Dream team! Polish researchers working on new method for treating Acute Lymphoblastic Leukaemia

Acute lymphoblastic leukemia (ALL) is a neoplastic disease originating from the B or T precursor lymphocytes formed in the bone marrow. It is considered a rare disease. ALL can occur in all age groups, but is most often diagnosed in children. Although the incidence of this disease decreases with age, the prognosis also worsens: the 5-year survival rate for young adults is 54 percent, and for people over 60 it is only 13 percent.

Director of the Institute of Haematology and Transfusion Medicine and project leader Professor Ewa Lech-Marańda said: “This is a very important project for our research team. It is based on the results of preclinical studies carried out by IHiT (Institute of Haematology and Transfusion Medicine - PAP), which showed that inhibition of the activity of certain enzymes (kinases) in leukaemic cells restores their sensitivity to standard treatment. 

“Therefore, we want to use innovative drugs from the group of kinase inhibitors, registered and used in other oncological indications, to overcome resistance to standard treatment based on glucocorticosteroids in those patients who, due to the advancement of the disease process, do not qualify for intensive treatment of relapses.”

The research project, which is financed by the Medical Research Agency, is in the preparation phase for the launch of research, which will be carried out in two parts, including a population study and a phase 1 clinical trial.

Dr. Eliza Głodkowska-Mrówka, MD, Ph.D. assistant professor at the Institute of Haematology and Transfusion Medicine and research director of the project said: “We plan to include all adult ALL patients who come to our hospital or cooperating units and consent to participate in the study. In the blood samples of these patients, we will search for biomarkers that can provide information about the patient's sensitivity to targeted treatment, the choice of optimal therapy and prognosis, which could be used in the routine diagnosis of the disease in the future.”

She added that the study offers an opportunity to develop a test that will personalise treatment for this type of leukaemia and may facilitate the selection of an effective therapy. This part of the methodology will be developed together with the Dutch company PamGene. Work on this part of the project will start in the first quarter of 2021.

In turn, the first phase clinical trial will include selected patients with recurrent ALL who do not qualify for intensive chemotherapy.

Dr. Głodkowska-Mrówka said: “This group of patients will receive a novel experimental therapy based on kinase inhibitors (trametinib, gilteritinib or everolimus) in combination with dexamethasone. The aim of the study will be to check the safety and effectiveness of this form of treatment and determine the pharmacokinetics of the investigated drugs. In the longer term, the results obtained in this trial will form the basis for another clinical trial, this time in the population of patients with kinase activation-dependent ALL, the aim of which will be to eliminate residual disease and improve treatment outcomes.”

The research project is planned for 5.5 years and several hundred people will take part in it. The research team consists of IHiT scientists, including haematologists, diagnosticians, bioinformatics experts, molecular biologists, pathomorphologists, radiologists, as well as coordinators, pharmacists and nursing staff.

Professor Lech-Marańda said: "We assume that the results of our research will significantly broaden our understanding of the biology of ALL cells, thanks to which in the near future we will be able to apply personalized therapies in the first line of treatment for this disease, which will improve treatment outcomes giving patients a better chance of living without leukaemia.”

(PAP)

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