Mycophenolate mofetil in combination with interferon beta-1a in the treatment of relapsing-remitting multiple sclerosis: a preliminary study

The efficacy of Mycophenolate mofetil (MMF) plus interferon beta-1a (IFNB-1a) in treatment of relapsing-remitting multiple sclerosis (RRMS) was evaluated. This was a pilot study with randomized, double-blinded, placebo-controlled design. Patients with RRMS and Expanded Disability Status Scale score (EDSS) of < 6.0 were included. Those with relapse within the previous two months and prior use of immunomodulatory/immunosuppressive drugs were excluded. Patients were randomized into MMF (n = 13) and placebo (n = 13) groups and received weekly intramuscular IFNB-1a plus either MMF or placebo. MMF started by 500 mg/d for one week and weekly escalated by 500 mg/d, until target divided dose of 2000 mg/d and continued for 12 months. Radiologic and clinical assessments were performed at baseline and then at month 12. After one year of therapy, difference between the two groups in number of new T2 lesions was not statistically significant (0.54 ± 0.77 in MMF vs. 1.85 ± 3.2 in placebo group, p = 0.169). Two patients in the placebo group had gadolinium-enhanced lesions and one patient had relapse. There were 3 patients in each group with more than one point progression in EDSS. Common side effect in the MMF group included gastrointestinal upset, but no patient discontinued the treatment. Combination of MMF with IFNB-1a in patients with RRMS is well tolerated, but the efficacy of such combination was not statistically significant in this pilot study and deserves further investigation with a larger sample size and a longer follow-up.