Chasing the Storm: Lessons from Studying Ruxolitinib as Potential Treatment for Severe COVID-19
Published July 2020 | Journal of Allergy and Clinical Immunology
As scientists around the world raced to understand how the SARS-Cov-2 virus was causing such devastating inflammatory responses, a scientist at Cincinnati Children’s made a suggestion in early 2020 to a team of doctors in China that prompted an intense look at a hopeful treatment for people with severe illness during the pandemic.
Gang Huang, PhD, Division of Pathology at Cincinnati Children’s, had been part of a team that was studying the benefits of ruxolitinib as a treatment to control the cytokine storms that can occur among children with the rare disease hemophagocytic lymphohistiocytosis (HLH). He informed a colleague in China—Jianfeng Zhou, MD, PhD—about the findings, which prompted a rapidly launched clinical trial.
By July 2020, some intriguing data were published from the multicenter study, which involved 20 patients receiving ruxolitinib plus standard-of-care treatment, and 21 people receiving a placebo in addition to standard care. Doctors reported significant declines of seven cytokines in the treatment group, improvements on chest CT scans, faster recovery from lymphopenia, and minimal side effects. While three control group patients died of respiratory failure, none died of respiratory failure in the treatment group.
However, the study was too small for the observed benefits to be considered statistically significant. Over the next year, hundreds more people with COVID-19 were treated with the drug in several larger clinical trials and compassionate use settings. The drug ultimately failed to show clear benefit in the RUXCOVID clinical trial (December 2020) and the DEVENT trial (March 2021). However, research continues to evaluate ruxolitinib in combination with other drugs.
Given concerns about breakthrough infections, Huang says alternative therapies to prevent runaway inflammatory reactions may still be important for high-risk groups in addition to vaccines and other antiviral therapies.
