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A new medical experiment aiming to reverse aspects of ageing in humans could begin soon. The project is being led by David Sinclair, a Harvard professor who studies ageing and helped found a company called Life Biosciences. The company has received approval from the US Food and Drug Administration (FDA) to test a treatment, known as ER-100, in a small group of people with glaucoma, a disease that damages the optic nerve and can cause blindness.
The treatment is based on a scientific idea called “cellular reprogramming”. This involves using special genes to reset cells to a more youthful state by changing their epigenetic controls — the chemical switches that turn genes on and off. The discovery behind this method won a Nobel Prize nearly 20 years ago. Scientists found that adding certain genes, known as Yamanaka factors, can turn ordinary cells back into stem cells, which are like blank slates that can develop into many different types of cells. Because of this, the technique is sometimes compared to pressing a “factory reset” button on a cell.
However, fully reprogramming cells can be dangerous. In animals, it has sometimes caused tumours. To reduce this risk, researchers are testing a safer approach called “partial reprogramming”, where the genes are only switched on for a short time. In the trial, viruses carrying three reprogramming genes will be injected into one eye of each patient. The genes will only be active while patients take a low dose of the antibiotic doxycycline, giving doctors control over how long the treatment works.
Sinclair previously reported that partial reprogramming restored vision in mice, although some scientists question whether this truly counts as reversing ageing. The new trial, which will involve about a dozen patients, is mainly designed to test safety. If successful, it could mark the first time scientists have tried to rejuvenate human tissues in this way.
The field has attracted huge investment as well as attention from figures like Elon Musk, who recently said he believes ageing may be reversible. Even so, Sinclair has faced criticism in the past for promoting anti-ageing ideas that some scientists believe are overstated. The upcoming trial will be an important test of whether this powerful and controversial technology can safely help people.
