The U.S. Food and Drug Administration (FDA) recently approved ruxolitinib, the first drug for patients with acute graft-versus-host disease (GVHD) who have an inadequate response to steroid treatment. Madan Jagasia, MBBS, MS, MMHC, chief medical officer and co-leader of the Translational Research and Interventional Oncology Program at Vanderbilt-Ingram Cancer Center (VICC), was a lead investigator of the REACH1 trial that was the basis for the FDA approval.
“Every year in the United States, about half of the people who develop acute GVHD do not respond adequately to steroids, making it an extremely challenging disease to treat,” said Jagasia, holder of the Beverly and George Rawlings Directorship, and professor of Medicine at Vanderbilt. “While allogeneic stem cell transplants have the potential to transform people’s lives, the onset of GVHD can significantly impact their prognosis. Ruxolitinib is now a new treatment option for acute GVHD patients who do not respond to corticosteroids who, until now, have had limited choices.” The overall response rate after 28 days of treatment was 54.9%. The recommended starting dose of ruxolitinib for GVHD is 5 mg twice daily. Incyte Corporation, which applied for the FDA approval, will market the drug under the brand name Jakafi.
GVHD occurs when donor immune (T cells) begin attacking the recipient’s healthy tissue after an allogeneic stem cell transplant. There are two types of GVHD — acute and chronic. The FDA approval of ruxolitinib is for the acute form of the disease in patients who have not responded to corticosteroids.
“This would not have been possible without the support of our amazing clinical trials team, our incredible physicians, mid-level providers and nurses who take care of these complex patients,” Jagasia said. “Last, but not least, we cannot thank our patients enough who are not only fighting these tough diseases but by their participation in studies, help others.”