Natalizumab Reported To Be a Safe and Effective Therapy for Pediatric Multiple Sclerosis

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A new study recently published in the journal BMC Neurology revealed that natalizumab (TYSABRI) is a safe and effective medication for pediatric cases of multiple sclerosis (MS). The study is entitled “Natalizumab in the pediatric MS population: results of the Italian registry” and was conducted by researchers at several hospitals and universities in Italy.

MS is a chronic, progressive neurodegenerative autoimmune disorder that results from the attack to the central nervous system (brain, spinal cord and optical nerves) by the body’s own immune system, causing inflammation and damage to the myelin layer that covers and protects neurons. Myelin loss leads to impairment in signal transmission along the nerve fibers, affecting motor function (coordination, balance, speech and vision), causing irreversible neurological disability and paralysis. It is estimated that more than 2.3 million people in the world suffer from the disease.

Natalizumab is a humanized monoclonal antibody used for MS treatment, and a promising option for pediatric patients who experience an active evolution of the disease and are poorly responsive to therapies with interferon-beta or glatiramer acetate. There is, however, a lack of data concerning the long-term effects of natalizumab in pediatric MS patients.

In the study, researchers investigated data from a registry established in Italy in 2007 with MS patients, aged less than 18 years old, under natalizumab treatment. In total, data from 101 MS patients was collected. The patient cohort had a mean age at MS onset of 12.9 years, a mean age at the initiation of natalizumab treatment of 14.7 years, and mean treatment duration of 34.2 months (almost three years).

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Researchers found that overall, natalizumab treatment led to a significant reduction in the annualized relapse rate and a decrease in disability as measured by the expanded disability status scale (EDSS), in comparison to the pre-treatment period. At the time of the last follow-up visit, 58% of the patients were found to be free from clinical disease activity (in terms of relapses and increased EDSS), and new MRI lesions. In terms of safety, no relevant adverse events were reported.

The team concluded that natalizumab is a safe, well-tolerated and effective therapy for the majority of the pediatric MS patients, and suggests that natalizumab should be given to this patient population, especially when they experience an aggressive disease course.

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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
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