Clinical Head of Neurology at the University Hospital of Donostia, Scientific Director of CIBERNED and Research Director of the Neurosciences area of ISS Biodonostia
Researchers in the United States have used stem cells created from patients with a very rare type of ALS, more prevalent in Brazil, to target a key gene in the stress response and reverse the damage suffered by motor neurons in the laboratory. They believe it is "a promising proof-of-concept for future therapeutic strategies" and "could help lay the foundation for genetically informed clinical trials".
The New England Journal of Medicine reports the publication of data from a clinical trial studying the drug Tofersen against a form of Amyotrophic Lateral Sclerosis (ALS) caused by mutations in the SOD1 gene, which accounts for 2% of sporadic ALS. Six months after treatment, some biomarkers of neuronal damage improved, but not the clinical condition of the patients. In an extension of the study, with no placebo group, a slowing of functional loss was observed, although the researchers acknowledge that there are limitations in interpreting this result.