New Skin Wound-Healing Compound Shows Positive Results in Phase 2 Clinical Trial
By C. Rajan, contributing writer
Researchers at the Virginia Tech Carilion Research Institute have recently developed a wound-healing peptide ACT1, which heals venous leg ulcers twice as quickly as the current standard of care. The discovery came about serendipitously while the researchers were working on studying electrical conductivity in the heart.
The first Phase 2, multicenter, randomized clinical trial, was performed by the biotechnology company, FirstString Research. The clinical trial used participants with ulcers as large as three and a half centimeters around that had been open for about 17 months. The patient group using ACT1 showed significantly better results than the group not using it, with 79 percent of their ulcers closing after 12 weeks of ACT1 application and compression bandage treatment, while only 36 percent of the control group's ulcers closed after 12 weeks of using only the standard-of-care compression bandage treatment. The results of the clinical trial demonstrate the effectiveness of ACT1 as a treatment option, and the patients did not report any side effects associated with the compound.
"This outcome highlights the importance of basic biomedical research," said Dr. Robert Gourdie, Director of the Center for Heart and Regenerative Medicine Research at the Virginia Tech Carilion Research Institute, and lead study author. "It was completely unexpected that our work – funded by the National Institutes of Health on electrical conduction in the heart – should lead to something that helps people who suffer from skin wounds." Dr. Gourdie and his team initially developed ACT1 in his laboratory at the Medical University of South Carolina while studying how to improve electrical communication between heart muscle cells.
Venous leg ulcers are a serious problem in the elderly and often lead to painful and hard-to-heal open wounds. The condition is caused by excessive pressure in the leg's veins, which in turn causes the tissues to break down.
"One of the problems you have with chronic wounds, like venous leg ulcers, is that the wounds get stuck in an inflammatory phase and they won't close," said Dr. Gourdie. "After doing some preliminary studies, we saw that treatment with ACT1 encouraged wound closure much more quickly than normal."
ACT1 works by binding to the ZO1 protein, preventing it from connecting to another protein known as connexin 43. ACT1 is the first such connexin-targeting drug to successfully complete a clinical trial, Gourdie says.
FirstString Research, founded by one of Gourdie's previous post-doctoral researchers, holds the exclusive rights to multiple patents for the ACT1 peptide, the underlying technology, and its applications. FirstString Research planned and conducted this first Phase 2 clinical trial to test the efficacy and safety of the compound, and is now planning phase 3 trials for FDA approval of the ACT1 peptide for use in people with venous leg ulcers.
Results for this study were recently published in the Journal of Investigative Dermatology.