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The Making of Medicine

A Promising Discovery About Myelofibrosis

Our Golam Mohi, PhD, has identified a promising new treatment approach for myelofibrosis, a potentially deadly bone marrow cancer. Right now, the only cure for myelofibrosis is a bone-marrow transplant, and not all patients are eligible for that, so a new treatment would be most useful.

Professor Mohi and his team were investigating the chronic inflammation seen in certain blood cancers when they identified a cytokine, called interleukin-1, that contributes to the progression of myelofibrosis. Targeting this cytokine, commonly called IL-1, could prevent the disease from progressing, sparing bone marrow the harmful scarring that is the hallmark of myelofibrosis.

“Our research provides new understanding on how inflammatory signaling mediated by interleukin-1 contributes to the development of bone marrow fibrosis and could lead to new therapeutic approach for this fatal bone marrow cancer," Professor Mohi said.

Mohi and his team found that increasing IL-1 in lab mice accelerated bone marrow scarring and fueled the excess production of blood cells. Reducing it, on the other hand, had the opposite effect. Scientists may be able to adapt this approach or use other means to block IL-1 and generate similar benefits in human patients, the researchers say, though much more research and testing is needed.

“Based on the findings from this study, we suggest that combination therapies targeting both JAK2 and IL-1 could be useful for treatment of myelofibrosis,” Professor Mohi said. “We hope that our exciting laboratory finding will translate into clinical trials and make significant improvement in the treatment of patients with myelofibrosis.”

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