Northwestern University researchers have developed a new way to deliver gene-based treatments for skin disease and even cancer in the form of a lotion that can be applied like a moisturizing cream.
The development, reported in the Proceedings of the National Academy of Sciences, is being hailed as a breakthrough in the engineering of new drugs to topically deliver gene therapy via commercial moisturizers for a range of skin conditions.
The Northwestern team – led by a physician-scientist and a chemist from the fields of dermatology and nanotechnology – said the technique offers great potential for life-saving new therapies.
“This allows us to treat a skin problem precisely where it is manifesting – on the skin,” said Dr. Amy S. Paller, a dermatology specialist with Northwestern and director of the university’s Skin Disease Research Center. “We can target our therapy to the drivers of disease, at a level so minute that it can distinguish mutant genes from normal genes. Risks are minimized, and side effects have not been seen to date.”
Paller and collaborator Chad Mirkin, director of Northwestern’s International Institute for Nanotechnology, said the technique allows drugs, applied directly to the skin, to penetrate all of the skin’s layers and selectively target disease-causing genes in cells – switching them “off” – while sparing normal genes.
Among the early targets of the novel treatment: melanoma and squamous cell skin cancer, psoriasis, diabetic wound healing and a rare genetic skin disorder that has no effective treatment known as epidermolytic ichthyosis. It could also be used to treat age-related wrinkles.
“The field of medicine needs new constructs and strategies for treating disease,” Mirkin said. “Many of the ways we treat disease are based on old methods and materials. Nanotechnology offers the ability to very rapidly create new structures with properties that are very different from conventional forms of matter. This collaborative study is a case in point.”
He said the technique can be applied to “a whole new set of diseases,” using genetic therapies and treatments involving nanotechnology, Mirkin said.
The researchers tested therapeutic ointments based on the technique on human skin, as well as the skin of laboratory mice. They found the drug penetrated the skin very deeply and selectively knocked down disease-related genes, effectively shutting them down.
After a month of applications, there was no evidence of side effects or inappropriate triggering of the immune system, they said.
Northwestern has one of nine Centers of Cancer Nanotechnology Excellence funded by the National Cancer Institute and one of six Skin Disease Research Centers funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
“This study is a landmark achievement in the area of gene regulation – I believe our work has a chance to positively and irreversibly change the field,” Mirkin said. “The skin is a very tough barrier to go through, which is why this effective gene knockdown has not been accomplished before. The power and elegance of this system are in its simplicity.”