Amyotrophic lateral sclerosis (ALS), like many other neurodegenerative disorders, is a disease severely lacking in a consistent ‘one drug benefits all’ therapy. As you can see, the ALS patient population is extremely heterogenous with familial genes only accounting for 10% of overall cases (top panel). Our lab is investigating the effects of two antioxidant drugs alongside the current drug prescribed for ALS, i.e. Riluzole, in patient-derived astrocytes. We are using a combination of RNA-sequencing and cellular function assays to determine genetic signatures that discriminate between patient responders versus non-responders so that we can group patients according to drug response (bottom panel). Our current aim is to identify gene signatures that can discriminate between patient responders and non-responders to selected drugs. These are very early days, but our future goal is to be able to identify the best drug for each specific patient, making a massive improvement in drug efficacy and patient well-being.
Ferraiuolo Lab 