Toxicity and Efficacy of Small Molecule Inhibitor AI-10-49 and Chemotherapy Drugs in the Treatment of Childhood Inv(16) AML
Background
Treatment protocols used in the treatment of pediatric acute myeloid leukemia (AML) are based on chemotherapy drugs, often resulting in side effects due to significant toxicity. In recent years, the emergence of targeted therapy strategies in cancer treatment has enlightened the hope for developing new drugs with higher potency and reduced toxicity in patients.
The Castilla laboratory studies how mutations found in AML cells disrupt the function of normal blood cells and keeps them expanding. The Castilla laboratory helped develop a new molecule, called A-10-49, which eliminates the leukemic cell with a specific mutation while sparing the normal cells toxicity.
Project Goals
The goal is to progress the use of this molecule to a clinical setting. The Castilla Lab is studying if AI-10-49 has a synergistic effect when used together with Ara-C and doxorubicin, drugs used for the treatment of AML patients. If successful, the combined use of these drugs at lower concentrations could reduce toxicity and increase its anti-leukemic effect in the patients. These experiments will provide important clues on the potential use of AI-10-49 for the treatment of pediatric AML.
