New drugs for the treatment of polycystic hepatorenal diseases

Polycystic hepatorenal diseases are hereditary genetic disorders characterized by the progressive development of multiple symptomatic cysts in the kidneys and/or the liver, which can produce alterations in the function of these organs and/or complications related to their growth. Currently, there are no effective pharmacological treatments and the only curative option is transplantation of these organs.

UPV/EHU researchers led by Dr. Fernando Cossío (UPV/EHU), scientific director of Ikerbasque, and the IIS Biodonostia Liver Diseases group, led by Ikerbasque researcher Dr. Jesús M. Bañales, have collaborated in the development of new drugs that have been shown to be effective in reducing the growth of liver and kidney cysts in experimental models of this disease, which can have great clinical significance. This multidisciplinary project led between both Basque institutions has had the collaboration of researchers from the University of Salamanca, led by Dr. José JG Marín, from the Idibell Institute of Catalonia, led by Dr. Manel Esteller, and from the Hormel Institute of Minnesota (USA), headed by Dr. Sergio Gradilone.

The drugs developed are based on the structure of ursodeoxycholic acid (UDCA), a bile acid present in our body in low concentration and that has protective properties for the liver. In fact, its administration is indicated for the treatment of some liver diseases. The researchers have relied on the structure and properties of this molecule to design and synthesize a family of chemical derivatives aimed at inhibiting a key protein in promoting the growth of hepatorenal cysts. Published results have shown that these new drugs are capable of blocking the growth of liver and kidney cysts in an animal model of this disease.

The promising therapeutic effects of these new drugs have managed to attract great interest from the international scientific community. So much so that this work was selected for oral presentation at the International Congress of the European Association for the Study of the Liver (EASL) held in Vienna. Likewise, it has recently been published in the prestigious international journal Hepatology (official journal of the American Association for the Study of the Liver), and has led to the Doctoral Thesis of Dr. Francisco J. Caballero (UPV/EHU, Biodonostia), the first author of said work directed by Drs. Fernando Cossío (UPV/EHU) and Jesús M. Bañales (Biodonostia, Ikerbasque).

This project recently received the national FIPSE award for innovation (Ministry of Science and Innovation) that allowed for the patenting of these drugs, which have been licensed to the ATLAS Molecular Pharma company, in the Basque Country, to promote their clinical study. Furthermore, this project has received competitive financing from the RIS3 Euskadi Program, the Ministry of Science and Innovation, the Carlos III Health Institute (ISCIII) and Ikerbasque.