The discovery is related to epigenetic therapy—a treatment in which drugs do not directly alter DNA, but instead affect the mechanisms that control the switching on and off of genes in a cell.
The study focused on certain aggressive forms of acute leukemia. In these diseases, a genetic error disrupts the normal control of gene function, causing oncogenes to remain constantly active.
Epigenetic-based drugs already exist and are used in clinical trials. However, until now, it was unclear why they work and how to make the treatment's effect more sustainable. Now, scientists have discovered that targeting the epigenetic proteins menin or DOT1L can lead to long-term "silencing" of cancer-causing genes in leukemia cells.
According to lead study author Daniel Neville, the "memory" of tumor cells associated with the DOT1L protein plays a key role. Anti-menin drugs erase this memory, and cancer cells continue to die even after treatment is stopped.
The results are planned to be tested in clinical trials, which Monash University and The Alfred Hospital plan to begin later this year. Doctors anticipate that this could potentially shorten the duration of therapy and reduce severe side effects, while maintaining or enhancing the treatment's effectiveness.
