Andalusian Centre for Molecular Biology and Regenerative Medicine (CABIMER)

In a paper published in Molecular Cell, a team of researchers led by the Spanish National Cancer Research Centre (CNIO) provides the first evidence that a possible cause of the hereditary type of ALS - familial ALS - is the accumulation in motor neurons of 'junk proteins', proteins with no function that accumulate unduly and prevent the cell from functioning properly. In addition, the research describes a new causal factor in the ageing process: nucleolar stress, which encompasses alterations in organelles called nucleoli. 

The European Medicines Agency (EMA) has recommended granting marketing authorisation in the European Union for a new therapy for the treatment of adult patients with amyotrophic lateral sclerosis (ALS), a rare and frequently fatal disease that causes muscle weakness and leads to paralysis. Qalsody (tofersen) is indicated for the treatment of adults with ALS who have a mutation in the SOD1 gene. There is currently only one treatment for ALS authorised in the EU (riluzole).