Drug Screen Identifies Leflunomide for Treatment of Inflammatory Bowel Disease Caused by TTC7A Deficiency

Mutations in the gene TTC7A cause intestinal and epithelial defects and a severe early onset intestinal disease. Using a cell-based drug screen and validation in a zebrafish model of disease Aleixo Muise and colleagues discovered that the FDA-approved drug Leflunomide may reverse cell and organ defects seen in TTC7A disease. In collaboration with Jay Thiagarajah and colleagues Leflunomide was found to also reverse changes in patient-derived cells, suggesting that the drug may be repurposed for the treatment of TTC7A disease.

Read more about this study:
Jardine et al Gastro 2020