Landmark cellular therapy to revolutionize treatment for children with leukemia

Kuwaiti will soon be the first patient to receive CAR-T cell therapy

Professor Ajay Vora

KUWAIT: This International Childhood Cancer Day, pediatric hematologist expert, Professor Ajay Vora, from Great Ormond Street Hospital in London (GOSH) discusses how advances in cellular therapy are set to revolutionize treatment for children with acute lymphoblastic leukemia (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 pediatric cancer. In the UAE, a recent study found a high rate of ALL (32%) among males.

According to Professor Vora, childhood leukemia 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 leukemia 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 trialed 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.

‘Gene-editing’
“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 leukemia 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 currently available at GOSH as part of its research trials, and will become available in the near future to patients from across the world once the research phase is over. “Cellular therapy has the potential to revolutionize the treatment of leukemia 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 hematology patients between April – Dec 2017.


This article was published on 13/02/2018