Treatment for neurofibromatosis type 1 may involve surgery, radiation therapy, or medication. In some cases, treatment may not be necessary unless symptoms become bothersome.

Neurofibromatosis type 1 (NF1), once known as von Recklinghausen disease, is a genetic disorder caused by a mutation of the NF1 gene. It causes tumors to grow along your nerves and can affect bone development and skin pigmentation.

Many people who have NF1 inherit it from their parents, but mutations in the NF1 gene can also happen randomly. Overall, NF1 affects 1 in 2,600 to 3,000 people.

Symptoms of NF1 can range from mild to severe. In most people, NF1 tumors aren’t cancerous, though cancer is possible. About 10% of people who have NF1 may develop a malignant peripheral nerve sheath tumor (MPNST), which is cancerous.

Because NF1 can affect people differently, treatment will depend on the type of tumor, its location, your specific symptoms, and how NF1 affects your health and daily life.

Surgery is one of the primary treatments used in NF1 when tumors cause symptoms such as pain, bleeding, numbness, or weakness. Your doctor may recommend surgery if you’ve developed MPNST or another NF1-related cancer.

The goal of surgery is to completely remove tumors without causing nerve and tissue damage or a loss of function. But because NF1 tumors can involve multiple layers of tissue and develop in hard-to-reach areas, they can be difficult or dangerous to fully remove.

Some surgical procedures are used in NF1 to address issues unrelated to the tumors, such as bone irregularities or scoliosis (spinal curvature).

Radiation therapy isn’t a first-line treatment for NF1. Because radiation can cause additional DNA damage and tumor growth, doctors reserve this type of treatment for special situations and almost never use it in young children.

Your doctor may add radiation therapy to your treatment plan if they cannot completely remove an NF1 tumor with surgery or if shrinking the tumor before surgery would allow for better removal.

For certain types of tumors, if surgery is not an option, your doctor may consider radiation therapy to help shrink the tumor and reduce its effects on surrounding tissue.

Chemotherapy uses potent drugs to kill tumor cells.

In NF1, doctors use chemotherapy in several ways. They might use it to help shrink NF1 tumors before surgery, to help remove any remaining cancerous cells after surgery, or to help shrink tumors when neither surgery nor radiation is an option.

Your doctor might also recommend a chemotherapy regimen if a cancerous tumor has metastasized (spread to other parts of your body).

Typically, chemotherapy is used in NF1 to treat cancerous tumors, such as MPNST, and tumors that are difficult to operate on, such as optic pathway gliomas, which can develop on your optic nerve.

Kinase inhibitors are medications that block the action of enzymes in your body that are essential to tumor growth and development. In NF1, kinase inhibitors focus on the MEK enzyme and are often referred to as MEK inhibitors.

When the MEK enzyme is blocked, tumor growth in NF1 can slow or stop. Tumors may shrink, and as they do, symptoms can decrease.

The Food and Drug Administration (FDA) has approved two MEK inhibitors for the treatment of NF1.

In 2020, the drug selumetinib (Koselugo) was approved for use in people over 1 year of age who have NF1 with inoperable plexiform neurofibromas (PNs), benign tumors that grow along nerves.

In 2025, the FDA approved mirdametinib (Gomekli) to treat NF1 in people over the age of 2 years who have inoperable PNs.

PNs are one of the most common types of tumors in NF1, occurring in up to 50% of people with the condition.

Your individual treatment plan for NF1 will depend on the type of tumors you have, their location, and the symptoms you’re experiencing.

Each type of treatment comes with risks and benefits. For example, surgery may be able to completely remove an NF1 tumor, but it can pose a risk of tissue damage and loss of function, depending on the tumor’s location.

Radiation and chemotherapy have a long list of possible side effects, such as chronic fatigue, hair loss, and skin changes. Chemotherapy can sometimes lead to anemia or an increased risk of infection. And in rare cases, radiation therapy may increase the risk of developing a secondary cancer.

While MEK inhibitors have been proven safe through rigorous clinical trial testing, about 20% of people experience side effects such as:

  • fatigue
  • skin rashes
  • diarrhea
  • nail infection
  • abdominal pain
  • nausea
  • swelling

In very rare cases, MEK inhibitors can lead to serious complications such as blood clots, heart damage, or intestinal blockage.

Tumor management is just one aspect of NF1 treatment. Your doctor may also recommend therapies that focus on improving your function and overall quality of life.

Physical therapy

NF1 can cause a range of challenges related to movement and motor skills. A physical therapist can help you build up your strength, endurance, coordination, and balance. Physical therapy can also help with flexibility, mobility, and even pain management.

For children with NF1, physical therapy can support proper growth and development.

Monitoring

Not everyone with NF1 needs immediate treatment, and most treatments have unpleasant side effects. If you don’t have any bothersome symptoms, your doctor may take a “wait and see” approach. During this time, they’ll monitor your health and keep close watch for any new or worsening symptoms.

There’s no cure for NF1, and the outlook varies, depending on each person’s individual situation. Moderate to severe NF1 is associated with decreased quality of life and significant changes to appearance and functionality.

According to a 2018 report from the American College of Medical Genetics and Genomics, NF1 is associated with a decrease in life expectancy of 8 to 15 years in comparison with the broader population. But improvements in treatment may help reduce that gap.

Treatments for NF1 depend on your symptoms, the type and location of any tumors you have, and any other complications you experience, such as bone changes or growth irregularities.

While surgery is still one of the first-line treatments for this genetic condition, emerging therapies such as MEK inhibitors now offer treatment options for inoperable tumors.

Not everyone with NF1 needs treatment right away. If you don’t have any significant symptoms, your doctor may take a “wait and see” approach until treatment is necessary.