Multiple sclerosis (MS) is a disease in which the immune systems attacks myelin surrounding axons in the central nervous system, leading to impaired neurotransmission and progressive axonal degeneration. Currently approved treatments for MS primarily target the immune response, but a new clinical trial is testing what could be the first therapy to reduce damage to the myelin sheath and protect the oligendrocytes that form it. The drug, called guanabenz, is FDA-approved to treat high blood pressure, but has been discontinued due to development of improved drugs. A recent study, published March 13 in Nature Communications (Way et al., 2015), demonstrates that guanabenz treatment prevents oligodendrocyte cell death, delays disease onset and reduces paralysis in mouse models of MS. The MRF is now supporting the Phase I clinical trial to test safety and tolerability of guanabenz in relapsing-remitting MS patients in combination with the FDA-approved anti-inflammatory drug for MS, Copaxone. Gaunabenz, as well as a newer derivative called Sephin 1, is also beneficial in mouse models of ALS (see April 2015 news story), so the outcome of these trials will also be of utmost interest to the ALS community.
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