FDA approves first oral drug to reduce MS relapses

The U.S. Food and Drug Administration has approved Gilenya capsules (fingolimod) to reduce relapses and delay disability progression in patients with relapsing forms of multiple sclerosis (MS).

“Gilenya is the first oral drug that can slow the progression of disability and reduce the frequency and severity of symptoms in MS, offering patients an alternative to currently available injectable therapies,” said Russell Katz, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research.

Gilenya is the first in a new class of drugs that block some blood cells in lymph nodes, reducing their migration to the brain and spinal cord, which may help with reducing the severity of MS.

MS is a chronic, often disabling, disease that affects the central nervous system—the brain, spinal cord, and optic nerves. According to the National Multiple Sclerosis Society, there are about 400,000 people in the United States and 2.1 million people worldwide with MS.

The progress, severity, and specific symptoms of MS are unpredictable and vary from one person to another. Symptoms can be mild, such as numbness in the limbs, or severe, such as paralysis or loss of vision.

Patients using Gilenya should be monitored for a decrease in heart rate upon starting the drug. Gilenya may also increase the risk of infections. Cases of serious eye problems (macular edema) have occurred in patients taking the drug and an ophthalmologic evaluation is recommended.

The most frequent adverse reactions reported by patients taking Gilenya in clinical trials include headache, influenza, diarrhea, back pain, elevation of certain liver enzymes and cough.

The drug will be available in 0.5 milligram capsules. Gilenya is made by Novartis, Basel, Switzerland.

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FDA Approves Ampyra to Improve Walking in Adults with Multiple Sclerosis

The U.S. Food and Drug Administration today approved Ampyra (dalfampridine) extended release tablets to improve walking in patients with multiple sclerosis (MS). In clinical trials, patients treated with Ampyra had faster walking speeds than those treated with an inactive pill (placebo). This is the first drug approved for this use.

MS is a chronic, often disabling, disease that affects the central nervous system—the brain, spinal cord, and optic nerves. There are about 400,000 people in the United States and 2.5 million people world-wide with MS.

The progress, severity, and specific symptoms of MS are unpredictable and vary from one person to another. Symptoms can be mild, such as numbness in the limbs, or severe, such as paralysis or loss of vision. About half of all people with MS experience cognitive impairments like difficulties in concentration, attention, memory, and judgment, although these symptoms are usually mild and are frequently overlooked. Depression also is common among MS patients.

“Trouble with walking is one of the most debilitating problems people with MS face,” said Russell Katz, M.D., director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research.

Ampyra, when given at doses greater than that recommended (10 milligrams twice a day), can cause seizures. The most common adverse reactions reported by patients taking Ampyra in clinical trials include urinary tract infection, insomnia, dizziness, headache, nausea, weakness, back pain, balance disorder, swelling in the nose or throat, constipation, diarrhea, indigestion, throat pain, and burning, tingling or itching of skin.

Ampyra should not be used in patients with moderate to severe kidney disease. In these patients, blood levels with the drug approach those associated with the occurrence of seizures.

Ampyra will be manufactured under licenses from Elan of Dublin, Ireland, and distributed by Acorda Therapeutics Inc. of Hawthorne, N.Y.

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“HICY” Drug Regimen Reverses MS Symptoms in Selected Patients

A short-term, very-high dose regimen of the immune-suppressing drug cyclophosphamide seems to slow progression of multiple sclerosis (MS) in most of a small group of patients studied and may even restore neurological function lost to the disease, Johns Hopkins researchers report. The findings in nine people, most of whom had failed all other treatments, suggest new ways to treat a disease that tends to progress relentlessly.

“We didn’t expect such a dramatic return of function,” says Douglas Kerr, M.D., Ph.D, associate professor of neurology at the Johns Hopkins University School of Medicine. “Although we’re very early in the game, we think this approach could be the linchpin of a significant advance for MS treatment.”

Researchers have used the so called HiCy treatments with some success at Johns Hopkins for a variety of other immune system disorders, including aplastic anemia, lupus and myasthenia gravis.

Cyclophosphamide kills immune-system cells but spares the bone marrow stem cells that make them. The usual method of delivering it in pulsed, small doses, however, can cause the drug to build up to toxic concentrations in patients’ bodies, causing a variety of side effects, including a greatly increased risk of infection.

Seeking an alternative way to use the drug, Kerr and his colleagues reasoned that HiCy might clear out the majority of a patient’s immune system in one fell swoop, then allow it to “reboot,” giving nerve cells a fresh start and an opportunity to repair themselves. In the current study, nine MS patients got a total single infusion of 200 milligrams per kilogram of cyclophosphamide intravenously over four days, a dose several times higher than that given in pulsed regimens but significantly lower than the total amount usually given patients over time.

Before treatment, Kerr says, the study participants were “the worst of the worst” among MS patients. Eight of the nine patients had failed conventional MS treatments, and several of them were wheelchair-bound.

Reporting in the June 9 Archives of Neurology, the Johns Hopkins team said the disease appeared to reverse course for seven of the nine patients over two years following treatments. Overall, the patients, men and women ranging in age from 20 to 47 at the beginning of the study, experienced a 40 percent reduction in scores of a standard test that measures disability. They also had an overall 87 percent improvement in scores on a composite test that measures physical and mental function.

MS, which affects approximately 400,000 people - predominantly women - in the United States, is believed to occur when the body’s immune system attacks the insulating sheath that coats nerve cells, causing it to degenerate. Consequently, electrical signals that the cells use to communicate with the rest of the body become progressively weaker, leading to symptoms that include numbness, tingling, cognitive problems and sometimes paralysis.

Researchers have identified four different subtypes of MS, and each is thought to be caused by a different autoimmune process. As a result, developing a treatment that effectively targets all types of MS has been challenging, says Kerr.

Kerr cautions that the “reboot” phenomenon didn’t work in all the patients. Two years after treatment, MRI images showed that the disease had reactivated in about half the study participants, suggesting that their renewed ability may not be permanent.

Kerr’s colleague Adam Kaplin, M.D., Ph.D., assistant professor of psychiatry and neurology at the Johns Hopkins School of Medicine, is leading efforts to improve HiCy therapy with a blood test in development that could predict which patients would benefit the most from HiCy treatment. Also, since immune cells that regrow after HiCy treatment may contain the same defect that leads to MS, Kaplin and his colleagues are working on a way to regrow only healthy immune cells.

Other Hopkins researchers who participated in this study include Chitra Krishnan, M.H.S., Robert A. Brodsky, M.D., Daniel B. Drachman, M.D., Richard J. Jones, M.D., Dzung L. Pham, Ph.D., Nancy D. Richert, M.D., Ph.D., Carlos A. Pardo, M.D., David M. Yousem, M.D., M.B.A., Edward Hammond, M.D., M.P.H., Megan Quigg, B.A., Carrilin Trecker, B.A., Justin C. McArthur, M.B.B.S., M.P.H., Avindra Nath, M.D., Benjamin M. Greenberg, M.D., M.H.S., and Peter A. Calabresi, M.D.