Scientists launch first cell rejuvenation trial
The therapy is being tested in a clinical trial led by Boston-based biotechnology company Life Biosciences. Researchers hope the approach could help restore damaged nerve cells in the eye and potentially offer a new treatment for glaucoma, a leading cause of blindness.
The experimental treatment uses gene therapy to activate three genes associated with so-called "partial reprogramming," a technique aimed at reversing some age-related changes in cells without erasing their specialized functions. Scientists believe this could allow older cells to regain characteristics of younger, healthier cells.
The trial focuses on retinal ganglion cells, whose nerve fibers form the optic nerve that carries visual information from the eye to the brain. Damage to these cells is a hallmark of glaucoma, and they normally have little ability to regenerate.
Interest in the technology grew after researchers led by Harvard geneticist David Sinclair reported in 2020 that activating the same three genes in mice promoted regeneration of damaged optic nerves and restored vision loss linked to aging and glaucoma.
According to Life Biosciences, studies in rodents and monkeys have not revealed serious safety concerns so far. However, experts caution that the technology remains at an early stage, and its long-term effects in humans are still unknown.
One of the main concerns is whether reprogramming cells could unintentionally trigger uncontrolled growth and increase the risk of cancer. For that reason, researchers say the eye is a suitable place for initial testing because any potential side effects are likely to be more contained than in other organs.
The company plans to enroll up to 12 people with glaucoma and may later expand the study to include patients with non-arteritic anterior ischemic optic neuropathy (NAION), a severe eye condition that damages the optic nerve and can lead to vision loss.
To deliver the treatment, scientists use a virus commonly employed in gene therapy to carry the three genes into retinal cells. The system includes an additional safety mechanism: the genes are activated only when patients take the antibiotic doxycycline. If the medication is discontinued, the genes switch off.
Researchers say the current goal is not whole-body rejuvenation but the treatment of specific age-related diseases. Even if the therapy proves effective, scientists note that it remains unclear whether successful regeneration of eye cells would demonstrate a broader ability to slow or reverse aging throughout the body.
Earlier, Qazinform News Agency reported that scientists discovered clues to human eye regeneration in apple snails.
