NOVEL BIOMARKERS OF AMYOTROPHIC LATERAL SCLEROSIS: ROLE IN PATHOGENESIS AND POSSIBLE THERAPEUTIC TARGETS

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease, which affects motor neurons, for which there is as yet no cure. It is a very difficult disease to diagnose. There is still no test or procedure to make the diagnosis of ALS, which can only be established through a clinical examination, often after ruling out other diseases that resemble ALS. Thus, the disease is usually diagnosed late and often not correctly. Specific protein biomarkers are needed for the development of an assay that can be easily done in hospital laboratories. Such a diagnostic test would make accurate early diagnosis possible and eventually prompt pharmacological treatment. In addition, disease biomarkers may give clues on the molecular mechanisms underlying ALS, which are still undefined, and can be useful therapeutic targets. Our laboratory has identified a number of candidate protein biomarkers that will be studied further in this project to evaluate their specificity as diagnostic markers and possible therapeutic targets. In particular, the protein cyclophilin A, a biomarker in patients and in animal models, seems to be involved in the process leading to motor neuron death. Studies along these lines could lead to the development of novel efficacious therapeutic approaches.