Microtubule inhibitors including colchicine for myotonic dystrophy

INDICATION: Myotonic dystrophy type 1 (DM1)

Colchicine and other microtubule inhibitors could treat DM1, which is caused by expansions of CTG repeats in the DMPK gene. A screen for compounds reducing RNA levels of CUG repeats in HeLa cells identified three microtubule inhibitors, colchicine, thiocolchicine and suprafenacine, among the top five hits. Colchicine reduced the number of expanded CUG repeat-containing foci in myoblasts derived from DM1 patients. In a mouse model of DM1, the compound decreased the abundance of expanded CUG repeat-containing mRNA and increased muscle levels of a myotonia protein biomarker. Next steps include evaluating whether colchicine can reverse DM1 phenotypes in animal models...