CAR T cells to fight acute myeloid leukaemia
Acute myeloid leukaemia (AML) is an aggressive cancer of the white blood cells. When AML comes back after previous chemotherapy, it can be very difficult to cure. In these situations, children often need a stem cell (bone marrow) transplant. This trial is testing an experimental therapy called BE-CAR33 in young patients aged 6 months to 16 years. BE-CAR33 uses immune cells taken from healthy donors. Scientists use an advanced gene-editing tool called 'base editing' to guide these cells to hunt down a specific flag on leukaemia cells, called CD33, while protecting healthy body tissues. The main goal of this early-stage safety study is to find out whether BE-CAR33 can safely destroy remaining leukaemia cells before a child has their planned transplant. Getting rid of as much leukaemia as possible gives the transplant the best chance of working long term.
At a glance
What is this study about?
Acute myeloid leukaemia (AML) affects the blood and bone marrow. Although standard treatments can clear the cancer, the disease can sometimes return (relapse). When this happens, doctors usually plan a stem cell transplant to replace diseased bone marrow with healthy cells from a donor. However, transplants work best when there is little to no cancer left in the body beforehand.
This clinical trial is testing a new type of treatment known as CAR T-cell therapy. Normal CAR T-cells are taken from the patient's own blood and re-engineered in a lab. In this trial, scientists use 'ready-made' cells from healthy donors. They use a precise genetic technique called 'base editing' to make tiny tweaks to the donor cells' DNA. These changes teach the cells to spot a marker on leukaemia cells called CD33, while stopping them from attacking healthy cells or being rejected too quickly.
The study is taking place at Great Ormond Street Hospital in London and will include up to 10 children and teenagers. Because this is a Phase I trial, the main aim is to test how safe the treatment is, what side effects it causes, and whether it can help clear cancer cells ahead of a planned stem cell transplant.
Key takeaways
- Tests an experimental 'ready-made' donor CAR T-cell treatment (BE-CAR33) for children with relapsed leukaemia.
- Aims to safely clear leukaemia cells before a planned stem cell transplant.
- Open to children aged 6 months to 16 years who test positive for the CD33 cancer marker.
- Requires a hospital stay of about 5 weeks, followed by transplant and ongoing check-ups.
- Led by specialist doctors at Great Ormond Street Hospital in London.
Who may be eligible?
This study is designed for children and young people aged between 6 months and 16 years old who have acute myeloid leukaemia that has returned (relapsed). The leukaemia cells must have the 'CD33' marker on their surface. Participants must already have a suitable donor lined up and be fit enough to undergo a planned stem cell transplant.
Children must weigh at least 6 kg (about 13 lbs) and have a reasonable level of day-to-day energy. Their leukaemia must not have spread uncontrollably to the brain or spinal cord.
Patients cannot join if they have an active, uncontrolled infection (such as a severe bacterial, viral, or fungal infection), severe immune complications from earlier treatments, or if they are taking high doses of steroid medications. Parents and patients must also be willing to take part in long-term health check-ups for up to 15 years.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- The patient is between 6 months and 16 years of age.
- They have acute myeloid leukaemia (AML) that has returned.
- The leukaemia cells have been confirmed as CD33-positive.
- A stem cell donor is already identified for a planned transplant.
- The child weighs at least 6 kg.
- There are no active, uncontrolled severe infections.
What does participation involve?
Taking part begins with screening tests, including blood checks and bone marrow samples, to ensure the trial is safe and suitable for the child. If enrolled, the child will first receive chemotherapy over a few days to prepare the body and make space for the new cells. They will then receive a single dose of BE-CAR33 cells through a drip into a vein.
Children will need to stay in hospital for around 5 weeks so the medical team can watch them closely for any reactions. Around 4 weeks after the BE-CAR33 treatment finishes, the planned stem cell transplant will take place. After leaving hospital, the child will have clinic check-ups every month for the first 3 months, and then every 6 months. Because gene-edited therapies are new, long-term safety check-ins will continue for up to 15 years.
Potential risks and benefits
Locations (1)
- Great Ormond Street HospitalApproximateLondon, England
Common questions
What is CAR T-cell therapy?
It is an immune treatment where T-cells (a type of white blood cell) are modified in a laboratory so they can recognise and destroy specific cancer cells.
What does 'base editing' mean?
Base editing is a very precise form of gene editing. It allows scientists to change single letters of genetic code inside the cell to make it safer and more effective against cancer.
Will my child still need a bone marrow transplant?
Yes. This treatment is designed to clear out as much cancer as possible beforehand, helping to make the planned transplant more successful.
How long will my child need to stay in hospital?
Patients will need an inpatient hospital stay of around 5 weeks for the BE-CAR33 infusion and safety monitoring, followed later by another stay for the transplant itself.
Where does this trial take place?
The study is running at Great Ormond Street Hospital for Children in London, UK.
How to find out more
Faizal Shaikh
Always speak to your GP or specialist before deciding to take part in a study.
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