New technology to treat acute intermittent porphyria
New technology to treat acute intermittent porphyria
Scientists from the Center for Applied Medical Research (CIMA) of the University of Navarra (Spain) have developed a new technology of messenger RNA (mRNA) that acts on the origin of the disease and quickly corrects the symptoms of acute intermittent porphyria in preclinical models . The results, published in Nature Medicine, are the result of collaboration with the biotechnology company Moderna Therapeutics (Cambridge, USA).
Acute intermittent porphyria is a rare genetic disease, or orphan, caused by the inability of the liver to metabolize porphyrin precursors. It occurs in the form of intermittent painful crises and can lead to significant neurological damage and even require liver transplantation.
In these patients, seizures can be caused by some medications, strict diets or prolonged stress. However, the main trigger is the female hormones associated with the menstrual cycle, which is especially evident in young women, with important work and family responsibilities.
"At present, some medications are able to control their symptoms, but there is no specific treatment to combat the origin of the disease," says Antonio Fontanellas, lead author of the article. According to Fontanellas, a researcher at the CIMA and a member of the Center for Biomedical Research in the Liver and Digestive Diseases Network (CIBERehd). "The research we have developed shows the efficacy of mRNA for the treatment of acute intermittent porphyria."
In front, Sara Arcelus, Antonio Fontanellas and Ana Sampedro. Behind, Matías Ávila, Iranzu Serrano, Pedro Berraondo and Daniel Jericó, of the CIMA Hepatology Program. (Photo: Manuel Castells)
The mRNA is the molecule that connects the accumulated information in the genes with the production of the proteins necessary for the proper functioning of the cell. "Our work suggests that the administration of this mRNA wrapped in patented lipid nanoparticles (micro-droplets of fat) can restore the activity of the deficient enzyme in the liver of mice with porphyria in less than two hours. It was also observed that it protects against key features of the disease, such as pain, hypertension and motor disability. Another advantage of this technology is the possibility of repeating its administration as many times as necessary to avoid the appearance of new crises, "says Fontanellas.
"In this joint work we demonstrate that mRNA technology restores enzyme deficiency within liver cells and normalizes markers of disease during attacks," explains Paolo Martini, scientific director of the Rare Diseases Program at Moderna Therapeutics. "These data further support our current efforts in the field of rare metabolic diseases, where mRNA could allow a particular tissue to produce therapeutic levels of a functional protein." (Source: CIMA of the University of Navarra)
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