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Research conducted by RCSI has found that combining venetoclax, a medication approved for leukaemia, with another drug called 5-azacytidine has shown promising results in treating multiple myeloma (MM), a type of blood cancer. This discovery offers new treatment options for the currently incurable disease, which affects around 400 people in Ireland each year. Despite advances in treatment, innovative strategies are essential for patients who are resistant to standard care.

The study, published in Haematologica, aimed to identify complementary drugs that could enhance the effectiveness of venetoclax in MM treatment. While venetoclax had previously been tested for MM, it was only found to be effective for a small proportion of patients. However, combining it with 5-azacytidine significantly increased its efficacy across multiple MM cell lines, suggesting a broader potential patient population could benefit from this new combination therapy.

Professor Tríona Ní Chonghaile, Associate Professor and research lead at RCSI, emphasized the significance of identifying more effective treatment options for MM. By re-evaluating existing treatments like venetoclax in new contexts and combining them with other drugs, the researchers were able to enhance efficacy across a wide range of patient samples. This highlights the importance of exploring different approaches to expand treatment potential for diseases like MM.

Professor Siobhán Glavey, Chair of the RCSI Department of Pathology and Clinician Scientist at Beaumont RCSI Cancer Centre, expressed optimism in the discovery of this new drug combination and its potential for MM treatment. The next step is to test the efficacy and safety of the combination in a clinical trial setting to bring this promising treatment strategy closer to patients. The combination of venetoclax and 5-azacytidine was found to be effective even in patient samples from different stages of cancer, including those who had previously received chemotherapy.

The research also delved into the mechanism of action of the two drugs when used together, demonstrating their effectiveness in patient samples across various stages of MM. Collaborating with experts from the Department of Haematology at Beaumont Hospital, the Department of Medical Oncology at Dana-Farber Cancer Institute in Boston, and the Department of Medicine/Haematology at the University of Galway, the study was able to shed light on a promising new treatment avenue for MM patients.

This study was supported by funding from the Leukemia Research Foundation, Breakthrough Cancer Research, and AbbVie, underscoring the importance of continued investment in research to uncover innovative therapies for difficult-to-treat diseases like MM. The findings offer hope for patients with MM, providing a potential new treatment option that could enhance outcomes and quality of life for those affected by this challenging blood cancer. Further clinical trials will be essential to validate the efficacy and safety of this new drug combination, moving closer to the goal of developing more effective treatment strategies for MM.

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