Researchers have made a significant breakthrough in the fight against one of the most common forms of acute lymphoblastic leukaemia. The study, conducted by WEHI and the Peter MacCallum Cancer Centre, has identified two new proteins essential for the development of this aggressive disease. By targeting these proteins, the research team was able to kill leukaemia cells in the lab and halt the growth of cancer cells.
This discovery could lead to enhanced treatment options in the future, with plans already underway to develop a clinical trial based on these findings. Acute lymphoblastic leukaemia is a form of blood cancer that appears suddenly and grows quickly, making it notoriously difficult to treat, especially in adults. Approximately 50% of Australian patients relapse after the first round of chemotherapy and become resistant to further treatments.
Associate Professor Ashley Ng, a corresponding author on the paper, emphasized the urgent need for new therapeutics, stating, "About 135,000 people live with a blood cancer or blood disorder in Australia, with 16 people dying every day from the disease." The incidence of blood cancer has increased by 47% in the past decade, highlighting the importance of this research.
The study focused on the proteins ERG and c-MYC, which are master regulators of several important pathways within the leukaemia cell. Researchers discovered that targeting a key gene, POL I, essential for ribosome biogenesis, could kill leukaemia cells and stop their growth. This approach was tested in pre-clinical and human tissue models, showing promising results.
Dr. Kira Behrens, the first author of the study, explained, "By targeting POL I with inhibitors, we were able to kill leukaemia cells and stop their growth in our pre-clinical and human tissue models." This discovery opens up a new pathway and potential drug target for treating leukaemia.
The research also involved collaboration with Associate Professor Elaine Sanij from the St. Vincent’s Institute of Medical Research, whose work focuses on targeting POL I and ribosome biogenesis in cancer therapy. The findings suggest that a subset of aggressive acute lymphoblastic leukaemia is dependent on ribosome production, making it sensitive to POL I inhibitors.
Professor Rick Pearson, former Associate Director of Laboratory Research at Peter Mac, highlighted the potential of this research to translate into successful clinical trials, offering a new treatment option for patients with acute lymphoblastic leukaemia. The study was supported by various organizations, including the German Research Foundation, the National Health and Medical Research Council, and the Leukaemia Foundation.
This research represents a significant step forward in understanding and treating aggressive forms of leukaemia, offering hope to patients and their families for more effective treatments in the future.