New MS Pill Puts Hope on the Horizon

Like all drugs, it has side effects. Patients who take it will need to be monitored. And it has not even been approved yet by the U.S. Food and Drug Administration. But Maria Melanson, MD, Clinical Director of the Waddell Center for Multiple Sclerosis, is among those who are clearly excited about the first oral drug for patients with relapsing-remitting multiple sclerosis. An FDA advisory committee recommended approval of the drug, fingolimod, on June 14.

Fingolimod has the potential to improve the lives of many of the Waddell Center’s more than 1,000 patients for two reasons: It will spare patients the discomfort – and in some cases misery – of injections. And, because it is an entirely new drug, it offers hope to patients who do not respond to current medications.

“In September the U.S. Food and Drug Administration will look at fingolimod and make a final decision as to whether the drug is going to get approved or not,” said Dr. Melanson. “This would be the first medication that patients with MS can take by mouth. This means a lot because we have a lot of patients who do not want to take injections. We have patients who have needle-phobia and are not willing to take injections.

“We also have patients who do not respond to the medications that we have available,” Dr. Melanson continued. “The more options we have, the better. This is a completely different therapy. It has a completely different mechanism of action than the medications we currently have. So this is a big step forward.”

According to a report by the National Multiple Sclerosis Society, fingolimod “binds to a docking site (sphingosine-1-phosphate receptor, or S1P) on immune cells, including T cells and B cells,” which cause damage to the nervous system in MS. The drug appears to work by keeping these immune cells corralled in the lymph nodes, where they are relatively harmless, and preventing them from migrating into the brain and spinal cord, where they attack the myelin sheath that covers the nerves. Damage to this myelin covering compromises the nerves’ ability to transmit impulses in a smooth, coordinated manner.

The FDA Advisory Committee recommended that the agency approve marketing of the drug, developed by Novartis, for people with relapsing-remitting MS, a kind of MS that is characterized by relapses followed by periods of stability. Fingolimod reduced relapse rates in two large clinical trials, and one trial suggested that the drug could slow the progression of disability.

Dr. Melanson said that if the FDA approves the drug in September, as expected, fingolimod could potentially become available to patients at the Waddell Center by November or December. But she stressed that the timetable is indefinite.

Safety profiles and monitoring will be required because of the drug’s side effects, Dr. Melanson explained. “There are a lot of services that we will have to put in place before the medication will be available,” she said. “Patients will have to be monitored in the clinic, and physicians will need to be confident starting the medication.”

Side effects documented during two large phase III clinical trials included a temporary reduction in heart rate, a slight elevation of blood pressure, swelling in the retina, elevation of liver enzymes and a small number of serious herpes infections.

“During monitoring we will have to check for heart function, liver function and lung function,” Dr. Melanson said. “Some of the side effects of fingolimod are transient. However, because the medication has been studied for only a short time, we don’t know what to expect long-term. We don’t have years and years of treatment experience to go by. Our knowledge is limited, so we will need to be diligent and to conduct careful monitoring for potential side effects.”

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