A new approach to treating organ damage in inflammatory diseases

2012

Challenge

The rare and debilitating genetic disorder known as neonatal-onset multisystem inflammatory disease (NOMID) causes persistent inflammation and ongoing tissue damage, often beginning within the first weeks of life. Because NOMID affects numerous organs and body systems, early diagnosis and treatment are important for preventing long-term organ damage.

Advance

IRP researchers led by Raphaela Goldbach-Mansky, M.D., M.H.S., discovered that blocking interleukin-1 (IL-1)—an inflammatory protein made by immune system cells—with increasing doses of the FDA-approved rheumatoid arthritis treatment, anakinra, could preserve organ function in most patients.

Impact

Although overproduction of IL-1 can lead to damaging inflammation, the immune system still requires certain levels of IL-1 to help fight infections. The results alleviate concern that treating NOMID by blocking IL-1 may leave the body vulnerable to infection by showing that anakinra is effective and well-tolerated in the treatment of NOMID.

Publications

Sibley CH, Plass N, Snow J, Wiggs EA, Brewer CC, King KA, Zalewski C, Kim HJ, Bishop R, Hill S, Paul SM, Kicker P, Phillips Z, Dolan JG, Widemann B, Jayaprakash N, Pucino F, Stone DL, Chapelle D, Snyder C, Butman JA, Wesley R, Goldbach-Mansky R. (2012). Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: a cohort study to determine three- and five-year outcomes. Arthritis Rheum., 64(7), 2375-86.