According to a new study conducted in the Intramural Research Program of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) (a component of the National Institutes of Health) and published in the august 10 issue of the New England Journal of Medicine, a drug called anakinra brings marked improvement both in symptoms and the inflammation underlying a rare and debilitating disorder called neonatal-onset multisystem inflammatory disease (NOMID) in children and young adults.
NOMID, also known as chronic infantile neurologic cutaneous articular (CINCA) syndrome, is an inflammatory disorder that affects numerous organs and body systems, including the skin, joints, eyes and central nervous system.
The first sign of this disease in most children is a rash that develops within the first six weeks of life, while other problems including fever, meningitis, joint damage, vision and hearing loss, and mental retardation, can follow.
NOMID is one of a group of illnesses that NIAMS has designated autoinflammatory diseases. This disease is progressive and often fatal, despite the treatment to control the inflammation — including high-dose corticosteroids, disease-modifying antirheumatic drugs such as methotrexate, and nonsteroidal anti-inflammatory drugs (NSAIDs) such as ibuprofen or naproxen. 20% of children with NOMID don’t survive into adulthood.
The mechanism of NOMID is not yet completely understood. BUT, research in recent years revealed that mutations in a gene called CIAS1 in approximately 60 percent of patients with the disease."This research shows the importance of studying rare but enormously instructive diseases, a unique strength of the NIH intramural science program," says NIH Director Elias A. Zerhouni, M.D.
"This study provides new insights on fundamental mechanisms of inflammation. More importantly this new therapy will reduce the pain and suffering of these young patients allowing them to live a fuller life than previously possible."
CIAS1 encodes cryopyrin, which belongs to a group of interacting proteins involved in regulating inflammation and programmed cell death, which plays a crucial role in ridding the body of cells that are no longer needed.
The mutations, scientists have found, lead to an imbalance of a cytokine, or chemical messenger, called interleukin-1 (IL-1), which is believed to drive the inflammation that causes damage in patients with the disease.
Anakinra, a drug approved for the treatment of rheumatoid arthritis is a biologic agent, a medicine based on compounds that are made by living cells and used to stimulate or restore the ability of the immune system to fight disease and/or infection.
Anakinra, known under the brand name Kineret® works by blocking the effects of IL-1beta (IL-1β) and have been in isolated case reports to be effective in treating the rash and other symptoms of NOMID.
Source: NIH/NIAMS News.
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