Treatment

Early detection and treatment improve overall outcomes for neuromyelitis optica spectrum disorder (NMOSD). The number and severity of attacks worsen the outcomes of the disease. Because of this, it is important to get the correct diagnosis and treatment as soon as possible.¹

Neuromyelitis optica treatments can be described as acute (immediate or short-term) or preventative.²

Acute treatments

Acute treatment for NMOSD is used to decrease symptoms of the disease right when they start. Acute treatment includes high-dose steroids and plasma exchange (plasmapheresis, or PLEX).²

High-dose steroids

Steroids (corticosteroids) are strong anti-inflammatory drugs. Your body normally makes steroids in the adrenal glands, but when given in doses larger than the amount your body makes, steroids work to quickly decrease inflammation.³

In NMOSD, high-dose steroids are essential to help prevent vision loss during an attack of optic neuritis (inflammation of the nerve fibers in the eye) or to treat transverse myelitis (inflammation of the spinal cord). Methylprednisolone is the most common steroid given to those with NMOSD.^2,4

By decreasing inflammation, high-dose steroids can help limit long-term, irreversible damage to the spinal cord, optic nerves, and even the brain that can occur during an NMOSD attack.⁴

Plasma exchange

Antibodies are proteins in your body that normally protect you from different germs that can make you sick. In autoimmune diseases such as NMOSD, some antibodies mistake healthy cells for harmful ones and attack them. This leads to different damage and symptoms depending on the location of the attack.⁵

In NMOSD, antibodies that attack a protein called aquaporin-4 (AQP4) can result in damage to nerves and their protective coating in the optic nerve, spinal cord, and brain. While many people with NMOSD have detectable AQP4 antibodies (AQP4-IgG) in their blood, 1 in 4 people do not. This adds to the complexity of the disease.^6,7

Plasma exchange may be an effective treatment in those who have sudden, severe attacks that do not respond to high-dose IV steroids. It involves removing some blood and using a machine to separate the blood cells from the fluid (plasma). Blood cells are then mixed with a replacement solution and returned to the body. This process filters the blood in order to remove harmful immune system antibodies.^8-10

Studies have shown that plasma exchange may improve acute NMOSD symptoms. However, this treatment should be started early for it to be effective. Many times, plasma exchange treatments are given in the hospital for proper monitoring.⁹

Preventative treatments

The goal of preventative treatment is to decrease the number or severity of future attacks. An attack can lead to worsening disability, making it critical to prevent them. Until recently, there were no drugs approved by the US Food and Drug Administration (FDA) for those with NMOSD.¹¹

Biologics are drugs made from living organisms or cells. The process of making biologics turns products made from organisms or cells into drugs that can prevent, treat, or cure diseases. Biologics can include transplant tissues, proteins, antibodies, stem cell therapies, and gene therapies. These are different from most traditional drugs. Traditional drugs are made from chemicals in a lab. Their ingredients are not directly derived from living cells.¹²

As of 2024, the FDA has approved 4 antibody biologic drugs for the treatment of those with AQP4 antibody (AQP4-IgG) positive NMOSD:¹¹

Soliris^® (eculizumab)

Soliris® (eculizumab) is a laboratory-made antibody used to decrease the risk of an NMOSD attack. Soliris is an antibody that targets and inhibits the complement pathway. Activation of this pathway plays a key role in the development of NMOSD and resulting nerve damage. Soliris can increase the risk of certain bacterial infections. The medicine is given by IV infusion, usually every 5 weeks.^13,14

Enspryng™ (satralizumab)

Enspryng™ (satralizumab) is the first approved treatment in NMOSD that was designed to block interleukin-6 (IL-6). IL-6 is a protein made by cells in the body and plays a role in the inflammation that occurs in NMOSD. Enspryng is an antibody that blocks IL-6 from attaching to the cell surface. Enspryng increases the risk of certain bacterial infections. The medicine is given by self-injection once per month. If your doctor prescribes this drug, you will receive training on the safe and correct way to inject it.^15,16

Uplizna™ (inebilizumab)

Uplizna™ (inebilizumab) works by lowering the number of your body’s B cells, which are part of your immune response. Your B cells make antibodies, including anti-AQP4 antibodies in NMOSD. With fewer of these cells, the immune system of those with NMOSD may not react as strongly to certain infections. Uplizna is given twice a year by IV infusion.^17,18

Ultomiris^® (ravulizumab)

Ultomiris® is the most recently FDA-approved NMOSD treatment. Ultomiris is an antibody that targets and inhibits the complement pathway, similar to Soliris. Ultomiris can also increase the risk of certain bacterial infections. The drug is given by IV infusion, usually every 8 weeks.¹⁹

Off-label drug use

For a drug to be approved by the FDA, a company must show data about the safe and effective use of the drug for a specific disease in a clinical trial. All drugs have side effects, so something being “safe” does not mean that it has no side effects. Instead, the FDA decides that an approved drug has more benefits than risks (side effects).²⁰

FDA approval takes time and requires careful evaluation. Sometimes, doctors decide an approved drug may benefit another condition that the drug was not approved to treat. This is called off-label use. It can also be off-label use if a drug is given in a different dose (such as taking 2 pills instead of 1) or in another way (like taking a liquid form instead of a pill) from the dose or way approved by the FDA.²⁰

Off-label uses are common in rare diseases where not many drugs are used to treat the condition. If you and your doctor decide on off-label use of a drug, you should understand that the FDA has not yet declared the drug is safe and effective when used the way you are taking it. However, this does not mean your use of the drug is unsafe. Often, it just means there has not been a large, official clinical trial of this specific drug for this specific rare disease. Talk to your doctor about your treatment questions and needs.²⁰

Some off-label drugs commonly used to treat NMOSD include:^21-26

• Rituxan^® (rituximab), a laboratory-made antibody that lowers B cells
• Mitoxantrone, an anti-cancer drug
• Campath^® and Lemtrada^® (alemtuzumab), which are laboratory-made antibodies
• Imuran^® and Azasan^® (azathioprine), which are drugs that decrease the immune response
• CellCept^® (mycophenolate mofetil), a drug that decreases the immune response
• Trexall^®, Otrexup^®, and Rasuvo^® (methotrexate), which are disease-modifying antirheumatic drugs (DMARDs)
• Actemra^® (tocilizumab), a laboratory-made antibody that targets IL-6