The clinical trial, which was approved by the U.S. Food and Drug Administration (FDA) earlier this year, could spark nothing less than a medical revolution. The Life Biosciences company has been granted permission to test a gene therapy that could halt the aging process in certain cells on human subjects. The drug ER-100 is designed to rejuvenate aged cells in the optic nerve. The goal is to reverse the previously irreversible loss of vision caused by glaucoma or by what is known as an “ocular infarction” (non-arteritic anterior ischemic optic neuropathy), thereby making it curable. What both diseases have in common is that they are caused by age-related cell damage.
One of the co-founders of Life Biosciences is Harvard professor David Sinclair. A biochemist and molecular geneticist, he is considered one of the most renowned researchers in the field of aging processes. For years, he has been drawing attention with bold statements about the future of gerontology—most recently at the World Governments Summit (WGS) in Dubai. There, Sinclair not only announced the clinical trial for ER-100, but also predicted that today’s therapies will seem outdated in ten to twenty years. Instead of curing diseases in the traditional way, medicine will focus on preventing or even reversing aging.
Blind monkeys regain their sight
The gene therapy technology used by Sinclair and his team is known as “partial epigenetic reprogramming.” It is based on the Japanese stem cell biologist Shinya Yamanaka’s discovery in 2006 that four specific proteins—the Yamanaka factors—can revert adult cells to a stem cell-like state. Yamanaka was awarded the Nobel Prize for this work in 2012. This discovery paved the way for numerous stem cell-based therapeutic approaches. Researchers such as David Sinclair have taken a step further and are pursuing this idea: If these factors are activated only briefly and then deactivated again, cells might be rejuvenated without completely reverting to a stem cell state. This approach has proved extremely successful in animal experiments—it was able to restore vision in blind primates.
The initial results of the clinical trial in humans are expected to be available in just a few months. If the results are positive, this could mark the beginning of a new era: an age of preventive medicine in which diseases are prevented before they even develop. Following the treatment of the optic nerve, Life Biosciences has already planned its next step. This will focus on the liver, followed by other organs—until, eventually, it may be possible to rejuvenate the entire human body.
This is a good reason for Evonik Foresight to take a closer look at this topic as part of the “GameChanger 2035” study. Evonik is already well-positioned for the future of medicine. The company has extensive experience with lipid nanoparticles and nanoparticle processing—key technologies that enable the active ingredients in modern gene therapies to be delivered to their site of action in a targeted, reliable manner and at the correct dosage. In addition, Evonik is already active in the research field of epigenetics. We need to continue building on this lead.
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