World — 09 October 2012

Researchers have identified a new therapy that appears to offer relief from the painful inflammation of the eye nerves and spinal cord that plague patients with neuromyelitis optica (NMO), a central nervous system disorder often confused with multiple sclerosis.

Using a drug typically prescribed for blood disorders, doctors at the Mayo Clinic say eculizumab successfully prevented attacks in 12 of the 14 patients studied over a one year period. Although not a cure, researchers are optimistic that the new therapy may one day lead to longer attack-free periods for  thousands of NMO patients worldwide.

“Disability in NMO is attack related and these attacks are usually severe. If untreated, they can have devastating, irreversible effects on function,” says lead author Sean Pittock MD, a Mayo Clinic neurologist.

NMO can cause blindness, along with weakness or paralysis in the legs or arms. Additional symptoms include painful spasms and a loss of sensation, along with bladder or bowel dysfunction from spinal cord damage. The disease affects people of all ages, races and ethnic groups, but is more common in women.

Immunosuppressants, often used to treat transplant patients, are the first line of treatment.  Although the attacks may be reversible, they can still be severe enough to cause permanent visual loss and problems with walking. Because NMO has only recently been identified as a syndrome distinct from MS, knowing how many people it affects is difficult.

In 2004, Mayo Clinic researchers discovered the antibody NMO-IgG, which they describe as the first serum biomarker for any form of inflammatory brain disease.  A year later, they identified the target of the antibody as the water channel aquaporin 4.

Doctors say those discoveries helped them better understand the cause and potential treatments for NMO. So far, the Mayo Clinic’s Neuroimmunology Laboratory has detected the antibody in roughly 3,500 U.S. patients.

To correctly diagnose the disease, doctors begin with a patient’s medical history and ask them about their symptoms. Additional procedures include a neurological examination, an MRI, blood serum tests and a spinal tap.

“We’ve learned there are many people who fit within the NMO spectrum who, in the past, were believed to have MS,” says co-author Dean Wingerchuk, MD, a neurologist at the Mayo Clinic in Arizona. “In addition, distinguishing NMO from MS is important for preserving the validity of therapeutic trials for MS by not enrolling patients with NMO.”

In their study, researchers followed the progress of 14 patients with active and severe disease symptoms, defined as two attacks in the previous six months, or three within the past year. They all received the same treatment of eculizumab intravenously every two weeks for one year.

Twelve patients were symptom free throughout the year. Two patients had one attack each, but these were mild and considered by the investigators to be “possible, not definite” attacks.

“If we can stop the attacks in NMO, and it appears we can, “said Pittock, “then we can hopefully prevent disability and allow patients to maintain function and a good quality of life.

The research was presented at the American Neurological Association’s annual meeting in Boston. The study was funded by Alexion Pharmaceutical (NASDAQ: ALXN), which makes eculizumab.


About Author

Richard Lenti

Richard Lenti has worked as a news writer for the last 20 years at various television stations in Los Angeles. He is a Golden Mike winner and a graduate of California State University, Fresno. With roots in print journalism, Richard is excited to be “published” once again; having people read his work as opposed to having it read to them. As a freelance writer his work has appeared in the Easy Reader, L.A. Jazz Scene, Irrigation and Green Industry, and the KCAL 9 Online website.

(1) Reader Comment

  1. Sounds like things are continuing to improve. There is always hope for those in need. MS is a serious problem so I am glad there is hope for people with it.