Will This Drug Relight the Dimming Hope of Finding Effective Therapies for COVID-19?

Michael Wang, MD., Ph.D., member of AFCR Scientific Advisory Board

Many people were disappointed by recent high-profile clinical trial results on Remdesivir, the drug that people had put high hopes on as the best candidate for an effective COVID-19 treatment.

A report published in The New England Journal of Medicine on May 22 revealed that the once very promising anti-viral drug Remdesivir only showed a relatively modest effect on hospitalized COVID-19 patients: shortening the time to recovery from 15 days for patients in placebo groups to 11 days for patients receiving the Remdesivir treatment. More importantly, the widely expected lower death rate wasn’t observed in this trial and serious side effects were reported for 21% of patients treated by the medication.

The disappointment has cast a shadow on people’s hope to more quickly put COVID-19 under control. But it won’t discourage doctors and researchers from continuing their search for better anti-viral medications. Indeed, data from a well-designed multicenter clinical trial on a drug called Ruxolitinib might be able to rekindle the hope.

Clinical trial results on Ruxolitinib were published in the Journal of Allergy and Clinical Immunology on May 26. The most noticeable data point of the study is the zero death rate for severe COVID-19 patients who received the treatment, compared with the 14% death rate for patients in the control group. This is very encouraging as no other drugs going through clinical trials have shown such a death reduction or life-saving benefit for severe COVID-19 cases. In addition, Ruxolitinib’s safety profile seems better than Remdesivir’s, as the former’s side effects recorded during its trial were fewer, milder and reversible.

Interestingly, Ruxolitinib was not originally developed as an anti-viral drug that directly targets virus structural proteins or the production processes within infected human cells. Rather, the main targets of Ruxolitibib are Janus kinases (JAK), so the drug is called a JAK inhibitor. One of its functions is to inhibit abnormal inflammatory reactions, or “cytokine storm,” in various disease conditions. Severe COVID-19 patients usually have life-threatening cytokine storm in multiple vital organs. The use of Ruxolitinib would help to mitigate the lethal immune damage caused by the cytokine storm in severe COVID-19 cases, saving lives.

The clinical trial was conducted jointly by doctors at Tongji Hospital of Tongji Medical College in Wuhan, China, and Cincinnati Children’s Hospital Medical Center in Cincinnati, Ohio. It is the first randomized controlled trial on the use of Ruxolitinib in patients with severe COVID-19. Despite the small number of patients involved in the trial (20 patients in the testing group and 21 patients in the control group), the findings are quite encouraging, as the trial was based on a novel therapeutic rationale and has opened a new area where safe and effective drugs to treat COVID-19 might be found. Of course, larger and further trials are needed in the future to demonstrate Ruxolitinib’s safety and therapeutic effects to control the overall disease deterioration and reduce the death rates of severe COVID-19 patients.

Our hope for ending the coronavirus pandemic will never die because our efforts and innovation on research and clinical trials will never stop. This will lead us to the final victory down the road.