A type of chemotherapy drug, called mitoxantrone, can potentially treat multiple sclerosis (MS) by suppressing blood cells that attack your myelin, the protective layer covering your brain and spinal nerves.

Approximately 400,000 people in the United States have multiple sclerosis (MS). MS doesn’t have a cure, but doctors and healthcare professionals prescribe medications to reduce immune-mediated activity and help people manage their symptoms.

The Food and Drug Administration (FDA) has approved one type of chemotherapy drug to treat MS. Mitoxantrone (Novantrone) is approved for people with:

Mitoxantrone isn’t approved to treat people with primary progressive MS.

Chemotherapy is also sometimes combined with autologous bone marrow transplant to treat MS. In an autologous bone marrow transplant, doctors typically use a different type of chemotherapy drug, such as cyclophosphamide (Cytoxan).

This procedure involves:

  1. removing the stem cells from your bone marrow that produce blood cells
  2. undergoing a high dose of chemotherapy
  3. infusing the stem cells back into your blood

Read on to learn more about how chemotherapy is used to treat MS and when a doctor may recommend it.

What is chemotherapy?

Chemotherapy is a drug therapy that contains chemicals that destroy fast-growing cells in your body. It’s most often used to treat cancer.

Chemotherapy can damage healthy cells in your body that divide quickly such as cells in your hair follicles or gastrointestinal (digestive) tract. Many chemotherapy side effects develop from damage to these healthy cells.

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MS is a progressive disorder that develops when your white blood cells attack the protective fatty layer around the nerves in your brain and spinal cord called myelin and neurodegeneration (cognitive impairment, depression, fatigue) occurs.

The chemotherapy drug mitoxantrone can potentially treat MS by suppressing the activity of white blood cells that attack myelin and reducing damage to your central nervous system.

An emerging therapy for MS is chemotherapy combined with an autologous bone marrow transplant. Combining chemotherapy with a bone marrow transplant allows doctors to administer a higher dose of chemotherapy — typically cyclophosphamide (Cytoxan) — than they otherwise would be able to.

Chemotherapy with an autologous bone marrow transplant may be a treatment option for people with severe relapsing-remitting MS that doesn’t respond to other treatments.

During an autologous bone marrow transplant, stem cells that create blood cells are removed from your bone marrow before you’re given chemotherapy. They’re reinfused into your blood afterward so that your immune system can regrow.

Mitoxantrone, sold under the brand name Novantrone, is the only chemotherapy medication that is FDA approved to treat MS in the United States.

The medication alemtuzumab (Lemtrada) is also FDA approved to treat MS. It’s considered a targeted therapy medication and not a traditional chemotherapy drug. It works by targeting a protein on your white blood cells called CD52. This reaction depletes and then repopulates white blood cells circulating in your blood.

Similar to alemtuzumab, rituximab (Rituxan) is also a targeted treatment that’s not necessarily considered chemotherapy. It’s also used in several different types of cancer treatment. Rituximab isn’t FDA approved for MS, but it’s frequently used off-label to treat MS.

Promising new treatments

Chemotherapy combined with a bone marrow transplant is still considered an experimental procedure and hasn’t yet been FDA approved. Two different drug regimens are frequently used. These include the BEAM-antithymocyte globulin (ATG) regimen, which includes the following chemotherapy drugs:

  • carmustine
  • etoposide
  • cytarabine-arabinoside
  • melphalan
  • ATG

And the cyclophosphamide regimen, which includes:

  • cyclophosphamide
  • ATG

Mitoxantrone is typically given at a dosage of 12 milligrams per square meter (mg/m2) through an intravenous (IV) infusion for 5 to 15 minutes every 3 months. It can be highly toxic, so doctors usually administer a maximum lifetime dose of 140 mg/m2.

The dosages and drugs used in chemotherapy combined with an autologous stem cell transplantation vary between clinical trials. The procedure generally consists of five phases:

  1. pretransplantation identification of potential risks
  2. stem cell removal
  3. chemotherapy
  4. reinfusion of stem cells
  5. supportive care posttransplantation

Chemotherapy drugs can cause many side effects. Congestive heart failure (CHF) is a rare complication that may occur during therapy or months or years after mitoxantrone therapy. The risk of cardiotoxicity increases with your total lifetime dose.

Other side effects can include:

Mitoxantrone has also been linked to an increased risk of developing acute myeloid leukemia.

Learn more about chemotherapy side effects.

Mitoxantrone has been associated with significant side effects. It’s fallen out of favor despite its potential effectiveness for treating MS.

In a 2021 study, researchers compared the drug mitoxantrone with the monoclonal antibody rituximab in people with advanced relapsing MS.

Progression of disability was seen in 21% of people who received rituximab and 32.9% of people who received mitoxantrone. Rituximab had a more favorable safety profile. The researchers concluded that mitoxantrone should only be used in rare and exceptional cases.

Chemotherapy combined with autologous stem cell transplant

Studies are still ongoing looking at the effectiveness of chemotherapy combined with an autologous stem cell transplant. In a 2022 review, researchers found that a stem cell transplant is potentially a highly effective and relatively safe option for highly active MS.

A 2021 study notes that the best candidates for chemotherapy combined with an autologous stem cell transplant are people younger than 50 years old with a shorter duration of MS. Researchers in the study recommend that the procedure only be performed at centers with substantial experience and expertise.

In a 2017 study, researchers found that MS didn’t progress over a 5-year period in 46% of people after thye received an autologous stem cell transplant.

Chemotherapy involves taking drugs that contain chemicals that kill cells in your body that multiply quickly.

The chemotherapy drug mitoxantrone is FDA approved to treat MS. It works by destroying the white blood cells that attack the protective layer around your nerves called myelin.

Chemotherapy combined with a stem cell transplant is an emerging treatment, but it still hasn’t been FDA approved to treat MS. It may be a treatment option for people with severe relapsing-remitting MS.