University of Chicago Researchers Uncover Potential Breakthrough in Paediatric Cancer Treatment

In a groundbreaking study led by researchers at the University of Chicago, a novel drug molecule targeting RNA modifications has shown promising results in suppressing tumour growth in neuroblastoma, a common paediatric cancer.

Neuroblastoma, which develops in nerve cells outside the brain, poses a significant challenge in paediatric oncology. Despite advancements in treatment, over 40% of patients with high-risk neuroblastoma do not survive. However, a new approach targeting RNA modifications associated with the disease could revolutionise treatment outcomes.

Published in Cell Reports, the study demonstrates that a drug molecule designed to inhibit proteins responsible for adding modifications to RNA transcripts effectively reduced the growth of neuroblastoma cells in laboratory experiments. Furthermore, in mouse models, this treatment led to a significant suppression of neuroblastoma tumour growth, offering hope for a more effective treatment strategy.

Dr. Susan Cohn, Professor of Paediatrics at the University of Chicago and senior author of the study, highlights the urgent need for innovative therapeutic approaches: "High-risk neuroblastoma remains very difficult to cure with current approaches. Survivors often face treatment-related toxicities and long-term health complications. Our research explores a completely different therapeutic strategy targeting RNA-modifying proteins, which could potentially transform neuroblastoma treatment."

The study builds upon advancements in understanding the role of RNA modifications in cancer progression. Collaborating with Dr. Chuan He, a renowned expert in RNA and DNA modification research, the team investigated the effects of targeting N6-methyladenosine (m6A), one of the most common messenger RNA modifications.

Dr. Monica M. Pomaville, lead author of the study and a former paediatric resident at the University of Chicago, underscores the significance of their findings: "Our research demonstrates that inhibiting METTL3, a protein involved in adding m6A modifications to RNA, reduces neuroblastoma cell growth and promotes the differentiation of neurons, a crucial aspect of combating tumour progression."

Moreover, the study explores the potential synergy between METTL3 inhibitors and immunotherapy, a promising avenue for enhancing treatment efficacy. By modifying the composition of immune cells within neuroblastoma tumours, researchers aim to transform "cold" tumours into "hot" ones, thereby improving their response to immunotherapy.

The findings offer a glimmer of hope for paediatric cancer patients and underscore the importance of continued research into innovative treatment modalities. As the team continues their investigations, the prospect of combining RNA modification-targeting drugs with immunotherapy holds promise for transforming the landscape of neuroblastoma treatment.