UA-developed afamelanotide shows promise in rare skin disorder

UA-developed afamelanotide shows promise in rare skin disorder

A synthetic hormone developed by Arizona Cancer Center and University of Arizona Department of Chemistry researchers two decades ago has been shown to offer relief for a rare skin disease in clinical studies in Australia and Europe.

Clinuvel Pharmaceuticals Ltd. of Melbourne, Australia, announced in July its drug Scenesse -- developed and patented at the UA under the name afamelanotide -- reduced and prevented painful reactions to sunlight experienced by people with erythropoietic protoporphyria (EPP) following a year-long Phase III trial. As a result of these positive clinical data, afamelanotide has been approved by the US Food and Drug Administration for a Phase II clinical trial in the US.

EPP is a rare genetic disease found mainly in people with fair skin. It is characterized by intolerance to light and results in pain, swelling, burning and scarring of sun-exposed areas of the skin. People with this condition are often forced to remain indoors during daylight hours. There are an estimated 10,000 people who suffer from EPP worldwide.

Afamelanotide, also known as Melanotan I, developed by UA Department of Chemistry and Biochemistry professor Victor Hruby, PhD, and the late Mac Hadley, PhD, a Biology and Anatomy professor, is a superpotent form of the naturally occurring alpha-Melanocyte Stimulating Hormone (MSH) which stimulates melanin production. Melanin is known for its photoprotective effect on the skin.

The Clinuvel drug was administered under the skin as a dissolvable implant about the size of a grain of rice. There were 91 participants in the double-blind study.

“The results of this long-awaited trial are positive, showing a major benefit in pain intensity and sun exposure,” said Robert Dorr, PhD, an Arizona Cancer Center researcher and professor of pharmacology in the UA’s College of Medicine, who was involved in the hormone’s early development.

Initial proof-of-principle clinical trials were conducted at the Arizona Cancer Center under the Chemoprevention of Skin Cancer program project grant led by David Alberts, MD, AZCC director. Initial study results were published in the Journal of the American Medical Association in 1991, Dorr said.

"Afamelanotide could prove a potent strategy for primary prevention of skin cancer through its longterm, natural tanning capability, even for redheads and blondes who always suffer sun burns when exposed to the sun," Dr. Alberts said.

- Aug. 5, 2010