Indiana University School of Medicine researchers and their collaborators have uncovered molecular insights expected to enhance treatment options for acute myeloid leukemia (AML), a rare and severe blood and bone marrow cancer.
With a new chemical compound engineered to overcome drug-resistant gene mutations not targeted by current treatments, the researchers aim to provide a new alternative with longer-lasting effects and improve outcomes for future patients battling this aggressive disease.
In a recent study published in the Journal of Clinical Investigation (JCI), researchers identified inhibitors, or blocking agents, that target FLT3 gene mutations, the most common mutation in acute myeloid leukemia.
FDA-approved drugs like quizartinib and crenolanib are currently used to target FLT3 mutations, but some leukemia patients develop resistance to these treatments. In their latest study, the scientists identified two new inhibitors that effectively target FLT3 mutations, including secondary mutations that were previously drug resistant.
The study’s co-author Herman Sintim, PhD, Richard B. Wetherill Professor of Chemistry and Drug Discovery and Distinguished Professor in Chemistry in the James Tarpo Jr. and Margaret Tarpo Department of Chemistry at Purdue University engineered the patent-pending compound named HSN748 for impending clinical use.
Sintim said the strong preliminary data made possible by collaboration between Purdue and IU subsequently attracted funding from the National Institutes of Health. This support enables the evaluation of HSN748 across different cancer models and enhances its prospects for further development funding. These next steps will be primarily managed by the compound’s license holder KinaRx, an early-stage biotechnology company that aims to develop innovative solutions to cancers that have become resistant to existing drugs and get them into the market.
