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Genetic research

Novel Strategy for Treating Aggressive Blood Cancers by Targeting 'Junk DNA'

Strategic Insights

— 1 min read

Researchers at King's College London have discovered a new approach to treating certain aggressive blood cancers by targeting a previously overlooked part of the genome, known as 'junk DNA', using existing cancer drugs. The study, published in the journal 'Blood', focused on myelodysplastic syndrome (MDS) and chronic lymphocytic leukemia (CLL), two cancers frequently driven by mutations in the ASXL1 and EZH2 genes. This novel strategy could offer new hope for patients with limited treatment options.

Key Takeaways:

  • The study targeted transposable elements (TEs), repetitive DNA sequences that make up nearly half the human genome, which were previously considered genetic 'junk'.
  • The research team discovered that in cancer cells with ASXL1 or EZH2 mutations, TEs become highly active, causing genomic instability and DNA damage.
  • The vulnerability of cancer cells to TE-induced DNA damage creates an opportunity for intervention using PARP inhibitors, drugs already approved for other cancers, such as those involving BRCA mutations.
  • The PARP inhibitors work by blocking DNA repair pathways, preventing cancer cells from repairing breaks caused by mobile genetic elements, leading to an accumulation of lethal damage and cell death.
  • The study used reverse transcriptase inhibitors to suppress TE activity, confirming that the drugs were acting specifically through this TE-mediated pathway.
  • The findings may extend to other cancers with similar genetic defects, potentially expanding the use of PARP inhibitors and offering a new therapeutic angle for malignancies currently lacking effective treatments.
  • The research team worked with myelodysplastic syndrome (MDS) and chronic lymphocytic leukemia (CLL), two cancers frequently driven by mutations in the ASXL1 and EZH2 genes.

Statistics:

  • The human genome is comprised of nearly 50% transposable elements (TEs), sometimes referred to as 'junk DNA' (As a rough estimate by geneticists, it is calculated that the genome is roughly 50% identical, 25% repeat elements, and 25% unique single-copy DNA, however, it was calculated at 22-34% identical and 66-78% multi-copy, and 6-10% single copy [The human genome. 2005]).
  • PARP inhibitors have been approved for use in certain cancers, such as those involving BRCA mutations [AstraZeneca press release January, 2020].
  • The study published in the journal 'Blood' concentrated on myelodysplastic syndrome (MDS) and chronic lymphocytic leukemia (CLL).
  • The study suggested that the findings may extend to other cancers with similar genetic defects.

Sources:

  • Oman News Agency, 15 Sep 2025
  • King's College London
  • Journal 'Blood'

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