Kappos L et al. (2006) Oral fingolimod (FTY720) for relapsing multiple sclerosis. N Engl J Med 355: 1124–1140

Multiple sclerosis is considered an autoimmune disease in which autoreactive T cells and macrophages attack myelin sheaths, leading to progressive focal demyelination and neurological disability. In a small trial involving patients with relapsing multiple sclerosis, Kappos et al. have demonstrated the therapeutic effect of fingolimod, a new oral immunomodulating agent.

In total, 281 patients were randomized to receive either 1.25 mg or 5.0 mg of fingolimod or placebo once daily. In a 6-month extension to the trial (months 7–12), those patients receiving placebo were randomized to one of the fingolimod doses (patients and researchers remained unaware of dose assignments).

At the end of the core study, the proportion of patients with gadolinium-enhanced lesions as evidenced by MRI was lower in both fingolimod groups than in the placebo group (P <0.001 for both). In addition, the annualized relapse rate was lower for individuals in both fingolimod groups (0.35/year in those receiving 1.25 mg and 0.36/year in those receiving 5.0 mg) than in those randomized to placebo (0.77/year). Adverse events associated with fingolimod included nasopharyngitis, dyspnea, and headache. During the extension phase, the number of gadolinium-enhanced lesions and relapse rates remained low in those patients who received continuous fingolimod, and at month 12 over 80% of these patients were free of gadolinium-enhanced lesions. Notably, the number of gadolinium-enhanced lesions decreased markedly among those patients who switched from placebo to fingolimod, as did the annualized relapse rate. On the basis of these results, the authors conclude that larger, longer-term studies of fingolimod are warranted.