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.
19.11.2014 It’s not every day that a company is pushed into the top ten of its sector, but that day has come for Actavis. Christmas came early for the new pharma giant after completing a US$66bn (€53bn) takeover battle for the Botox manufacturer Allergan.
18.11.2014 Merck KGaA and US rival Pfizer have signed a immuno-oncology alliance worth billions: Merck will share the development and commercialisation of its cancer antibody with its US partner – for €2.3bn.
14.11.2014 Cosily coinciding with World Diabetes Day on 14th November, French biopharma Les Laboratories Servier has struck a US$1bn deal with US pharma Intarcia Therapeutics for the exclusive rights to Intarcia’s unprecedented therapy for type 2 diabetes.
12.11.2014 Glitz and glamour are not words often used to describe science and mathematics. But once a year, celebrities from the science and showbiz world come together at the Breakthough Prizes, a glitzy television bash that honours scientists for their achievements. French life scientist Emmanuelle Charpentier was among this year’s winners.
04.11.2014 Galecto Biotech has entered an agreement with Bristol-Myers Squibb for the acquisition of the company and its lead drug, providing the Swedish company with the potential of reaching over US$400m.