A severe type of leukemia characteristic of childhood (although it can also appear in adulthood), acute T-cell lymphoblastic leukemia (ALL-T), highly resistant to chemotherapy and with a high relapse rate -25% in children and 50% in adults- could have a new treatment, thanks to research that has identified two related enzymes -phosphoinositide-3-kinase (PI3K ), gamma and delta- that play an important role in the development of this aggressive disease.

The researchers who conducted the study, who belong to the Medical Center of Columbia University, also observed that a double inhibitor of PI3K gamma / delta, called CAL-130, is able to prolong survival significantly in a mouse model of the pathology.

The drug, an experimental PI3K enzyme inhibitor, is able to prolong survival significantly in a mouse model of acute T-cell lymphoblastic leukemia

T-cell acute lymphoblastic leukemia, as the name suggests, is a cancer that affects T cells (a type of white blood cell) during its development, and causes mutations in DNA, resulting in the generation of T cells anomalies that proliferate rapidly and invade vital organs, whose function is altered. If left untreated, the disease is deadly.

The aim of the research was to analyze the role played by the PI3K gamma and delta enzymes in the T-ALL, and to verify if the CAL-130 had the ability to influence the progression of this pathology. The scientists found that these enzymes are indeed essential for the development of T-ALL, as well as for the survival of cancer cells. In addition, they were able to verify -using for this a mouse model of the disease-, which when administering CAL-130 significantly decreased the number of T cells, and increased the average survival of the treated animals, which happened to be 45 days, against 7.5 days of those not treated.

Although CAL-130 is in an experimental phase and has not been tested in human patients, the effects it has on blood samples taken from patients with T-ALL have been analyzed, and the result is encouraging since it prevents the cells from Carcinogens proliferate and promote apoptosis - a process of cell death.

Thomas Diacovo, professor of Pediatrics and Pathology and Cell Biology at Columbia University, who has led the research, explains that if the new medicine demonstrates its effectiveness in humans, and is able to selectively combat the activity of these enzymes in the T cells of the tumors that affect people, could contribute to less use of traditional chemotherapy, thus reducing the toxic side effects that these drugs have in younger patients, and that make them more prone to suffer complications and develop secondary cancers.

2018 Demystifying Medicine: Is Childhood Leukemia Curable? (November 2019).