Antibody development for safer stem cell transplant in acute myeloid leukaemia

Stem cell transplant is often used to treat children with leukaemia, but the pre-transplant treatment used to replace bone marrow can have side-effects. This project is pioneering a way of using specially designed antibodies to create space for donor stem cells. If successful, this approach should make it possible to carry out transplants in a much safer way.

Our funding is helping to develop new approaches to stem cell transplant, which is often used to treat children with leukaemia. The aim is to pioneer the use of specially designed antibodies to create space for donor stem cells.

Project Details

  • Project Title

    Development of an anti c-kit antibody to improve outcomes after stem cell transplantation for acute myeloid leukaemia

  • Lead Researcher

    Professor Persis Amrolia

  • Research Centre

    UCL Institute of Child Health

  • City & Institution Postcode

    London, WC1N 1EH

  • Start Date

    12 June 2014

  • Duration

    3 years

  • Grant Amount


Professor Persis Amrolia in the lab


Stem cell transplant is often used in the treatment of children with certain types of acute leukaemia. As part of the preparation for transplant, young people are given an intensive treatment that creates space for the new stem cells from the donor.

However, this treatment can have side-effects and sometimes causes long term health issues. In this project, Professor Amrolia is pioneering a new approach to preparing children for their transplant which avoids the need for intensive chemotherapy. He is developing a new immune medicine – an antibody – that targets a protein called c-kit. This revolutionary antibody will only go to work on harmful cells, an approach that means it could cause far fewer problems outside the bone marrow.

What difference will this project make?

This project will provide the evidence needed to develop an antibody that can be used on children with certain forms of leukaemia. This will then enable the first clinical trials – these will aim to show how effective the combination of this new antibody and gentler chemotherapy conditioning can be.

If successful, this new approach would represent a major breakthrough for young people with these types of acute leukaemia, as it would help to prevent relapse and reduce the short and long-term problems relating to the use of intensive chemotherapy.

In addition, the antibodies developed by Professor Amrolia and his team could be linked to drugs or labelled radioactively. This would allow delivery of larger doses directly to the bone marrow in patients at high risk of relapse, and this would mean that the chance of the leukaemia coming back would be greatly reduced.

About the Research Team

Professor Amrolia is Professor of Transplantation Immunology and UCL Institute of Child Health and Honorary Consultant in Bone Marrow Transplant at Great Ormond Street Hospital. He has worked for 15 years on the development of gentle methods of transplantation, and has a track record in the translation of scientific work into clinical practice.

He is collaborating with Professor Kerry Chester at UCL Cancer Institute, an expert in the field of antibody engineering, who will provide expertise in the development of the antibody. They will be joined by Professor Dominique Bonnet from the London Research Institute, who will lead pre-clinical testing.