Although multiple sclerosis (MS) is more likely to be diagnosed in adults between the ages of 20 and 40, children can also develop the autoimmune disease. According to a recent study, between 8,000 and 10,000 children under the age of 18 are diagnosed with MS each year, and many more could be living with the condition but have yet to be diagnosed.
Researchers say the first drug for children with MS vastly outperformed another common MS medication in a new clinical trial. Fingolimod (Gilenya) reduced relapse rates by 82 percent in patients aged 10 to 17 compared with interferon beta-1a, a drug commonly used to slow the progression of the degenerative nerve disease.
Nearly 86 percent of children on fingolimod remained relapse-free after two years of treatment, compared with only 39 percent of children taking interferon beta-1a, researchers reported.
"I do recommend doctors consider using fingolimod as first-line treatment in pediatric MS," said lead researcher Dr. Tanuja Chitnis, director of the Partners Pediatric MS Center at the Massachusetts General Hospital for Children.
Based on results from this clinical trial, the U.S. Food and Drug Administration in May approvedthe use of fingolimod in children, Chitnis said.
That makes fingolimod "the first drug approved in the U.S. for pediatric MS," Chitnis said.
Other drugs like interferon beta-1a are used in children, but their use is considered "off-label," said Bruce Bebo, executive vice president of research for the National MS Society.
"We consider this a major development, a major milestone in the MS treatment landscape," Bebo said of fingolimod's approval for use in children.
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Multiple sclerosis occurs when the immune system turns on the nervous system and attacks the protective sheath that covers nerve fibers, disrupting communication between the brain and the rest of the body.
MS causes vision problems, numbness or tingling, tremors, slurred speech and fatigue in patients. If left unchecked, it eventually will make it difficult for the person to walk.
Fingolimod is believed to treat MS by suppressing blood levels of lymphocytes, white blood cells that promote the immune system attack on nerve fibers, said Bebo, who was not involved with the trial.
"It slows down dramatically the pathways that lead to relapsing MS," Bebo explained.
The FDA approved fingolimod for use in adults in 2010, and the new trial is part of agency requirements to test new drugs in pediatric patients, Bebo said.
The peak age of MS onset is the 30s and 40s, but about 3 to 5 percent of patients develop symptoms of the disease in childhood, researchers said in background information.
For the clinical trial, 215 young patients were randomly assigned to take either fingolimod, which is an oral drug, or interferon beta-1a, which is injected.
Fingolimod kept MS from progressing in more than 8 out of 10 children taking the drug for two years, more than double the percentage of kids taking interferon beta-1a.
Fingolimod also slowed the development oflesions on the brain and spinal cord, a hallmark of MS. The rate of new or newly enlarged lesions discovered through MRI was 4.4 with fingolimod and 9.3 with interferon beta-1a.
Both drugs carried a high risk of adverse events, 89 percent with fingolimod and 95 percent with interferon beta-1a.
Serious adverse events occurred in about 17 percent of patients taking fingolimod, and included seizures, infection and low white blood cell counts.
"The ultimate decision is based on a clear benefit-risk discussion between the physician and family-patient. A careful review of potential side effects and monitoring is required," Chitnis said.
If your child is struggling with multiple sclerosis, visit our Health Conditions tab on BlackDoctor.org for more information.
SOURCES: Tanuja Chitnis, M.D., pediatric neurologist and director, Partners Pediatric MS Center, Massachusetts General Hospital for Children, Boston; Bruce Bebo, Ph.D., executive vice president, research, National MS Society; Sept. 12, 2018, New England Journal of Medicine
