NUNZIUM

News That Matters

12.12.2022
THEME: HEALTH

Gene editing depleted acute leukemia from a girl and may have been able to cure her

Leukaemia is cancer that affects the blood and bone marrow, where blood cells are produced. It is characterized by the uncontrolled growth of abnormal white blood cells, which can crowd out healthy blood cells and prevent them from functioning correctly, leading to symptoms such as fatigue, weakness, infections, and easy bruising or bleeding. Several different types of leukaemia are classified based on how quickly the disease progresses and the type of blood cells affected. Treatment often involves chemotherapy, radiation therapy, or bone marrow transplant and may include targeted drug therapies or immunotherapy. The prognosis varies depending on the type of leukaemia and the stage at which it is diagnosed. Still, many people with the disease can be successfully treated and go on to lead healthy, everyday lives. Alyssa, 13, was diagnosed with T-cell acute leukaemia in May last year. Chemotherapy and bone-marrow transplant were unable to rid it from her body. A team of doctors at Great Ormond Street, led by Prof Waseem Qasim, used a technology called base editing - invented only six years ago - on Alyssa with outstanding results. They used the most advanced methods to build her a new living drug based on a personalized T-cell capable of hunting down and killing Alyssa's cancerous T-cells. Base editing allows scientists to zoom into a specific part of the genetic code and alter the molecular structure of just one base, converting it into another and changing the genetic instructions. After a month of treatment, she was in remission and given a second transplant to rebuild her immune system. As she was left vulnerable to infection, she spent 16 weeks in the hospital and couldn't see her brother, who was still going to school, in case he brought germs. There were worries after the three-month check-up found signs of cancer again. But her two most recent investigations have been positive. Alyssa is the first patient to be treated with this technology. This outstanding result undoubtedly suggests that science has found a way to reduce leukaemia mortality. However, more statistics are needed to determine if the method can be called a "cure", which would be close to a miracle compared to the alternatives. Genetic manipulation, a high-speed moving area of science, confirms its true potential - which experts say goes across many incurable diseases.