25.03.2013 - Italian researchers have tracked down the signalling pathway causing
polycystic kidney disease and identified a promising target.
Polycystic kidney disease (PKD) is one of the most common life-threatening genetic diseases, affecting an estimated 12.5 million people worldwide. Due to multiple fluid-filled cysts, both kidneys enlarge and can also damage liver, pancreas and - more rarely - heart and brain. Currently no treatment exists for the autosomally and recessively inherited forms of PKD.
The PKD-1 gene is mutated in 85% of the autosomal dominant form. Italian researchers have now found that the disorder can be targeted by an existing diabetes drug in combination with a glucose analogon. The team headed by Alessandra Boletta from San Raffaele Institute in Milan found that the PKD-1 mutation caused a switch in sugar metabolism towards aerobic glycolysis due to the inactivation of the master regulator AMPK (liver kinase B1-AMP activated protein kinase).
Forced activation of AMPK either by rapamycin, the diabetes medicine metformin or the non-metabolised sugar analogon 2-deoxy-glucose (2-DG) triggered reduction of the number of cysts in a mouse model. AMPK activation revitalized the ERK signalling pathway which re-activated mTORC1 that in turn normalised glycolysis. The researchers believe that a combination therapy of 2-DG and metformin offers a treatment opportunity for the progressive disease.
16.04.2015 The Swiss biotech industry can look back on 2014 as a good year, but political uncertainties do remain. This was one of the topics discussed during the Swiss Biotech Day in Basel, which attracted more than 400 participants.
14.04.2015 Belgian Thrombogenics NV has launched a new oncology company with Gent-based life sciences institute VIB. The new venture will develop an antibody for the treatment of a fast-growing brain tumour that affects children and adolescents.