2 Genes Identified as Potential Targets for Multiple Sclerosis Therapy

Bundles of Neurons
Mayo Clinic researchers have found that two genes in mice were associated with good central nervous system repair in multiple sclerosis. The results of this study could lead to the development of more effective therapies for multiple sclerosis patients and for predicting multiple sclerosis patients’ outcomes.

“Most multiple sclerosis genetic studies have looked at disease susceptibility, or why some people get multiple sclerosis and others do not,” says Allan Bieber, study author. “This study asked, among those who have multiple sclerosis, why do some do well with the disease while others do poorly, and what might be the genetic determinants of this difference in outcome.”

Image: Bundles of neurons, each neuron with a myelin sheath coating.

Multiple sclerosis is a disease of the central nervous system that includes the brain, spinal cord and nerves, and symptoms include loss of muscle coordination, strength, vision, balance and cognition.  Multiple sclerosis is called a demyelinating disease because it results from damage to myelin, the insulating covering of nerves. Multiple sclerosis occurs most commonly in those between the ages of 20 and 40, and is the most frequent neurological disorder in young adults in North America and Europe. Approximately 330,000 people in the United States have multiple sclerosis.

The researchers used two different strains of mice with a chronic, progressive multiple sclerosis like disease. One strain progressed to paralysis and death. The other underwent the initial damage induction phase of the disease and then spontaneously repaired the damage to the central nervous system and retained most neurologic function. Using the powerful genetic mapping techniques that are available for mice, the team mapped two strong genetic determinants of good disease outcome.

“It’s possible that the identification of these genes may provide the first important clue as to why some patients with multiple sclerosis do well, while others do not,” says Dr. Bieber. “The genetic data indicates that good central nervous system repair results from stimulation of one genetic pathway and inhibition of another genetic pathway. While we’re still in the early stages of this research, it could eventually lead to the development of useful therapies that stimulate or inhibit these genetic pathways in patients with multiple sclerosis.”

The research suggests that there may be a small number of strong genetic determinants for central nervous system repair following demyelinating disease, rather than a larger number of weak determinants.

“If that’s true, it may be possible to map the most important genetic determinants of central nervous system repair in patients with multiple sclerosis and define a reparative genotype that could predict patients’ outcomes,” says Moses Rodriguez, a Mayo Clinic. “Such a diagnostic tool would be a great benefit to patients with multiple sclerosis and is consistent with the concepts of ‘individualized medicine.’”
References:
1. Allan Bieber, et al. Mayo Clinic.

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