Medications Used Off-Label
Mycophenolate Mofetil
immune system attack." c-nmssatomrichtext_nmssatomrichtext-host=""> Mycophenolate mofetil is an immunosuppressant (stops an immune response) taken by mouth twice daily. It works by blocking an enzyme that certain white blood cells — specifically B- and T-cells — need to carry out an immune system attack.- Mycophenolate mofetil has been studied as a monotherapy (use of a single drug to treat a disease or condition) and in combination with other DMTs, such as interferon-beta medications. The results suggest that mycophenolate mofetil may reduce the annual number of MS relapses, limit new areas of central nervous system (CNS) damage and slow disease worsening.
- Side effects and risks include increased risk of infection [including opportunistic infections, such as progressive multifocal leukoencephalopathy (PML)], nausea, diarrhea, stomach pain, weakness, dizziness, difficulty sleeping, increased risk of skin cancer and lymphoma, stomach ulcers and bleeding, elevation in liver enzymes, and yellowing of the skin (jaundice). It can also cause fetal death or malformations.
Cyclophosphamide
- Cyclophosphamide is an immunosuppressant medication that is given intravenously (into a vein) or orally. It works by binding to cell DNA and interfering with cell division and replication.
CNS inflammation and a variable effect on disease worsening. Cyclophosphamide may be given as part of the chemotherapy regimen included in aHSCT. aHSCT is also not FDA-approved to treat MS." c-nmssatomrichtext_nmssatomrichtext-host=""> Several clinical trials in people with MS demonstrated a reduction in relapses, fewer new areas of CNS inflammation and a variable effect on disease worsening. Cyclophosphamide may be given as part of the chemotherapy regimen included in aHSCT. aHSCT is also not FDA-approved to treat MS.- Side effects and risks include nausea, vomiting, hair thinning/loss, low white blood cell count, risk of infections, risk of cancers, infertility, inflammation of the bladder with bleeding, and fetal abnormalities.
Azathioprine
- Azathioprine is an oral immunosuppressant that targets activation, proliferation (growth), and differentiation of both T and B lymphocytes (type of white blood cell).
- Azathioprine has been used off label in MS for over 30 years. Several clinical trials of azathioprine as a monotherapy and in combination with other DMTs have demonstrated an effect on relapse reduction, new CNS inflammation and disease worsening.
- Side effects and risks include abdominal pain, severe nausea, vomiting, loss of appetite, mouth sores/ulcers, increased risk of infection, hair loss, change in hair color and texture, risk of malignancies and blood abnormalities, and fetal abnormalities.
Minocycline
- Minocycline has been studied in MS in combination with interferon beta-1a (Rebif) and in combination with glatiramer acetate (Copaxone) to determine whether adding minocycline to these DMTs provides added benefit.
- Interferon beta-1a plus minocycline was found to be no more effective than interferon beta-1a plus placebo in time to first relapse, annualized relapse rate, number of new or enlarging T2 lesions on MRI, or change in brain volume.
- Minocycline plus glatiramer acetate was found to be safe and well-tolerated and reduced the number of T1 gadolinium-enhanced lesions, the total number of new and enlarging T2 lesions, and the total T2 burden of disease compared to minocycline plus placebo.
- Side effects and risks include gastrointestinal problems, liver damage, mild to severe skin conditions, respiratory problems, kidney toxicity, muscle and joint pain, blood cell abnormalities, CNS disorders and potential for fetal abnormalities.
Rituximab
- Rituximab is a monoclonal antibody (a protein made in the laboratory) that targets a specific protein on the surface of B-lymphocytes (white blood cells known to cause inflammation and damage in MS). It is given intravenously, with the first dose in 2 IV infusions 2 weeks apart. The dosing regimen is then repeated every 6 months.
- Several clinical trials have demonstrated that rituximab is effective in reducing clinical relapses and limiting new inflammation in the CNS. It may also limit progression (worsening) of MS.
- Side effects and risks include infusion reactions (symptoms that occur during or soon after an infusion), infections (including opportunistic infections, such as PML), allergic reactions, headache, fatigue and anemia. Rituximab should be used with caution during pregnancy.
Statins
- Statins are oral medications that are FDA-approved to lower cholesterol.
clinically isolated syndrome (CIS).One statin — simvastatin — was found to significantly reduce the annualized rate of brain atrophy (loss of brain cells called neurons) compared to placebo in secondary progressive multiple sclerosis (SPMS). Simvastatin was also found to be safe and well-tolerated.Continued research is underway looking at statin use in MS." c-nmssatomrichtext_nmssatomrichtext-host=""> Statins were not found to have a benefit as an add-on to beta-interferon medication. They were also not found to reduce disease activity in people with relapsing-remitting MS or clinically isolated syndrome (CIS).One statin — simvastatin — was found to significantly reduce the annualized rate of brain atrophy (loss of brain cells called neurons) compared to placebo in secondary progressive multiple sclerosis (SPMS). Simvastatin was also found to be safe and well-tolerated.Continued research is underway looking at statin use in MS.- Side effects and risks include headache, difficulty sleeping, flushing of the skin, muscle aches, inflammation and potentially severe muscle and kidney damage, weakness, drowsiness, nausea and vomiting, abdominal cramping or pain, bloating or gas, diarrhea, constipation, rash, and fetal abnormalities.