February 28, 2012

RA Drug Slows Damage of Rare Disease

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Rheumatoid arthritis drug prevents organ damage associated with rare disease

(dailyRx News) Inflammation is at the root of many common diseases, including rheumatoid arthritis. As such, it may be possible that certain rheumatoid arthritis drugs also can treat other inflammatory diseases.

Kineret (anakinra), a drug used to treat rheumatoid arthritis, may slow down organ damage in people with a rare disorder called neonatal onset multisystem inflammatory disease (NOMID).

"Get treated for NOMID to stop organ damage."

In a recent study, Raphaela Goldbach-Mansky, M.D., M.H.S., of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), and colleagues found that Kineret not only blocks inflammation in the inner ear, eyes, and brain, but also can prevent hearing and vision loss.

NOMID is a rare genetic disease that affects many parts of the body, including the skin, joints, eyes, brain, and spinal cord. At first, the only sign of the disease may be a rash. However, the disease can eventually lead to fever, meningitis (a type of bacterial infection), joint damage, vision loss, hearing loss, and brain damage.

Kineret is part of a class of drugs called biologic response modifiers or biologics. These drugs block the activity of a protein called interleukin-1.

In patients with NOMID and many other diseases, the body produces too much interleukin-1, which leads to harmful inflammation.

Past research has shown that blocking interleukin-1 can relieve symptoms of NOMID. This study, however, is the first to show that Kineret actually can slow organ damage if the drug is given at higher doses.

"Inflammation prolonged over many years will eventually cause irreversible damage and loss of function," says Dr. Goldbach-Mansky.

For example, inflammation of the inner ear leads to hearing loss in NOMID patients. Similarly, inflammation can put pressure on parts of the brain, leading to thinning of the optic nerve and vision loss.

For their study, the researchers gave daily doses of Kineret to patients between the ages of 10 months to 42 years. Patients received one to five milligrams of Kineret per kilogram of body weight per day. That is, their daily doses depended on their body weight. Participants were treated for at least 36 months and for as long as 60 months.

Initially, Dr. Goldbach-Mansky and colleagues found that the doses Kineret they were giving to patients were not enough to control organ inflammation.

Once the researchers increased doses, they were able to prevent organ inflammation, which protected organ function in the majority of patients.

On top of that, the researchers discovered how to predict who has the highest risk of losing their hearing and vision.

According to Dr. Cailin H. Sibley, the study's first author, "The few patients in the study who had hearing loss were also the ones who continued to have inflammation in the inner ear." In other words, where inflammation was prevented, organ function was preserved.

"We also found that people who had thin optic nerves when we assessed their vision were more likely to lose vision than those who had thick optic nerves, simply because they had already lost fibers due to untreated disease and, therefore, started with a huge disadvantage," says Dr. Sibley.

These findings suggest that diagnosing and treating NOMID early can protect organs from permanent damage.

"We are continuing the study with an emphasis on enrolling very young children to prospectively show that we can prevent any organ damage from developing if we start treatment early in life," says Dr. Goldbach-Mansky.

It is important to note that Kineret does not cure NOMID. The inflammation-fighting benefits only last as long as a patient takes the drug. However, this study shows that Kineret may offer a huge helping hand to those suffering from this rare disease.

"Without Kineret, people with NOMID are at risk of progressive organ damage that results in hearing and vision loss, cognitive impairment and, in many cases, early death," Dr. Goldbach-Mansky explains.

"As many as 20 percent of children with this genetic disorder do not live to adulthood," she says.

"This study shows that treatment over five years is safe and effective, and can prevent organ damage," she concludes.

This research was funded in part by the NIAMS, the Intramural Research Programs of the National Cancer Institute, the National Institute of Deafness and Other Communication Disorders, the National Eye Institute, the National Institute of Mental Health, the National Institute of Neurological Disorders and Stroke, and the NIH Clinical Center.

The study was published in the journal Arthritis & Rheumatism.

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Last Updated:
March 2, 2012