Breakthrough discovery unlocks new path in treatments for leukaemia

treatments for leukaemia
© shutterstock/SL Tharidu

A groundbreaking discovery by researchers at Université libre de Bruxelles may help innovate new treatments for leukaemia.

Led by François Fuks, the study has uncovered a previously unknown role of a gene that has mutated in over 50% of leukaemia patients. The discovery may help develop advanced treatments for leukaemia.

The current landscape of leukaemia treatment

Leukaemia, a type of blood cancer affecting diverse demographic groups, sees around 320,000 new cases diagnosed annually in Europe.

Notably, a third of diagnosed cancers in children belong to this category. While chemotherapy stands as the primary treatment, the exact origins often remain elusive, shrouded in molecular and cellular mysteries.

Detecting leukaemia early and devising effective treatments pose significant challenges in the realm of oncology.

Leveraging mRNA

The spotlight on messenger RNA (mRNA) has recently intensified, notably in the context of COVID-19 vaccines.

In this latest development, researchers from Université libre de Bruxelles (ULB) and the Bordet Institute at Brussels University Hospital (HUB) are forging an innovative pathway: exploring novel anti-cancer therapies utilising the intricate mRNA alphabet, known as RNA epigenetics.

The chemical structure of RNA, akin to DNA with its four well-known letters (A, U, G, C), harbours additional letters, such as m5C or methylation of mRNA.

This m5C plays a pivotal role in gene regulation through interactions with proteins termed ‘readers.’ However, the specifics of these m5C readers and their involvement in cancer have remained elusive.

The research has identified a new RNA reader, SRSF2, shedding light on its pivotal role in leukaemia development.

Notably, the SRSF2 gene exhibits high mutation rates and is implicated in up to 50% of certain leukaemia types. The team’s research underscores how the mutated SRSF2 inhibits its function of regulating mRNA by disrupting its ability to read m5C in RNA.

Advancing cancer therapy

By analysing hundreds of samples from leukaemia patients, the researchers identified a subgroup with significantly reduced survival prospects due to the compromised m5C reading ability of SRSF2.

These findings not only deepen our understanding of leukaemia onset but also herald a paradigm shift in its diagnosis and treatment centred on RNA epigenetics.

Practically, this breakthrough opens doors for specific diagnoses pinpointing patients with poor prognoses tied to the compromised ‘m5C reader’ function of SRSF2.

Furthermore, the prospects of a new therapeutic approach loom on the horizon, potentially involving the development of inhibitors to restore SRSF2’s ability to read m5C accurately—a faculty diminished in patients harbouring the SRSF2 mutation.

This landmark discovery not only unravels the intricate mechanisms underlying leukaemia but also holds promise for tailored treatments, offering renewed hope in the battle against this complex disease.

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