in your body for long periods of time, recognising and attacking the specific cancer cells. Researchers are still looking into how long they might stay in the body.
There are different types of CAR T-cell therapy made by different companies. Examples include:
- tisagenlecleucel (Kymriah)
- axicabtagene ciloleucel (Yescarta)
Which cancer types?
The first trials of this treatment mostly looked at blood cancers such as acute lymphoblastic leukaemia (ALL) and chronic lymphocytic leukaemia (CLL). These cancers develop from another type of lymphocyte called B cells.
Researchers have produced CAR T-cells for these cancers to target a protein on the surface of the cell called CD19. This protein is on the surface of nearly all B cells.
In the UK, there are trials looking at CAR T-cell therapy for different types of cancer, including:
- myeloma - a cancer of the plasma cells in the bone marrow
- neuroblastoma – a rare cancer that develops from particular types of nerve cells called neuroblasts, it mostly affects children under the age of 5 years old
CAR T-cell therapy is now available through the Cancer Drugs Fund in England for some children with leukaemia and some adults with lymphoma. This followed decisions by NICE in December 2018 and January 2019.
Only a small number of centres in England can provide this specialist treatment. There are 7 centres for adults and 9 centres for children and young people.
Children and young people
CAR T-cell is recommended for those up to the age of 25 who have a type of leukaemia called B cell ALL. It will be used in one of the following situations:
- newly diagnosed children or young people whose leukaemia hasn’t gone away with 2 cycles of treatment
- their disease has come back (relapsed) following a stem cell or bone marrow transplant
- their disease has relapsed twice or more
- children and young people whose leukaemia had gone away with treatment, but it’s come back, and chemotherapy isn’t working now
- their disease has come back once but they can’t have a stem cell transplant because either they aren’t well enough, or they don’t have a donor
For adults in England it will also be available for one of the following types of lymphoma:
- diffuse large B cell lymphoma
- primary mediastinal B cell lymphoma
It is for those adults whose lymphoma has continued to grow or relapsed following at least 2 treatments.
So this treatment is only suitable for a small number of children and young people, and around 200 adults each year. It is not used as a treatment outside of clinical trials for other types of cancer in children or adults.
Wales and Northern Ireland
Wales is setting up a centre so that they can offer this treatment. Meanwhile, suitable patients from Wales and Northern Ireland will be able to travel to hospitals in England to have this treatment.
In February 2019, the Scottish Medicines Consortium (SMC) approved CAR T-cell therapy for children and young people with B cell ALL. Children and young people may be able to travel to England for treatment.
This treatment has not been approved in Scotland for adults with lymphoma. But if your specialist thinks this is a suitable treatment for you, they can apply for funding on an individual basis.
This is a new treatment, so doctors might not know about all the possible side effects yet. Two known side effects include:
• cytokine-release syndrome
• no B cells or fewer B cells after treatment with CAR T-cells that target CD19
Cytokine release syndrome
Cytokines are group of proteins in the body that play an important part in boosting the immune system. CAR T-cell therapy stimulates the immune system to make large amounts of cytokines. It causes symptoms such as:
- fever (high temperature)
- dizziness due to low blood pressure
- difficulty breathing
This syndrome might happen in the first week of treatment. You can have treatment to reverse the syndrome.
Fewer B cells in some types of CAR T-cell therapy
In treatment for some leukaemias, CAR T-cells are designed to recognise a protein called CD 19. CD 19 is found on the surface of most B cells. B cells are a type of white blood cell and, like T cells, they play an important part in fighting infection.
CAR T-cell therapy that targets the CD 19 protein also destroys the B cells. It kills normal B cells as well as cancerous B cells. This either reduces the number of B cells or destroys them all. This makes it difficult for you to fight infections.
You might need treatment for this side effect. This treatment is called immunoglobulin therapy. It contains antibodies to help you fight infection.
Research is still trying to find out if this is a temporary or long term side effect.
Cancer Research UK Science blog
To read more about the science behind this treatment read the science blogs.