New treatments found for tough blood cancers (MDS, CLL & PARP inhibitors)

Medical Xpress / King's College London / Blood ^ | Sept. 11, 2025 | Bernd B. Zeisig et al

[ConservativeMind]

Researchers have identified a new way to treat certain blood cancers using existing drugs, by turning a once-dismissed part of our DNA into a therapeutic target.

The study focused on myelodysplastic syndrome (MDS) and chronic lymphocytic leukemia (CLL). These cancers often have mutations in two key genes, ASXL1 and EZH2, which normally control the activity of other genes by switching them on or off. When these genes are damaged, cells lose control of their ability to create new cells, resulting in abnormal cell growth.

Nearly half of human DNA is made up of repetitive sequences called transposable elements (TEs), once thought to be useless "junk DNA." The researchers found that in cancers with damaged ASXL1 and EZH2 genes, TEs become unusually active. This overactivity stresses the cancer cells and damages their DNA, creating a weakness that can be turned into a treatment opportunity.

PARP inhibitors are drugs already used to treat some other cancers. They usually work by preventing cancer cells from repairing damaged DNA. In this study, they acted differently. When TEs move around in the genome, they cause breaks in the DNA that are however protected by PARP proteins for repair. Blocking PARP prevents this repair, causing damage to build up until the cancer cells die.

To confirm this new mechanism, the researchers used reverse transcriptase inhibitors, which stop TEs from copying themselves. This removed the effect of PARP inhibitors, showing that the drugs were working through a unique TE-related pathway rather than their usual role in BRCA-related cancers.

"This discovery offers new hope for patients with hard-to-treat cancers, by using existing drugs in a completely new way, turning what was once thought to be useless DNA into a powerful target for treatment," says Professor Chi Wai Eric So.

[ConservativeMind]

PARP inhibitors stop DNA repair, helping kill off cancer. However, this was a completely new benefit found against two cancers, myelodysplastic syndrome (MDS) and chronic lymphocytic leukemia (CLL), and their strange use of “Junk DNA” to keep them growing.

These are available today.

[ConservativeMind]

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