Each week 200 or more Americans are diagnosed with multiple sclerosis, according to the National Multiple Sclerosis Society. The autoimmune disease causes the body's natural defenses to attack the central nervous symptom, resulting in symptoms that range from mild, including numbness in the limbs, to severe symptoms like disability, loss of vision or paralysis. Most people are diagnosed between 20 and 50, NMSS says.
There are four types of MS, with 85 percent of patients initially diagnosed with what's called relapsing-remitting MS. People with relapsing-remitting MS often have attacks - called relapses, flare-ups or exacerbations - that may worsen neurological function, which are followed by partial or complete recovery (remission) periods.
The trials for BG-12 were phase III, which is often the step closest to a drug's approval when scientists gather more data on its safety and efficacy. BG-12 is manufactured by Massachusetts based-Biogen Idec, which is seeking FDA approval on the drug. The drugmaker funded the new studies, which were published online Sept. 20 in the New England Journal of Medicine.
The first study - known as the DEFINE trial - looked at 1,200 people to determine whether a BG-12 pill could reduce the frequency of relapses compared to people with MS who were taking a placebo pill.
Participants were split into three groups: those taking 240 milligrams of the pill twice a day, those taking 240 milligrams three times a day and those taking a placebo. At two years, both groups taking BG-12 experienced significant reductions in relapses (49 percent and 50 percent reductions respectively) compared with placebo-takers.
Taking BG-12 also led to significant reductions in risk of MS progressing to disability for the patients. MRI scans confirmed pill-takers had fewer new or active MS lesions than scans of people taking placebo.
The next trial, led by Dr. Robert J. Fox, medical director at the Cleveland Clinic's Mellen Center for Multiple Sclerosis, included 1,400 people with MS to determine whether BG-12 could reduce the average yearly relapse rate for patients at two years.
Similar to DEFINE, this study - known as the CONFIRM trial - measured relapse rates of patients taking either a 240 milligram dose of BG-12 twice a day or three times a day, comparing them a group taking placebo. A fourth group was assigned to receive the injectable MS drug, glatiramer acetate, which is sold as Copaxone and was FDA-approved in 1996 to reduce the frequency of relapses in patients with relapsing-remitting MS.
The results showed that patients taking BG-12 twice daily reduced their relapses by 44 percent at one year and those taking it three times per day reduced it by 51 percent, compared with placebo takers. People taking glatiramer acetate reduced their risk by 29 percent, but the study was not designed to compare that drug with the two experimental doses of BG-12. The study did not show a reduction in progression to disability though between BG-12 groups and the placebo group.
"We don't have a cure yet," Dr. Fox told, "but we have gotten that much closer to getting good control of this disease."
In both studies, side effects included flushing (saddening redness of the skin) and gastrointestinal issues like diarrhea, nausea, and upper abdominal pain.
"You have to characterize it as a miracle," 49-year-old Stephen O'Malley, a Cleveland father of two who has been enrolled in the experimental treatment, told CBS This Morning. "I feel healthier than I felt seven years ago when I got diagnosed."
Recently, athat the commonly prescribed injection, interferon beta, did not stop the progression of MS, but may benefit patients in other ways including reducing relapse.
Last week, the FDA approved Sanofi Aventis' once-daily tablet
"This is an exciting time in the development of new drugs for MS, especially considering the fact that we had no effective treatments just a few decades ago," NMSS chief research officer Dr. Timothy Coetzee told WebMD. But he added that some people do not respond to certain treatments, so the new oral drugs may not replace standard first-line therapies.
"My guess is that the injectables will remain an important treatment option," Coetzee said. "The more options we have the better."