For decades, people with multiple sclerosis have injected themselves daily with immunosuppressant drugs for lack of a better option, in hopes of mitigating the disease's neurodegenerative effects. But newly designed oral therapies for the disorder have proliferated over the past few years, foretelling a future with fewer pricks.

“People are very excited about having a pill instead of an injection,” says Todd Eagar, an immunologist at the University of Texas Southwestern Medical Center in Dallas. “Those daily injections are just really taxing on patients.”

The first immunomodulating pill to treat multiple sclerosis, Gilenya (fingolimod), manufactured by Swiss drug giant Novartis, gained approval from the US Food and Drug Administration (FDA) last year. And results announced last month hint that there's more on the way.

France's Sanofi published phase 3 trial results from its lymphocyte inhibitor Aubagio (teriflunomide) on 6 October showing similar efficacy as established injectable treatments (N. Engl. J. Med. 365, 1293–1303, 2011). The day before, US-based Biogen Idec announced phase 3 trial data for its twice-daily pill BG-12 (dimethyl fumarate). “It has shown unexpectedly promising data,” says Jon Searles, a senior analyst at the research and consulting firm Decision Resources in Burlington, Massachusetts. “Clinicians are impressed with the drug's efficacy on clinical endpoints of relapses and disability.”

Amidst the advances come some notable stumbles, though. In June, Germany's Merck KGaA dropped its pill Movectro (cladribine) after the FDA demanded additional trials. Meanwhile, laquinimod, under development by Israel-based Teva and Sweden's Active Biotech, has hit a snag: trial results released in August showed no difference in relapse rate compared to placebo. Despite the drug's poor showing, however, laquinimod's developers have not followed Merck's suit, because the pill promises more than standard immunosuppression: mouse studies suggest it may also protect the brain from further degeneration through other mechanisms, as well.

Biogen Idec and Novartis have also released data from rodent studies suggesting neuroprotective qualities of BG-12 and Gilenya, respectively. “A big unmet need is going to be strategies to repair damage, and it's one thing that might distinguish one drug from another,” says Jeffrey Cohen, a neurologist at the Cleveland Clinic in Ohio who was involved with the development of Gilenya.

Easy to swallow: Oral MS options. Credit: istockphoto

Ultimately, doctors may suggest mixing and matching oral drugs for multiple sclerosis to combine various mechanisms of action. But comparative and combination studies won't begin until—and if—the drugs are approved. And, because the mechanisms aren't well understood, clinicians are anxious about experimentation. Nonetheless, they wait with bated breath for the pipeline's outflow.

“If they all get through the regulatory approval process, then, if nothing else—even if we don't have comparative data—then we'll have a lot of choices,” says Fred Lublin, a neurologist at Mount Sinai Medical Center in New York.