Drugs Used in MS That Aren't Approved For MS

Azathioprine (Imuran™)

• Immunosuppressant; disrupts the synthesis of DNA and RNA and thus targets cells (e.g., stimulated lymphocytes) that are dividing rapidly
• Used to prevent rejection of kidney transplants; also used to treat lupus, severe rheumatoid arthritis, and colitis
• Administered orally or via injection
• Adverse effects: increased risk of cancer, lowering of the white blood cell count/bone marrow suppression, serious infections, loss of fertility in men, liver toxicity, severe GI symptoms
• MS: tried in conjunction with steroids and IFN-b, may limit progression and lesion formation in RRMS and SPMS

Cladribine (Leustatin™)

• Cancer drug (chemotherapeutic) of the group known as purine analogues; is incorporated into the DNA of cells such as lymphocytes where it causes strand breaks leading to inhibition of synthesis and cell death
• Used to treat hairy cell leukemia
• Administered via IV injection
• Adverse effects: increased risk of bone marrow suppression and infection, neurological damage (paraparesis, quadriparesis) and kidney damage
• MS: small number of studies, possible benefit in SPMS and RRMS

Cyclophosphamide (Cytoxan™)

• Cancer drug (chemotherapeutic), of the group known as alkylating agents
• Blocks cell growth and suppresses the immune system by cross-linking cellular DNA
• Can be administered orally or via IV
• Adverse effects: increased risk of cancer, loss of fertility (men and women), cystitis, serious infections
• MS: typically given in pulses (e.g., monthly); may be beneficial in combination with IFN-b

Cyclosporine (Neoral™, Sandimmune™)

• Immunosuppressant, used to prevent rejection of kidney, liver, and heart transplants
• Enters T cells and binds to cyclophilin causing inactivation of calcium dependent activation of the cell
• Also used to treat conditions such as rheumatoid arthritis, psoriasis
• Administered orally or via injection
• Adverse effects: increased risk of infection, high blood pressure, kidney and liver problems, cancer, tremors, seizures
• MS: consensus is that risks outweigh benefits (AAN, MS Council for Clinical Practice Guidelines advise against using it)

Intravenous immunoglobulin G (IVIg) (Gamimune N™; Gammagard™; Gammar-IV™; Iveegam™; Sandoglobulin™; Venoglobulin-I™)

• Ig antibodies collected from the plasma of human donors
• Used to treat immunological deficiencies (e.g., severe combined immunodeficiency or SCID), autoimmune disorders
• Administered via IV or IM injection
• Adverse effects: increased risk of kidney disease, most side effects mild and/or uncommon
• MS: studies indicate it may be helpful in RRMS (relapse rate and disability) and CIS (development of CDMS)

Methotrexate (Folex™, Rheumatrex™, Methotrexate LPF™, Trexall™)

• Cancer drug (chemotherapeutic), of the group known as antimetabolites
• Binds to an enzyme (dihydrofolate reductase) which is required for tetrahydrofolate synthesis, a necessary step for DNA synthesis and cellular replication
• Also used to treat conditions such as psoriasis and rheumatoid arthritis
• Administered orally or via injection
• Adverse effects: increased risk of liver and kidney toxicity, decreased white blood count, infection, cancer such as leukemia
• MS: may stabilize progression and/or reduce relapses; has been tried in combination with IFN-b

Minocycline (Minocin™, Dynacin™)

• Antibacterial of the tetracycline family
• Prevents bacteria from multiplying by binding to the 30S subunit of the ribosome; also has anti-inflammatory effects
• Used to treat pneumonia, acne, and other infections, as well as rheumatoid arthritis
• Can be administered orally or via IV
• Adverse effects: Increased sensitivity to sunlight, mostly minor side effects such as dizziness
• MS: one small study (10 RRMS cases) showed reduction in lesion activity