“Junk” DNA Holds Promise in Fight Against Drug-Resistant Blood Cancers
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New research suggests previously dismissed non-coding DNA could be a key target in developing treatments for aggressive blood cancers.
For decades, vast stretches of DNA were written off as “junk” – sequences that didn’t code for proteins and were presumed to have no biological function. Now, a groundbreaking study reveals these once-ignored regions may hold the key to combating stubborn, drug-resistant blood cancers like myelodysplastic syndrome and chronic lymphocytic leukemia. scientists are discovering that this non-coding DNA plays a critical role in gene regulation, and, surprisingly, can be exploited to disrupt cancer cell growth.
The Unexpected Role of “Junk” DNA
The genome is far more complex than previously understood. Scientists now recognize that non-coding DNA isn’t useless at all, but rather a crucial regulator of cellular processes. A specific category of this non-coding DNA, known as transposable elements (TEs), are sequences capable of moving around the genome, inserting themselves into new locations.
An international team lead by researchers at King’s College London (KCL) has discovered that these tes become activated in persistent blood cancers, contributing to the disease’s progression. “This discovery offers new hope for patients wiht 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,” explains a biologist from KCL.
Targeting Cancer’s Vulnerability
The research, published in Blood in July 2025, focuses on how mutations in the genes ASXL1 and EXH2 – commonly found in myelodysplastic syndrome and chronic lymphocytic leukemia – trigger this TE activation. These mutations disrupt protein production, leading to uncontrolled cell growth and genomic instability.
The team found that the damage caused by ASXL1 and EXH2 mutations leads to the duplication and spread of “junk” DNA throughout cancer cells. This activity stresses the cancer cells, making them increasingly reliant on poly (ADP-ribose) polymerase repair proteins (PARPs) to survive. Crucially, drugs that suppress parps proved effective in killing the cancer cells in laboratory settings, while largely sparing healthy cells.
[. Placeholder for a visual representation of the PARP inhibitor mechanism.]
“This study sets the stage for a novel and broader approach of creating synthetic lethality for human cancers,” the researchers wrote in their published paper. Synthetic lethality refers to a situation where a combination of genetic alterations is lethal to a cell, while either alteration alone is not.
Beyond Blood cancers: A Wider Impact?
The researchers are optimistic that these findings extend beyond the two blood cancers initially studied. PARP blockers are already used to treat other forms of cancer, suggesting a broader applicability of this approach. The exact mechanisms may differ, but the principle of targeting DNA repair pathways in cancer cells remains consistent.
This research builds on a growing body of evidence highlighting the importance of TEs. Recent studies have shown these DNA regions contribute to the body’s immune defenses, influence brain responses to fear, and even play a role in preventing interbreeding between species. “Transposable elements that account for almost half of the human genome… have been reported in recent years to be reactivated in driving disease progress, and multiple cellular processes including gene expression, DNA damage, and immune responses,” the researchers note.
The re-evaluation of “junk” DNA is revolutionizing our understanding of the genome and opening up exciting new avenues for cancer treatment. This research underscores the importance of continued examination into the non-coding regions of our DNA,which may hold the key to unlocking further medical breakthroughs.
