Modified protein could lead to effective vitiligo therapy

A genetically modified protein demonstrated a reversal of vitiligo in lab mice and could lead to therapies for the condition in human patients.

Researchers with Loyola University Chicago Stritch School of Medicine genetically modified one of the amino acids in HSP70i, a protein that plays a crucial role in the autoimmune response that causes vitiligo, according to a university news release. The modification led to a mutant HSP70i, which displaced the normal protein and thus reversed vitiligo’s immune response.

When investigators gave mice with vitiligo the mutant HSP70i, the results were “striking,” according to the news release.

“Mouse fur, which is affected by vitiligo, had the coloring of a salt-and-pepper beard,” the university stated. “But when the mice were vaccinated with mutant HSP70i, the fur turned black,” leaving the mice looking like normal.

Some of the effects that were seen in the mice were also demonstrated in human skin specimens, which demonstrates the potential for therapeutic opportunities in human patients.

Researchers are seeking regulatory approval and funding for a clinical trial in humans. The university has submitted a patent application for the protein.

The study, which was supported by a grant from the National Institutes of Health’s National Institute of Arthritis and Musculoskeletal and Skin Diseases, was reported in Science Translational Medicine.