RNA-based Therapy Shows Promise against Aggressive Childhood Brain Tumors


Researchers at Johns Hopkins Kimmel Cancer Center have found a potential breakthrough in treating aggressive childhood brain tumors by targeting a non-encoding stretch of RNA. The study, published in Cell Reports, focused on group 3 medulloblastoma, the most aggressive and challenging type of brain cancer in children. Led by senior author Ranjan Perera, Ph.D., the team demonstrated a novel approach that effectively shrinks tumors in mice.

Medulloblastoma is the most prevalent malignant brain cancer among children, with group 3 medulloblastoma being particularly difficult to treat and often fatal. With a lack of targeted therapies currently available, the research by Perera and his colleagues offers a promising new direction in addressing this devastating disease in children.

The study targeted long noncoding RNA molecules known as lnc-RNAs, which play a role in driving the expression of genes responsible for cancer growth. By blocking lnc-HLX-2-7, a specific noncoding RNA that promotes tumor growth in group 3 medulloblastoma, the researchers were able to inhibit the cascade of cancer-gene expression, leading to a significant reduction in tumor size in the mice.

The team developed an innovative intravenous therapy using antisense oligo nucleotides, which can bind to lnc-HLX-2-7 and prevent it from promoting cancerous gene expression. By combining this therapy with cisplatin, a chemotherapy drug commonly used in medulloblastoma treatment, the researchers observed a further reduction in tumor size and an extension in the survival of the mice.

Collaborating with Johns Hopkins neurosurgeons, Perera and his team plan to advance their research to human studies to evaluate the safety and effectiveness of the therapy. The findings provide valuable insights into the role of lnc-HLX-2-7 in promoting tumor growth and offer new possibilities for targeted therapies in treating aggressive childhood brain tumors.

Dr. Charles Eberhart, a study co-author and researcher at the Kimmel Cancer Center, emphasized the importance of understanding the mechanisms behind tumor growth, particularly the elevated levels of the MYC gene in these tumors. The study’s identification of the link between lnc-HLX-2-7 and MYC opens up new avenues for potential therapeutic interventions in combating group 3 medulloblastoma.

As research continues to uncover the complexities of childhood brain tumors, innovative approaches like RNA-based therapies show promise in revolutionizing treatment strategies and improving outcomes for young patients facing aggressive cancers.

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