Combination therapy holds promise for rare immune disorder

Memphis, Tennessee, June 12, 2020

Researcher smiling and facing forward.

Co-senior author Kim Nichols, M.D., discovered combining ruxolitinib with the currently used drug dexamethasone was more effective in treating HLH.

St. Jude scientists and their colleagues are studying a rare immune disorder. This disorder is called hemophagocytic lymphohistiocytosis (HLH).

In HLH, an overactive immune system produces too many cytokines. These chemicals in the bloodstream recruit and activate even more immune cells.

“For 20 years treatment for HLH has stayed the same,” said Kim Nichols, MD, of Oncology. “But we know many patients either do not respond to therapy or later relapse, so we dug into the biology to come up with a different treatment strategy.”

The JAK1/2 inhibitor ruxolitinib has been shown to work well in models of HLH. Nichols and her team wanted to study how this drug interferes with cytokines. They also wanted to test whether any one cytokine was more important to inhibit.

The researchers found that the cytokine interleukin-2 drives HLH treatment resistance. They found that combining ruxolitinib with the currently used drug dexamethasone was more effective. The study supports testing this combo in a clinical trial.

Blood published a report on this work.

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