- A new clinical trial is testing whether reprogramming cells to be younger could stop age-related blindness.
- Life Biosciences is currently focused on cells in the eye because the experimental drug can be injected directly into the eye, limiting effects on the rest of the body.
- The trial is still in its early stages, but some doctors are cautiously hopeful this genetic approach could slow cellular aging in the liver and beyond.
WHAT IF YOU could not just delay aging, but actually reverse it? Some scientists are doing just that. A clinical trial is testing whether a genetic treatment can reprogram old cells to behave like younger ones.
Right now, the trial is focused on reversing cellular aging in the eyes. But if successful, it could open the door to other opportunities of slowing down aging in other parts of the body. Here’s what we know so far, plus where things go from here.
How are Scientists Making Cells Young Again?
LET’S GET TECHNICAL. Scientists from the biotech company Life Biosciences are investigating the safety and potential side effects of an experimental medication called ER-100. It’s designed to treat two eye conditions associated with aging: open-angle glaucoma (OAG) and non-arteritic anterior ischemic optic neuropathy (NAION).
While not exclusive to older adults, people after 40 are at a higher risk of developing glaucoma. People after 50 are also have a greater chance of an optic nerve injury like NAION. Both diseases can potentially lead to blindness.
A single ER-100 injection would deliver genetic instructions for making three proteins that may reverse epigenetic changes (factors like age and disease can leaves marks on cells and over time affect how genes function). If all goes well, ER-100 would stop cellular decline and potentially restore function in retinal ganglion cells. These are visual cells that absorb what you’re seeing in the outside world and transmitting that information to the brain.
The treatment could “reset” the way cells age without messing with the underlying DNA, explains Sharon Rosenzweig-Lipson, PhD, chief scientific officer of Life Biosciences. “You can think of this like buffing a scratch out of a record, where the music plays well again without destroying the record,” she explains.
Why Start With Eyes?
THE COMPANY FOCUSED on ocular diseases (to start, at least) because the treatment can be delivered directly into the eye through an injection without impacting the rest of the body.
“By starting with epigenetic restoration in the eye, we hope to determine whether restoring aged cells to a younger, healthier state can enable more effective cell function and become a viable therapeutic strategy for reversing age-related diseases not only in the eye but across other cell types, tissues, and organs,” explains Rosenzweig-Lipson.
The trial is in its beginning phases—the company just treated its first patient and has plans to enroll up to 17 more in this phase of the clinical trial—but Rosenzweig-Lipson has high hopes the drug could be used to treat “a vast breadth of age-related diseases” in the future.
Are Doctors For or Against This Unorthodox Approach?
SOME DOCTORS ARE hopeful but cautious.
On one hand, there are no treatments to restore vision loss with these age-related conditions. “The optic nerve is an extension of the brain, and we do not have treatments to repair damaged brain tissue,” points out Andrew R. Carey, MD, associate professor of clinical ophthalmology and neurology at the Wilmer Eye Institute at Johns Hopkins Medicine. “If this treatment works, it will be able to restore vision to blind eyes and improve the quality of life and function of visually disabled patients.”
Amanda Henderson, MD, an ophthalmologist at Johns Hopkins Medicine, is another who’s ”excited” to see the progress of the trial. “The preclinical studies are promising.”
That said, Dr. Henderson is wary of one particular side effect: development of tumors While the ER-100 treatment uses a special formulation to lower that risk, doctors won’t know for sure what’s happening until the human trial is complete.
Another concern is the precarious game of gene manipulation. “The unintended consequences can include having the genes turned on in tissues other than the optic nerve, having the genes be overactive, and not being able to turn the genes off at the desired time,” warns Dr. Carey. “In addition, the treatment could turn on or turn off alternate genes resulting in unforeseen complications.”
A Glimpse at a Future With Age-Reversal Treatments
AGAIN, ER-100 IS in early stage clinical trials, so there’s a lot of research left to do. Still, Rosenzweig-Lipson is hopeful the treatment can eventually be used to help with liver disease and other conditions.
“ER-100 represents a differentiated approach by investigating whether restoring more youthful patterns of gene expression can help existing cells function more like they did earlier in life,” she says. “If successful, this strategy could open the door to treating disease by targeting one of its underlying biological drivers.”
Korin Miller is a freelance writer specializing in general wellness, sexual health and relationships, and lifestyle trends, with work appearing in Men’s Health, Women’s Health, Self, Glamour, and more. She has a master’s degree from American University, lives by the beach, and hopes to own a teacup pig and taco truck one day.
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