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Open-label study of pirfenidone in patients with progressive forms of multiple sclerosis
James D Bowen1, Kenneth Maravilla, Solomon B Margolin
1Department of Neurology, University of Washington, Seattle, Washington, USA. jbowen@u.washington.edu
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.
Insights
Pirfenidone, an immune-modulating drug, was explored as a multiple sclerosis (MS) treatment. While generally well-tolerated, it did not show significant clinical improvement in this early-phase study.
Area of Science:
- Neuroimmunology
- Clinical pharmacology
Background:
- Pirfenidone is an oral immunomodulatory medication.
- It has been investigated as a potential therapy for multiple sclerosis (MS).
Purpose of the Study:
- To evaluate the safety and tolerability of pirfenidone in patients with progressive multiple sclerosis.
- To assess the clinical, quality-of-life, and MRI outcomes of pirfenidone treatment.
Main Methods:
- An early-phase study involving patients with primary progressive MS and secondary progressive MS.
- Pirfenidone was administered with slow dose titration up to 2400 mg/day.
- Safety, clinical status (EDSS, NRS), quality of life (SF-36), and MRI lesions were monitored over one year.
Main Results:
- Pirfenidone was generally well-tolerated, with nausea being the primary reason for dose reduction in some patients.
- The Neurologic Rating Scale showed a slight worsening (P=0.03), while other clinical measures like EDSS remained stable.
- MRI data indicated minimal changes in lesion count and activity over the study period.
Conclusions:
- Pirfenidone was tolerated by patients with progressive MS, maintaining clinical stability for one year.
- Further placebo-controlled, blinded studies are necessary to establish the definitive clinical efficacy of pirfenidone for MS treatment.