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Ground-breaking cellular therapy set to revolutionise treatment for children with leukaemia

08/15/2018

Professor Ajay Vora discusses how advances in cellular therapy are set to revolutionise treatment for children with acute lymphoblastic leukaemia (ALL). 

Acute lymphoblastic leukemia (also called ALL or acute lymphocytic leukemia) is a cancer of the blood and bone marrow.  Leukemia is one of the ten most common malignancies in Saudi Arabia, Kuwait and UAE, and a major form of paediatric cancer. In the UAE, a recent study found a high rate of ALL (32%) among males.

According to Professor Vora, childhood leukaemia outcomes have improved in the past four decades, with 90% of children with ALL surviving. These improvements in outcomes have also translated for children in the Middle East as well.

“There are a number of new pioneering treatments being developed at GOSH to treat leukaemia using immune cells, and some of these are being tested in clinical trials”, explains Prof. Vora. “These treatments are experimental and are only considered when routine chemotherapy treatments haven’t worked. A Kuwaiti patient will soon be the first international patient to receive CAR-T cell therapy at GOSH.”

CAR-T cell therapy, a type of cellular therapy currently being trialled at GOSH to treat ALL, involves collecting a patient’s own white blood cells (or immune cells) and modifying them so they can attack specific cancers.

“One major issue, especially in infants, is the difficulty in collecting enough of the right type of immune cells (T cells)”, Prof. Vora explains. “For such cases, new approaches are being tested that use T cells from a healthy donor but are modified using ‘gene-editing’ so they don’t need to be matched to the patient. This means the cells are ‘universal’ and can be produced in advance and stored frozen, ready to be used in multiple patients.” 

The gene-edited T cells (called UCART19) are given to patients immediately after a course of chemotherapy and antibody therapy. They are expected to circulate around the body and fight any leukaemia carrying the CD19 marker. Responses are assessed after one month, and if remission had been achieved, the patient will proceed to have a bone marrow transplant. At that stage the cells are removed and do not persist long-term.

This innovative treatment is now available to international patients at GOSH. Not only this, but the team are opening another two academic studies to add to their current portfolio of three. There are also on going collaborations with industrial partners to develop new CAR-T cell products for acute myeloid leukaemia (AML) and T-cell acute lymphoblastic leukemia (T-ALL). 

“Cellular therapy has the potential to revolutionise the treatment of leukaemia in children. Currently, it provides a chance to treat  patients who have failed to respond to other treatment. If further research confirms preliminary data on efficacy, in future, it will provide a less toxic alternative to haemopoietic stem cell transplantation,” Prof. Vora concludes.

GOSH, who treats 1,500 children from the Middle East every year, has treated 271 haematology patients between April – Dec 2017.

About Professor Ajay Vora

Professor Ajay Vora is a Consultant Paediatric Haematologist at Great Ormond Street Hospital. His daily practice involves the care of children with a variety of malignant and non-malignant blood disorders. He has a special interest in childhood leukaemia and cord blood transplantation. He is chair of the UK NCRI childhood leukaemia sub-group and I-BFM ALL committee. He is chief investigator of the current and previous front line childhood and infant ALL clinical trials and is involved with several research projects in this area. Until March 2017, he was programme director of the Trent Regional Paediatric Haemopoeitic Stem Cell transplant service.

He has taken a leading role in clinical trials in childhood Acute Lymphoblastic Leukaemia and haemopoietic stem cell transplantation nationally and internationally. He is Chief Investigator of several national and international phase 3 clinical trials and Principal Investigator of over 20 trials. He has 150 peer reviewed publications and am editor of a book on childhood ALL.

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