Kathy Costello, adult nurse practitioner and associate vice president of clinical care for the National Multiple Sclerosis Society, says several genes have been identified as posing a risk for multiple sclerosis development, but there’s not enough definitive data yet to prevent the disease onset through gene therapy.
There are many new developments in treatment on the horizon, though, including an oral medication that was approved for relapsing forms of multiple sclerosis. Fingolimod, approved for adult MS patients since 2010, received a breakthrough therapy designation from the FDA for children and adolescents ages 10 years and older with relapsing MS.
Other medications are in development that restrict the activation and circulation of immune cells, therefore reducing the inflammatory process in multiple sclerosis.
Another medication to watch is ibudilast, used in Japan and Korea for a variety of issues like post-stroke and bronchial asthma since the 1980s. Researchers are looking into the medication's potential benefit for problems including multiple sclerosis, amyotrophic lateral sclerosis, substance abuse, and neuropathic pain, Costello says. In recent phase 2 clinical studies, patients experienced a significant reduction in the progression of whole brain atrophy. The research may move to phase 3 trials soon and ibudilast has been designated as a fast-track product in terms of its development as a possible treatment for progressive MS, says Costello.
Additional treatments in the research pipeline have a similar focus, by reducing activation of the immune system, reducing the activation of immune cells, or blocking the entry of those cells into the central nervous system.
"They're each coming at the immune system problems from various directions. This is good news for people with multiple sclerosis who may need to switch to a different medication due to a suboptimal response or side effects. Having medications with differing mechanisms of action will allow greater chance to impact the disease process,” says Costello.
There is also interest in how gut microbiota and exercise can impact multiple sclerosis, particularly regarding what exercises are best for stimulating different parts of the brain and improving mobility.
Stem cells transplants may also be an option. "Interim results from a study of hematopoietic stem cell transplant showed the procedure was well-tolerated and there may be benefit to rebooting the immune system," Costello says. "You are essentially suppressing the immune system and then reintroducing the patient's own stem cells, and in multiple sclerosis the hope is they will regenerate in a less harmful way.