Multiple Sclerosis Drug Shows Promise in Clinical Setting

Fingolimid (Gilenya, Novartis), an oral compound approved for the treatment of relapsing-remitting multiple sclerosis (RR-MS), was found tolerable and safe in patients, researchers reported in PLoS One.

Maria Rasenack of the Department of Neurology, University Hospital Basel and colleagues conducted a retrospective, non-randomized, open-label, observational study in 105 patients with RR-MS and measured cytokines in longitudinal serum samples.

Compared with the year before fingolimod was started, the annualized relapse rate was reduced by 44%. Also, the percentage of patients with a worsening of the Expanded Disability Status Scale decreased.

Accordingly, the fraction of patients with no evidence of disease activity (no relapse, stable EDSS, no new active lesions in MRI) increased from 11% to 38%. The efficacy and safety were comparable between highly active patients or patients with relevant comorbidities and our general patient population.

"The efficacy in reducing relapses was comparable to that observed in the phase III trials," the researchers wrote. "In our cohort fingolimod was safe and efficacious irrespective of comorbidities and previous treatment."

The drug is a sphingosine 1-phosphate (S1P)-receptor modulator which binds to the S1P-receptors and induces their downregulation on the cell surface. This downregulation leads to a sequestration of lymphocytes in lymph nodes which has been proposed to be the major mode of action.

Researchers have been concerned about possible effects on cardiac conduction and immune response.

"Since patient groups with certain cardiac risk factors were excluded from pivotal trials, the safety and tolerability of fingolimod in these patient groups is less studied," researchers wrote. “It is obvious that a different patient population is treated in clinical practice compared to clinical trials, which may influence tolerability and efficacy of the drug.”

That concern is the reason assessing the drug's impact in routine clinical practice is important.

Potential study limitations are that the "data on efficacy are to be seen with caution because of the lack of a control group, the lack of blinding, and the limited follow-up," they said.