A New Purpose: Heart Failure Drug Shows Promise in Treating Leukemia


A repurposed drug, known as proscillaridin A, has been found to show potential in fighting the MYC gene mutation in patients with leukemia.

A study published in theJournal of Experimental and Clinical Cancer Research has found thata cardiotonic, known as proscillaridin A, primarily used to treat heart failure or cardiac arrhythmia is showing promise in treating patients with leukemia.

In the study, investigators observed proscillaridin A’s anticancer and epigenetic properties in juvenile leukemia wherein patients expressed the MYC gene, which, when subjected to mutations or overexpression, induces uncontrolled cell proliferation. Investigators found that the molecule attacks leukemia stem cells.

There are currently no effective, approved treatments to target the MYC gene alteration in leukemias. In order to evaluate the accuracy and efficacy of this technique, investigators utilize many techniques including those in molecular biology, next-generation sequencing, and pharmacology. As a result, the investigators observed that the molecule preferentially attacks leukemic stem cells, which drive the spread of the disease.

Using proscillaridin A to treat leukemia is a major advancement in drug repositioning, or using drugs designed for certain therapeutic indications for other disease. According to the study authors, proscillaridin A drugs have passed critical toxicology, safety, and pharmacokinetics testing and can be fast tracked for use in clinical trials.

“Each cancer is unique, and to increase the chance of survival, precision medicine is a promising way forward by developing patient- specific therapeutic strategies,” Noël J.M. Raynal, researcher at CHU Sainte-Justine and professor at Université de Montréal. "It is therefore essential to analyze the different characteristics of each cancer in genomic, epigenetic and proteomic terms in order to identify optimal therapies. Research on drug repositioning opens a new path toward innovative therapeutic options in the treatment of cancer."

While survival rates for pediatric leukemia have been improving, two-thirds of patients have long-term adverse effects from treatments that are high in toxicity, including neurological and metabolic disorders, and in some cases, secondary cancer. The repurposing of proscilaridin A to treat pediatric leukemia is step forward in making treatment less toxic and improving patient quality of life.

“In the medium term, we home to complete the preclinical characterization of this drug to eventually initiate clinical trials,” said Elodie M. Da Costa, the first author of the study. “Our ultimate goal is to identify more specific and less toxic therapeutic strategies for children with leukemia characterized by the MYC gene in order to improve survival rates and quality of life.”


  1. Heart failure drug proscillaridin A targets MYC overexpressing leukemia through global loss of lysine acetylation. BMC website. Published June 13, 2019.https://jeccr.biomedcentral.com/articles/10.1186/s13046-019-1242-8. Accessed June 14, 2019.

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