Phase 1 Trial of Cyclosporine for Hospitalized Patients with COVID-19

Blumberg, E. A.; Tebas, P.; Frank, I.; Marshall, A.; Chew, A.; Veloso, E. A.; Carulli, A.; Rogal, W.; Gaymon, A. L.; Schmidt, A. H.; Barnette, T.; Jurek, R.; Noorchashm, H.; Hwang, W.-T.; Noll, J. H.; Fraietta, J. A.; June, C. H.; Hexner, E. O.

Coronavirus Disease 2019 (COVID-19) remains a global health emergency with limited treatment options, lagging vaccine rates and inadequate healthcare resources in the face of an ongoing calamity. The disease is characterized by immune dysregulation and cytokine storm. Cyclosporine A (CSA) is a calcineurin inhibitor that modulates cytokine production and may have direct antiviral properties against coronaviruses. To test whether a short course of treatment was safe in COVID-19 patients, we treated 10 hospitalized, oxygen requiring, non-critically ill patients with CSA at a starting dose of 9mg/kg/day. Five patients experienced adverse events, none were serious, and transaminitis was most common. No subject enrolled in this trial required intensive care unit (ICU)-level care and all patients were discharged alive from the hospital. Further, CSA treatment was associated with significant reductions in serum cytokines and chemokines important in COVID-19 hyper-inflammation, including CXCL10. In conclusion, short courses of CSA appear safe and feasible in COVID-19 patients requiring oxygen and therefore, may be a useful adjunct in resource-poor or resource-limited health care settings.