A team of Institute for Translational Medicine Research (IIMT – CONICET / Universidad Austral) validated a new experimental therapeutic strategy that could benefit patients with severe liver disease.
In a major advance of Argentine translational science, researchers from the Laboratory of Experimental Hepatology and Gene Therapy (IIMT) identified for the first time an effective therapeutic pathway to treat fulminant hepatitis, a condition that can cause death in up to 40% of cases without transplantation.
The team led by Dr. Guillermo Mazzolini discovered that to block the RAC1 protein using the experimental molecule 1D-142 —originally developed for cancer treatment— reduces liver damage and improves survival in preclinical models.
The study was recently published in Journal of Hepatology Reports and represents a key step towards the development of new therapies for liver diseases without effective treatment.
“We seek to position ourselves in the development of technologies that generate concrete solutions to real health problems,” highlighted Dr. Mazzolini, who is also Dean of the Faculty of Biomedical Sciences at the Universidad Austral and Senior Researcher at CONICET.
The molecule was tested in three animal models and in human liver tissue, showing promising results: it significantly reduced inflammation, necrosis, and biochemical markers of liver damage, with no evidence of toxicity in healthy livers. Furthermore, the technology already has a international patent pending.
The work was made possible thanks to a collaborative approach that included researchers from INTI and the biotechnology company Spectrum, in a synergy of public and private science with potential impact on public health.
The team behind the discovery:
