Dr. Ted Allison and his team at the University of Alberta have discovered that telbivudine, a blood brain barrier-permeable thymidine analogue approved for treatment of Hepatitis B, is effective in ameliorating Amyotrophic Lateral Sclerosis (ALS). Results from in vitro studies have shown that telbivudine binds with tryptophan 32 residue in superoxide dismutase 1 (SOD1) protein and prevents SOD1 misfolding. In vivo studies in zebrafish models have shown that administration of telbivudine results in a significant improvement in motor neuron function and swim performance along with reduced toxicity effects in comparison to uridine and 5 Fluorouridine.
- Telbivudine has an established safety profile as it is FDA approved for treatment of hepatitis B
- Telbivudine permeates the blood brain barrier
Technology Management Group
TEC Edmonton – University of Alberta