TARGETING PROTEINS IN ORDER TO BLOCK THE GROWTH OF TUMORS AND THE PROGRESSION OF LEUKEMIA FOR CHILDREN IN RELAPSE
This trial was cofinanced by the 2016 Children without Cancer race.
A Phase II study evaluating the activity of Pegylated recombinant human Arginase (BCT-100) in Relapsed/refractory Cancers of childhood.
About Leukemias
Childhood leukemia is similar to that in adults, but it can, however, present certain particularities. Acute leukemia is a type of cancer characterized by the dissemination of cancerous cells originating from bone marrow stem cells. These cells multiply uncontrollably, invade the bone marrow, and enter the bloodstream, potentially spreading throughout the body. This proliferation impairs the production of all blood cells (white and red blood cells, platelets). As of 2023, this disease is the most common cancer in children, accounting for about 30% of pediatric cancer cases.
The two main types in children are acute lymphoblastic leukemia (80% of cases) and acute myeloid leukemia. The exact causes of leukemia in children are not always clearly defined, but they are believed to be related to genetic, environmental factors, or a combination of certain events occurring at the level of bone marrow stem cells.
Learn more about leukemias: link
About the PARC Project
PARC is a phase II trial focusing on leukemias and other solid tumors in relapsed children. It aims to test the effectiveness of a new treatment that blocks the growth of tumors and leukemias by targeting proteins. BCT-100 is an arginase that metabolizes an amino acid, arginine, depriving malignant cells of it. This trial introduces children to the new and innovative field of tumor metabolism inhibition.
Initially, researchers evaluated the optimal dose of BCT-100 to use in children and some adults (49 patients) and then assessed its effectiveness in leukemias, neuroblastomas, sarcomas, and high-grade gliomas (the effectiveness is measured after 8 weeks of treatment). Overall, this treatment was very well tolerated by patients; however, no effectiveness was detected for all patients and their different types of cancers.
This project was funded through donations collected during the 2016 Enfants sans Cancer race.
Trial Summary
Sponsor: University of Birmingham
Principal Investigator: Dr. Francis Mussai
Program Duration: 2018 – 2022 (2 years of recruitment, 2 years of follow-up)
Phase II Recruitment (ongoing): 49 patients have been recruited
Countries Involved: United Kingdom, Australia, Netherlands
Funding by Imagine for Margo: €130,000
Doctor Gudrun Schleiermacher speaks about the program:
