Immunotherapy after stem cell transplant in children with high-risk acute lymphoblastic leukaemia

01 December 2008

Prof Persis Amrolia & Dr Nick Goulden, UCL Institute of Child Health

Chemotherapy is used as the first defence against childhood acute lymphoblastic leukaemia but children who do not respond must undergo stem cell transplant (SCT), a high risk procedure that does not always succeed. This project is pioneering a new technique to treat children who relapse after SCT, using reprogrammed immune cells from the stem cell donor to help fight the leukaemia.

Amount of grant: £348,658  |  Date of award: December 2008

Professor Persis Amrolia and Dr Nicholas Goulden, UCL Institute of Child Health.


When a child is first diagnosed with leukaemia, doctors immediately start an aggressive chemotherapy regime to try to kill the leukaemia cells.

For some children this is enough. But others do not respond so well and have to undergo stem cell transplantation (SCT) – a high-risk procedure which is currently the last resort.

For some children even this will not work; some leukaemia cells remain - the cells multiply once more and the child will relapse. When a child relapses after SCT, there is nothing more that doctors can do.

The problem is that the leukaemia cells have become resistant to the chemotherapy drugs, so we need to find another way to kill them.

The research project

The research team is using immune cells from the stem cell donor to help fight the leukaemia.

This is truly groundbreaking work; it is a world first...if successful, it will give new hope to children...

They have found a way to genetically ‘reprogramme’ these cells so that they seek out and destroy the remaining leukaemia cells.

When infused into the child, the reprogrammed immune cells search the child’s system for leukaemia cells, latch on to them and kill them.

Key to this process is that minimal damage is caused to the child’s body – by this stage of treatment the child will be extremely weak.

The team has shown that this technique can work in the laboratory.

Our funding is enabling them to take it to the hospital wards in the hope that they can save those children who have run out of other treatment options.

This is truly groundbreaking work; it is a world first. It will be the first time that gene therapy has been used to treat children with leukaemia. If successful it will give new hope to children for whom every other treatment option has failed.


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