Background: Pirfenidone is an oral medication with a number of actions affecting the immune system. It has been proposed as a possible treatment for multiple sclerosis (MS).
Methods: An early-phase study of progressive forms of MS was conducted. Pirfenidone was slowly titrated to 2400 mg/day. Safety, clinical, quality-of-life, and magnetic resonance image (MRI) outcomes were measured.
Results: Twenty people were enrolled (13 with secondary progressive and seven with primary progressive MS). The mean age was 47.7 years; the mean Expanded Disability Status Scale (EDSS) was 5.15; 75% were female. Eighteen patients achieved the full dose, although five additional patients eventually had to decrease the dose, primarily because of nausea. The Neurologic Rating Scale showed a slight worsening, from 69.8+/-8.4 at baseline to 71.8+/-8.9 at one year (P = 0.03). Other clinical outcomes remained stable, including the EDSS, ambulation index, and nine-hole peg test. The Short-Form Health Survey (SF-36) quality-of-life measure remained unchanged. Comparisons of MRI scans at baseline and one year found that 715 plaques were unchanged, six were better, and 10 were worse. Three patients had plaques that improved and two patients had plaques that were worse. There were eight gadolinium-enhancing lesions on the baseline scans and 14 on the one-year scans.
Conclusions: Pirfenidone was well tolerated in patients with MS. Patients with primary progressive or secondary progressive MS tolerated the medication and remained clinically stable during the one year of follow up. Placebo-controlled blinded studies are needed to determine clinical effectiveness.