0
0
In a monumental breakthrough, researchers at Rockefeller University have pioneered a groundbreaking therapy for fibrolamellar hepatocellular carcinoma (FLC), a rare and often fatal liver cancer. Their innovative approach utilizes small interfering RNAs (siRNAs) to target the oncogene responsible for FLC, offering new hope for patients battling this devastating disease.
Published in Molecular Therapy, the study marks the first time siRNAs have been employed to combat FLC, representing a significant leap forward in cancer treatment. Led by first author Christoph Neumayer and overseen by Sanford M. Simon, the research team harnessed the power of siRNAs to silence the oncogene within FLC cells, preventing the production of disease-causing proteins and halting tumor formation.
FLC, triggered by the fusion of two genes, DNAJB1 and PRKACA, poses a formidable challenge due to its aggressive nature and limited treatment options. However, the Rockefeller team’s groundbreaking discovery offers a glimmer of hope for patients battling this rare form of liver cancer.
“We have unlocked a promising avenue for treating FLC and potentially other types of cancer,” remarked Neumayer. “Our findings demonstrate the immense potential of siRNA therapies in revolutionizing cancer treatment.”
The fusion of DNAJB1 and PRKACA genes, known as DNAJB1::PRKACA, drives the development of FLC, but the precise mechanisms underlying its oncogenic activity remained elusive until now. Building on their earlier breakthrough in identifying the fusion as the root cause of FLC, the researchers devised a multifaceted strategy to combat the disease, including repurposing existing drugs and developing novel therapeutic approaches.
Central to their approach was the targeted delivery of siRNAs into FLC cells, facilitated by a liver cell-specific receptor called ASGR1. By attaching custom siRNAs to ASGR1’s ligand, the researchers achieved precise delivery of therapeutic molecules into tumor cells, effectively silencing the oncogene and inhibiting tumor growth.
“Our study demonstrates the remarkable specificity and efficacy of siRNA therapy in combating FLC,” Neumayer explained. “By precisely targeting the fusion oncogene, we were able to halt tumor progression without causing harm to healthy cells.”
Importantly, the researchers observed no liver toxicity in animal models, underscoring the safety and tolerability of the siRNA therapy. Furthermore, the therapy’s high degree of specificity ensures minimal off-target effects, making it a promising candidate for the treatment of other cancers as well.
“Our findings lay the foundation for a new era of precision medicine in cancer therapy,” Neumayer concluded. “With further research and development, siRNA therapies have the potential to revolutionize cancer treatment and offer hope to patients worldwide.”
