Researchers Identify Two Genes as Potential Therapeutic Targets for Multiple
ROCHESTER, Minn., Sept. 11 /PRNewswire-USNewswire/ -- A Mayo Clinic study has
found that two genes in mice were associated with good central nervous system
repair in multiple sclerosis (MS). These findings give researchers new hope
for developing more effective therapies for patients with MS and for
predicting MS patients' outcomes. This study will be presented at the Congress
of the European Committee for Treatment and Research in Multiple Sclerosis in
Dusseldorf, Germany, on Sept. 11, 2009.
"Most MS genetic studies have looked at disease susceptibility -- or why some
people get MS and others do not," says Allan Bieber, Ph.D., a Mayo Clinic
neuroscientist and author of this study. "This study asked, among those who
have MS, why do some do well with the disease while others do poorly, and what
might be the genetic determinants of this difference in outcome."
Mayo Clinic provides care for nearly 2,500 patients with MS each year. MS is a
disease of the central nervous system that includes the brain, spinal cord and
nerves. MS is called a demyelinating disease because it results from damage to
myelin, the insulating covering of nerves. It 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 MS. Symptoms include loss of muscle coordination,
strength, vision, balance and cognition.
Dr. Bieber and a team of Mayo Clinic researchers used two different strains of
mice with a chronic, progressive MS-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 MS 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 MS."
According to Dr. Bieber, 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
"If that's true, it may be possible to map the most important genetic
determinants of central nervous system repair in patients with MS and define a
reparative genotype that could predict patients' outcomes," says Moses
Rodriguez, M.D., a Mayo Clinic neurologist and director of Mayo Clinic's
Center for Multiple Sclerosis and Central Nervous System Demyelinating
Diseases Research and Therapeutics. "Such a diagnostic tool would be a great
benefit to patients with MS and is consistent with the concepts of