Multiple sclerosis (MS) is a demyelinating disease of the central nervous system (CNS) resulting in damage to the myelin or insulating covers in the brain and spinal cord. This damage impairs the ability of different parts of the nervous system to communicate resulting in a host neurological symptoms or signs. Symptoms (Figure) include impaired vision, muscle weakness, trouble with sensation, trouble with coordination, and often psychiatric problems accompany the above signs. MS is usually progressive, with symptoms worsening over time. There is no cure for MS and current treatments are not effective in prevention of damage to the myelin. Therefore, the current treatments do not halt progression of MS and improved treatment is urgently needed.
While the cause of MS not well understood the disease is mediated by the innate immune response attacking the CNS producing toxic products such as reactive oxygen and nitric oxide species. The implication of nitric oxide species in MS has inspired us to create a novel class of drug candidates for MS designed to prevent damage caused by nitric oxide species. We have therefore synthesized and demonstrated efficacy of four such drug candidates in three different MS models. Our most thoroughly investigated candidate so far (MB11) prevents damage to myelin in mouse models of MS. Such protection is not observed with any currently approved treatment – no other treatment protects myelin in the mouse models of MS. Because this is still an early stage discovery project our current goal is to optimize the activity of this innovative and new drug candidate. Our long term goal is an oral drug taken daily to treat MS in humans.