Zurich: Novel pharmaceutical treatment for scleroderma

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A novel pharmaceutical treatment for scleroderma, comprising the administration of miR-29, has been discovered by Steffen Gay, Oliver Distler and Britta Maurer of the Department of Rheumatology at Zurich University.

Scleroderma is a chronic autoimmune disease characterised by a fibrosis of the skin and internal organs. The high morbidity and mortality is the consequence of the failure of affected organs such as the lungs. To date there is no therapy available to slow or prevent the disease progression.

Therefore, there is a substantial need for a specific anti-fibrotic therapy. miR-29 is a small non-coding micro RNA that is involved in regulating gene expression. It was found to be strongly down-regulated in scleroderma fibroblasts as well as skin biopsies from scleroderma patients compared to healthy controls.

Furthermore, the enforced expression of mi-R29 in scleroderma skin fibroblasts resulted in down-regulation of collagen on mRNA and on protein level. These experiments provide robust evidence that the miR-29 family negatively regulates major pathogenic pathways in scleroderma.

The researchers say their work demonstrates miR-29 is a valuable candidate for the development of a treatment for scleroderma.

A patent application has been filed.