Breakthrough Stem Cell Treatment Shows Promise in Reversing Liver Disease

Mortality due to end-stage liver disease ranks as the 12th leading cause of death in the United States. Liver transplantation remains the primary treatment for this condition, but a severe shortage of organ donors highlights the urgent need for alternative therapeutic approaches. The quest for innovative treatments has led researchers at Boston Medical Center and Boston University’s Center for Regenerative Medicine to explore the potential of a groundbreaking stem cell treatment.

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Stem Cell Therapy Using mRNA Technology

The study, published in Cell Stem Cell, unveils a novel stem cell treatment employing mRNA technology encapsulated into lipid  nanoparticles (LNP). This technology, previously successful in the development of COVID-19 vaccines, demonstrates the potential to enhance the natural repair mechanism of the liver. The research identifies a specific receptor on stem cells that can be activated by the ligand protein, vascular endothelial growth factor A (VEGFA). This discovery opens avenues for potential non-invasive treatment options for chronic liver disease.

To comprehend the therapeutic potential, researchers conducted studies on zebrafish and mice liver disease models. In zebrafish subjected to induced liver disease, exposure to VEGFA resulted in a significant increase in newly generated hepatocytes. Mouse studies, inducing acute and chronic liver diseases, demonstrated that VEGFA mRNA-LNP injections induced robust conversion of biliary epithelial cells (BEC) to hepatocytes, showcasing a five-fold increase compared to control-treated mice. Moreover, the treatment exhibited full reversion of steatosis and fibrosis in the chronic model.

Implications for Clinical Care and Future Perspectives

The potential of this stem cell therapy is far-reaching, offering a mechanism for the liver to heal itself, potentially reducing the need for liver transplants. The treatment activates liver stem cells, fostering the proliferation of biliary epithelial cells and generating new, healthy hepatocytes. Notably, the study suggests that VEGFA mRNA-LNP could serve as an alternative treatment for severe acetaminophen intoxication, preventing liver failure and reducing the need for transplantation in cases where conventional interventions prove time-limited. This breakthrough opens avenues for novel treatments, utilizing the liver’s natural repair mechanism, bringing hope to those suffering from chronic liver diseases and challenging the current transplantation paradigm.

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Author

Sinead Huang