Gene therapy may benefit patients with scleroderma

Clinicians may one day be able to treat scleroderma more effectively, as researchers have uncovered how gene expression signatures can identify patients who respond well to particular therapies.

Investigators with Northwestern University’s Feinberg School of Medicine, Chicago, conducted a small pilot study to assess patients’ response to therapy with mycophenolate mofetil. Patients whose conditions did respond to the treatment were classified in the inflammatory gene expression subset, according to a news release. Patients who did not improve were classified in the normal-like or fibroproliferative gene expression subsets.

Researchers identified a mycophenolate mofetil gene expression signature that was composed of genes with expressions that changed “significantly” during treatment in the patients who showed improvement, but the signature was absent in the patients who did not respond to therapy.

A larger trial is under way, which may allow researchers to develop more effective treatment strategies for patients with systemic sclerosis.

The trial results were published in the Journal of Investigative Dermatology.