How Neuromyelitis Optica Spectrum Disorder Is Treated

Treatment for neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disorder affecting the central nervous system, involves managing symptoms and preventing relapses.

With NMOSD, the immune system will attack healthy nerve tissues, most commonly in the spinal cord and optic nerve. This can lead to an array of potentially severe symptoms, including vision loss, limb paralysis, and the loss of bladder or bowel control.

Some treatments for NMOSD target the main antibody involved in the autoimmune assault, called anti-aquaporin-4 (AQP4) autoantibody. Others target the inflammation that arises from the assault.

This article describes the different medications used to treat NMOSD, as well as blood-based procedures that can help temper the autoimmune response. It also looks at whether dietary changes can help support the medical treatment of NMOSD.

Prescriptions

Prescription medications are the first line of treatment for neuromyelitis optica spectrum disorder. There are three main goals of NMOSD treatment:

• To treat acute relapses (flare-ups that develop rapidly and severely)
• To prevent future relapses
• To treat the various symptoms caused by the autoimmune assault on the spinal cord, optic nerve, or brain

Different drugs would be prescribed to address each of these concerns.

Treatment of Acute Relapses

During an acute NMOSD relapse, the first goal is to quickly reduce inflammation. This limits any injury to the central nervous system and helps alleviate symptoms. There are several medications that can be used for this purpose.

Medrol (Methylprednisolone)

Medrol (methylprednisolone) is a corticosteroid drug used in the first-line treatment of an acute NMOSD relapse. Corticosteroids (a.k.a. steroids) work by suppressing the immune system and rapidly bringing down the inflammation.

Medrol is taken by mouth in tablet form. The recommended dose is 1 gram taken once daily for three to five days.

Common side effects include nausea, vomiting, heartburn, headache, dizziness, trouble sleeping, appetite changes, increased sweating, and acne.

Intravenous Immunoglobulin Therapy

If a person with NMOSD does not respond to methylprednisolone, a treatment called intravenous immunoglobulin (IVIg) therapy may be pursued.

IVIg therapy involves a mixture of various immune proteins, called antibodies. They are delivered into the bloodstream through a needle in your arm. It may be given alone or used in combination with an immunosuppressive drug called Imuran (azathioprine).

IVIg therapy is not yet fully supported by medical research. However, a small study conducted in 2014 reported that it was effective in five of 10 participants with NMOSD who did not respond to corticosteroids.

Side effects include headache, flushing, chills, muscle pain, wheezing, rapid heartbeats, lower back pain, dizziness, and nausea.

Cytoxan (Cyclophosphamide)

Another option is a chemotherapy drug called Cytoxan (cyclophosphamide), which is used to treat different types of cancer. Studies suggest that Cytoxan, delivered intravenously, may enhance the effects of methylprednisolone in people with NMOSD.

Side effects include nausea, vomiting, diarrhea, flushing, sweating, itching, stomach pain, temporary hair loss, and rash.

Prevention of Acute Relapses

In 2019, the U.S. Food and Drug Administration (FDA) approved Soliris (eculizumab), an intravenous drug used specifically for the treatment of NMOSD. It was the first such drug approved for this use. 

Soliris is a man-made antibody, called a monoclonal antibody, that is engineered to block the action of anti-AQP4. It is only approved for use in people with NMOSD who test positive for anti-AQP4.

Soliris is given intravenously once weekly for five weeks and every other week thereafter to ensure the sustained control of NMOSD.

A 48-week study published in the New England Journal of Medicine reported that, among 143 people with NMOSD who tested positive for anti-AQP4, those given Soliris had a 98% reduction in the number of acute relapses. Moreover, when relapses occurred, they tended to be milder and less likely to require hospitalization.

Side effects include headache, tiredness, fatigue, nausea, vomiting, diarrhea, muscle pain, back pain, sneezing, sore throat, and stuffy nose.

Some immunosuppressive drugs can reduce the immune system's attack on healthy nerves and prevent severe relapses of NMOSD. These therapies may not entirely prevent relapses, but they may reduce the severity of an attack that might otherwise cause permanent nerve damage.

Options include:

• CellCept (mycophenolate mofetil)
• Imuran (azathioprine)
• Rituxin (rituximab)

Common side effects of immunosuppressive drugs include nausea, vomiting, diarrhea, and an increased risk of infection.

Treatment of Symptoms

The symptoms of NMOSD can vary by the part of the central nervous system it affects. For instance, NMOSD in the spinal cord can cause muscle weakness, limb paralysis, muscle spasms, and loss of bowel or bladder control.

NMOSD affecting the optic nerve can cause visual disturbances, loss of color vision, and vision loss. NMOSD in the brain can cause depression, spasms, pain, and uncontrollable hiccups or vomiting.

Among the drugs commonly used to treat NMOSD symptoms are:

• Tegretol (carbamazepine): This anticonvulsant drug decreases nerve impulses. It may be given in low doses to control spasms caused by NMOSD.
• Gablofen (baclofen) or Zanaflex (tizanidine): These are antispasmodic drugs used to treat permanent spasticity (abnormal muscle tightness) caused by NMOSD.
• Amitriptyline or Cymbalta (duloxetine): These are antidepressants used to treat depression caused by NMOSD lesions in the brain.
• Ultram (tramadol): This is an opioid painkiller that can help control NMOSD-induced pain.
• Oxytrol (oxybutynin) or Detrol (tolterodine): These are bladder relaxants that may help people with neurogenic bladder (the loss of bladder control due to nerve-related injuries).

Specialist-Driven Procedures

There are specialist-driven procedures that may be used to support drug therapies for NMOSD. They may also be used on their own when other treatment options fail. Both involve procedures that reduce the amount of anti-AQP4 autoantibodies in the blood so that relapses are less frequent or severe.

Plasma Exchange

For people with NMOSD who do not respond to methylprednisolone, there is a procedure called plasma exchange. This removes the fluid part of your blood, called plasma, and replaces it with substitute fluid. The blood is then returned to the body with less anti-AQP4 in it.

Plasma exchange is done using a machine called a cell separator. The blood is drawn from a vein in one arm. After the plasma is removed and substitute fluid is added, the blood is returned to the body through a vein in the other arm.

Side effects of plasma exchange include dizziness, numbness, tingling, and fainting. These tend to resolve within minutes or hours of completing the procedure.

Plasmapheresis

Plasmapheresis is another procedure aimed at removing anti-AQP4 autoantibodies from the blood. Plasmapheresis involves many of the same processes as plasma exchange but doesn't completely replace the plasma. Instead, the plasma may be manipulated or "cleaned" before being returned to the body.

A 2013 study found that plasmapheresis was well tolerated in people with NMOSD. Fifty percent of those who had undergone plasmapheresis had a significant improvement immediately after the procedure.

Home Remedies and Lifestyle

There are no home remedies or lifestyle changes that can treat NMOSD in any way. However, a diet rich in fatty acids is thought by some to be of benefit.

In 2018, researchers from Iran examined the brain and spinal cord scans of 126 people with multiple sclerosis (MS) and 68 people with NMOSD.

The researchers compared these images to the dietary information provided by each of the study participants. They concluded that the high intake of "bad" saturated fatty acids is linked to the worsening of central nervous system injuries caused by MS and NMOSD.

They found that eating "good" polyunsaturated fats and limiting saturated fats were associated with less fatigue and disability in people with NMOSD. Polyunsaturated fats can be found in foods like salmon, avocados, olives, and olive oil, while saturated fats are found in foods with animal fat.

Summary

Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disease mainly affecting the spinal cord and optic nerve. The treatment of NMOSD is focused on treating acute relapses, preventing future relapses, and treating the various symptoms caused by the autoimmune assault.

Medrol (methylprednisolone) remains the first-line treatment for acute NMOSD relapses. Future relapses can be prevented with a drug called Soliris (eculizumab) that blocks the action of the antibody involved in the autoimmune assault. Newer drugs like Enspryng (satralizumab-mwge) and Uplizna (inebilizumab-cdon) offer a similar mechanism of action.

If medications fail to provide relief from acute relapses, procedures like plasma exchange and plasmapheresis can remove harmful antibodies from the blood.

A Word From Verywell

Neuromyelitis optica spectrum disorder is a chronic, debilitating disease that has no cure. But, as with other incurable diseases, there are emerging therapies that may one day lead to either a cure or an effective chronic therapy.

Until then, work closely with your healthcare provider to find the treatment options that work best for you as an individual. Keep appraised of developing NMOSD treatments and consider participating in clinical trials if your current treatment options are limited.

Most importantly, build a support network of people who understand what NMOSD is and what they can do to help you maintain the highest possible quality of life.