Childhood cancer immunotherapy using blood cells from umbilical cord blood

26 June 2014

Professor Waseem Qasim, UCL Institute of Child Health

Immunotherapy is an important new approach in the treatment of cancer. Some immunotherapy approaches rely on the collection of patients’ own immune cells, which are genetically engineered before being returned to the patient to fight the cancer. Unfortunately, following chemotherapy, many children have very low numbers of the necessary immune cells, making this approach unfeasible. Professor Qasim is pioneering an approach using immune cells taken from donated umbilical cord blood as an alternative to using patients’ own cells.

Amount of grant: £255,156 | Date of award: June 2014


Immunotherapy is becoming an increasingly important tool in the treatment of different cancers, including childhood cancers. It exploits the fact that cancer cells often have molecules on their surface, which can be detected by the immune system. Immunotherapy is used to harness the immune system into attacking the tumour cells by using these molecules as targets.

Immunotherapy approaches often involve the use of patients’ own immune cells, collected from patients and engineered in the laboratory to recognise and kill tumour cells before being returned to the patient.

Unfortunately, however, many children have very low numbers of the key immune cells after chemotherapy, and the cells may not perform well. This makes the treatment unfeasible for them.

To address this problem, Professor Qasim is pioneering an approach using immune cells (T cells) taken from donated umbilical cord blood. Cord blood T cells are immunologically naïve and multiply readily. Experience suggests that they can have a powerful effect, with less risk of a serious adverse immune reaction known as "graft versus host disease" than if cells were taken from other donors.

During this project the team will refine the technology and methodology to generate the engineered cord blood T cells as proof of concept. The data generated will be used for regulatory submissions ahead of full scale production of a cord T cell bank for clinical studies in children.

About the research team

There is no doubt that the research team is qualified to carry out the proposed project and translate its results into clinical testing. The research centre is a world-leading institution in cell and gene therapy.
External reviewer
Professor Waseem Qasim is leading the development of T cell therapies at Great Ormond Street Hospital (GOSH) with a group of laboratory and clinical investigators.

GOSH has developed a core of expertise for the collection, gene modification and application of experimental treatments.

Dr Martin Pule is a world-leading expert in the design and application of anti-cancer receptors and is leading a multi-centre European study in adult patients.

Professor John Anderson is an expert in children’s cancer and is running trials of cell therapy for neuroblastoma, brain and bone cancers.

Professor Paul Veys is head of the Bone Marrow Transplant unit at GOSH and is an authority on cord blood transplantation.

Together, this team has a unique combination of skills and experience that will enable the successful development of new therapies, through from the laboratory to clinical treatments for children – ‘from bench to bedside’. 

What difference will this project make?

Within the tenure of the project the team expect to be in a position to be able to offer cell therapy to children who would be ineligible for treatment reliant on using their own immune cells. In the first instance, this will use well-matched cord blood T cells that will be engineered ‘on demand’ for individual patients. Subsequently, they aim to be able to provide ‘ready to go’ cells that can be given to any patient when needed.

Read more: Treating childhood cancer


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