• Nusinersen, sold as Spinraza, is approved for both adults and children with spinal muscular atrophy (SMA), although most research has focused on how the drug affects children.
• One new study found that nusinersen was safe and could help improve motor function for adults living with SMA.
• In another new study, adults with SMA reported that nusinersen helped improve quality of life.

About one out of three people with SMA are adults, but more is known about how this condition and its treatments affect children. Spinraza — a form of nusinersen injected into the fluid around the spinal cord — is approved by the U.S. Food and Drug Administration (FDA) for people of all ages with SMA. However, the clinical trials that led to the drug’s approval were based on studies in infants and children up to the age of 12.

Two studies published recently further explored nusinersen treatments in adults. This new research helps demonstrate that nusinersen is a safe therapy that can improve motor function and well-being in adults living with SMA.

Learn more about how gene therapies like nusinersen work.

Nusinersen in Adults: Effectiveness and Safety

In the first study, researchers wanted to know more about nusinersen’s ability to treat SMA symptoms in adults and find out more about its safety and side effects. They combined data from 12 different studies that included more than 400 adults diagnosed with SMA.

The researchers found that nusinersen led to improvements in motor function. Study participants who took the drug had higher scores on tests like the Hammersmith Functional Motor Scale Expanded and the Revised Upper Limb Module.

Some data also showed that nusinersen led to improvements in muscle strength, although other research could not detect any changes in this area. Additionally, nusinersen did not seem to lead to major changes in endurance or lung function.

This study also found that nusinersen was generally safe. About 2 percent of participants — or one out of 50 people — experienced serious side effects. These included:

• Post-lumbar puncture syndrome — A headache-causing condition that can arise following nusinersen injection
• Kidney problems, including infection, pain, and kidney damage
• Bacterial meningitis — A severe infection of the membranes that surround the brain and spinal cord
• Skin infections
• Seizures

Overall, about 3 percent of adults in the studies decided to stop using nusinersen, either because of side effects or because they didn’t notice any improvement in SMA symptoms.

Nusinersen and Quality of Life

In the second study, Italian researchers measured how nusinersen affected adults’ quality of life. They collected questionnaires from 78 adults with SMA type 2, 3, or 4. These questionnaires asked participants to reflect on how their SMA affected several different areas of life, such as:

• Relationships with a partner, family, friends, or others
• Social activities, such as visiting friends
• Daily activities, like personal care, housework, or running errands
• Work
• Independence
• Body image
• Thoughts about the future
• Disease symptoms, including weakness, pain, or tiredness

Researchers found that after 14 months of taking nusinersen, participants’ quality of life improved. The study participants reported feeling better about all of the above areas of life, except for certain disease symptoms (pain and muscle locking). Researchers also found that the study participants had better motor function after taking nusinersen.

Is Nusinersen Worthwhile for Adults?

It can be challenging for adults with SMA to find specialists who work with people in older age ranges. Obtaining health insurance that covers treatments can also be difficult. Additional research into treatments for adults with SMA, including the studies above, may help more people access effective treatments.

Although more research was needed to fully understand nusinersen’s effects on adults, these studies show that the drug is promising. “Our meta-analysis confirms that nusinersen is a valuable treatment option for adult patients with longer-disease duration,” wrote the authors of the first study.

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