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.
23.07.2014 In the largest genomic study published on any psychiatric disorder so far, researchers have indentified 83 new locations in the human genome associated with the risk of developing schizophrenia.
15.07.2014 British specialty pharma company Prostrakan is handing over £230m for Archimedes Pharma Limited, a company that brings its lead product Pecfent and its proprietary drug delivery technology platform into the deal.
11.07.2014 Among the seven newly launched Joint Technology Initiatives (JTI) of Horizon2020 are two with deep roots in biotech: IMI is going into its second round while the newly established Bio-based Industries partnership invests in bioeconomy.
04.07.2014 Genentech, a member of Swiss Roche Group, is set to acquire US-biotech Seragon Pharmaceuticals Inc. With the move, Genentech will obtain the rights to investigational breast cancer treatments.
03.07.2014 Nicox S.A. is stretching beyond the European market: The French eye health specialist is set to acquire Aciex Therapeutics Inc., a US-based pharma company with several ophthalmic therapeutics nearing market maturity.
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