What's Hot in 2016: Metabolic Diseases
Diabetes and obesity is behind an alarming increase in non-alcoholic steatohepatitis (NASH). No approved therapies exist but there are glimmers of hope.
The landscape of therapies for treating metabolic diseases is changing rapidly. Restoring normal glucose levels, while necessary, is no longer going to be sufficient for moving candidate therapies through regulatory approval. New entries into the area will have to address the morbidity and mortality associated with diabetes, obesity and metabolic syndrome. Non-alcoholic steatohepatitis (NASH) is one of those complex conditions attracting a great deal of attention. The abnormal accumulation of lipid in the liver with attendant inflammation, hepatocyte degeneration and subsequent scarring is becoming quite prevalent. Estimates suggest that between 2% and 5% of the adult population are affected, and an alarming number of pediatric patients are presenting with this disorder as well. There are no approved therapies for NASH, but there are some glimmers of hope. Two years ago, precious few drug companies were studying NASH, today, at least 34 companies are eyeing this growing public health problem, with the developer of one late-stage compound, obeticholic acid (OCA), granted FDA's breakthrough status earlier this year. However, because NASH tends to accompany diabetes, obesity and other metabolic disorders it is unlikely that a single agent will be sufficient and the need for disease biomarkers and/or imaging models are clearly needed to assess NASH.
—Joe Cornicelli, Scientific Advisor, Discovery Research Services