Blood Disorder Drug Ruxolitinib Offers Hope for Severe Malaria Recovery

A new clinical trial led by QIMR Berghofer, in collaboration with University of Sunshine Coast Clinical Trials Network has found a medication currently used for some blood disorders could help the body fight malaria more effectively. The findings mean that the drug, ruxolitinib, could potentially be used alongside standard treatment to boost recovery and strengthen people's immune systems against future infections. Malaria kills more than 600,000 people each year, and three-quarters of those deaths are in children under the age of 5. Current treatments for malaria work by killing the parasite that causes most malaria deaths, Plasmodium falciparum. However, even with these treatments, fatality rates from severe malaria remain high. In addition, while patients develop some immunity after infection, this protection is often incomplete, leaving many vulnerable to reinfection. How ruxolitinib could help malaria treatment Head of QIMR Berghofer's Clinical Malaria Group Associate Professor Bridget Barber says the research overcomes a key hurdle. "While antimalarial treatments are effective at killing the parasite, they don't directly address the inflammation that contributes to severe illness and death. These findings suggest that we may be able to improve clinical outcomes by targeting the host inflammatory response as well as the parasite itself," she said. The research, published in Science Translational Medicine, looked at how the immune system responds to malaria via the body's "early warning system" known as type 1 interferon signaling. To do this, researchers enrolled 20 healthy adult volunteers who had never been exposed to malaria. Participants were deliberately infected with Plasmodium falciparum under closely monitored conditions. Eight days later, all participants received standard malaria treatment (artemether-lumefantrine), while 11 were also given ruxolitinib. Three months later, participants were re-infected with malaria to test how their immune systems responded to a second infection. Study results and future directions The research revealed ruxolitinib was safe and well-tolerated. Compared with the placebo group, participants who received ruxolitinib showed a lower inflammatory response, and favorable changes in markers linked to disease severity. QIMR Berghofer's Program Director of Infection and Inflammation, Professor Christian Engwerda, says the results are encouraging. "One of the biggest challenges in efforts to eliminate malaria is the limited efficacy and duration of protection provided by current vaccines. By boosting the immune system without causing detrimental inflammation with drugs like ruxolitinib, we may be able to overcome these challenges," he said. The researchers say it's important to note that the study was conducted in healthy volunteers who did not live in malaria-endemic regions. Further studies in malaria-endemic regions will be needed to determine whether these findings translate into improved outcomes for patients most affected by the disease.