Which medications in the drug class Immunosuppressants are used in the treatment of Multiple Sclerosis?

Updated: Oct 08, 2019
  • Author: Christopher Luzzio, MD; Chief Editor: Jasvinder Chawla, MD, MBA  more...
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Immunosuppressants are used for their ability to suppress immune reactions. Agents such as methotrexate have shown some effectiveness in delaying progression of impairment of the upper extremities in patients with secondary progressive MS. Azathioprine has been studied in clinical trials and has shown modest effects on relapses and progression of disease. Methotrexate and azathioprine have not been approved by the US Food and Drug Administration for use in MS. Antineoplastic agents with immunosuppressive properties, such as cyclophosphamide and mitoxantrone, have been used in patients with MS.

Azathioprine (Azasan, Imuran)

This immunosuppressive antimetabolite drug is an imidazolyl derivative of 6-mercaptopurine. It is cleaved in vivo to mercaptopurine and converted to 6-thiouric acid by xanthine oxidase. Azathioprine is generally used in the treatment of transplant rejection or severe, active, erosive rheumatoid arthritis, but it has been used off-label for MS.

Methotrexate (Rheumatrex, Otrexup, Rasuvo, Trexall, Xatmep)

Methotrexate interferes with DNA synthesis, repair, and cellular replication. It inhibits dihydrofolic acid reductase, which participates in the synthesis of thymidylate and purine nucleotides. Methotrexate has been used off-label for MS.


Mitoxantrone is an immunosuppressive agent approved for the treatment of secondary progressive or aggressive relapsing-remitting MS. It is used for reducing neurologic disability and/or the frequency of clinical relapses in patients with secondary (long-term) progressive, progressive relapsing, or worsening relapsing-remitting MS (ie, patients whose neurologic status is significantly abnormal between relapses). Mitoxantrone is not indicated in the treatment of patients with primary progressive MS.

Mitoxantrone therapy can increase the risk of developing secondary acute myeloid leukemia (AML) in MS patients and in patients with cancer.


Cyclophosphamide has been used for the treatment of progressive MS. Evidence of benefit is mixed. This agent has not been approved for MS but has been used off-label in MS patients. Cyclophosphamide is associated with leukemia, lymphoma, infection, and hemorrhagic cystitis.

Cladribine (Mavenclad)

Cladribine is a prodrug of the active moiety Cd-ATP. The mechanism by which it is used for multiple sclerosis is not fully elucidated, but is thought to involve cytotoxic effects on B and T lymphocytes through impairment of DNA synthesis, resulting in depletion of lymphocytes. It is indicated for relapsing forms of multiple sclerosis (MS), to include relapsing-remitting disease and active secondary progressive disease; because of its safety profile, use is generally recommended for patients with inadequate response to, or inability to tolerate, alternate indicated drugs.

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