Scientists develop new compound for multiple sclerosis treatment

Scientists from the Florida campus of The Scripps Research Institute have developed the first of a new class of highly selective compounds that effectively suppresses the severity of multiple sclerosis in animal models. The new compound could provide new and potentially more effective therapeutic approaches to multiple sclerosis and other autoimmune diseases that affect patients worldwide.

As per the claim by the researchers, SR1001, a high-affinity synthetic ligand (first in a new class of compound, that is specific to both RORα and RORγt and which inhibits T_H17 cell differentiation and function),  binds specifically to the ligand-binding domains of RORα and RORγt, inducing a conformational change within the ligand-binding domain that encompasses the repositioning of helix 12 and leads to diminished affinity for co-activators and increased affinity for co-repressors, resulting in suppression of the receptors’ transcriptional activity. SR1001 inhibited the development of murine T_H17 cells, as demonstrated by inhibition of interleukin-17A gene expression and protein production. 

Furthermore, SR1001 inhibited the expression of cytokines when added to differentiated murine or human T_H17 cells. Finally, SR1001 effectively suppressed the clinical severity of autoimmune disease in mice. These data demonstrate the feasibility of targeting the orphan receptors RORα and RORγt to inhibit specifically T_H17 cell differentiation and function, and indicate that this novel class of compound has potential utility in the treatment of autoimmune diseases.....

Ref : http://www.nature.com/nature/journal/v472/n7344/full/nature10075.html