This project to manufacture innovative drugs combating age-related diseases is based on pioneering work by V.P. Skulachev, member of the Russian Academy of Sciences and director of the A.N. Belozersky Research Institute of Physico-Chemical Biology. The project recently won approval from the Supervisory Council of RUSNANO. Dr. Skulachev's work is commonly known as the Skulachev ions (SkQ). The budget for the project will not exceed 1.83 billion rubles. RUSNANO will invest up to 710 million rubles.
The goal of the project is to bring to Russian and international markets drugs for treatment of ophthalmic disease and pharmaceuticals that have systemic effects. The project company plans to produce medicinal substances from which, on a contract basis, finished drug products will be prepared in Russia. The company will license its manufacturing technology to foreign companies in order to enter international markets.
Skulachev ions are innovative antioxidants whose molecules are about 1.5 nanometers. The ions neutralize active forms of oxygen at the cell's mitochondria, its energy-producing “power plants”. The uniqueness of the Skulachev ions is that positively charged parts of the molecule retain their hydrophobic properties, allowing ions to penetrate the membrane of the mitochondrion and concentrate themselves in its interior space. Thanks to targeted delivery to the mitochondria, only a very small amount of the powerful antioxidant is required to achieve the desired effect. The size of the molecule and its structure have been designed so that the antioxidant part positions itself inside the cell with the exactitude of only a few nanometers and rests close to the most susceptible spot in the mitochondrial membrane for the active form of oxygen. This explains why, for an antioxidant, the Skulachev ion has such high biological activity, and it is responsible for the high potential for its application as an innovative drug intervention.
Preclinical studies with laboratory animals have shown that the ophthalmological drugs based on mitochondrial antioxidants prevent the development of glaucoma and halt age-related dystrophy of the retina. The drug with systemic demonstrated its efficacy in prevention and treatment of such illnesses of aging as cardio-vascular diseases, osteoporosis, and rheumatoid arthritis.
The applicant-awardee for the project is Mitotech, an innovation company working in medical nanotechnology. Financing will be done in several stages, which will depend on outcomes of clinical testing that was begun in late 2009. The ophthalmological drugs are expected to enter the market in 2013 and the drugs with systemic effect—2016. Earnings from the project in 2016, are estimated at up to 1.20 billion rubles annually through production in Russia and 7.00 billion as one-time payment through licensing of technology to foreign pharmaceutical manufacturers.
Vladimir Skulachev, scientific head of the project, member of the Russian Academy of Sciences, and director of the A.N. Belozersky Research Institute of Physico-Chemical Biology explained, “These pharmaceutical drugs lessen age-related changes in tissues at all stages of aging. Preclinical studies demonstrated marked reduction in death rates among middle-aged animals and, because of this, a rise in average lifespan. The outer limits of lifespan rise only slightly; the animal, however, remains active, not showing typical signs of aging, almost to his death. This characteristic of mitochondrial antioxidants makes it possible to create drugs to treat a host of dangerous, age-related illnesses. The antioxidants help fight changes that come with age, not death at a very old age.”
“Regardless of its scientific courage, this project is interesting, first of all for its orientation toward applied science. If successful in clinical trials, the project will bring a new generation of drugs into the market; there is nothing like it anywhere in the world. The drugs act on an entirely new principle, one much more effective in treating numerous widely prevalent illnesses,” says RUSNANO Managing Director
Olga Shpichko. “In the near future, we shall be able to confirm that developments and scientific-technological potential in the Russian Federation in innovative pharmacology stand at world-class levels. The breakthrough scientific conception in the project is a technological platform for creating biologically active drugs with a broad spectrum of operation. They will lay the foundation for a domestic pharmaceutical future. The technological base proposed by the project already has many instances of use with confirmed efficacy such as veterinary drugs.”