First human trial of cellular reprogramming approved in bid to treat blindness

A small Boston biotechnology company is preparing to launch the first human test of a controversial technique that aims to reverse aspects of biological ageing, marking a significant milestone for a field long confined to animal studies.

Life Biosciences has received clearance from the US Food and Drug Administration to begin a clinical trial using cellular reprogramming to treat glaucoma, a common cause of irreversible blindness.

The study will involve around a dozen patients and will test whether damaged optic nerve cells can be restored to a healthier state rather than replaced or bypassed.

The approach is rooted in work championed by the Harvard geneticist David Sinclair, who argues that ageing is driven by the loss of epigenetic information, the chemical markers that control gene activity.

Life’s treatment delivers three so-called Yamanaka factors into the eye using a viral vector, partially resetting cells without erasing their identity.

To manage safety risks, the genes are activated only while patients take a low dose of the antibiotic doxycycline, allowing doctors to halt the process if adverse effects emerge.

Reprogramming has drawn intense interest and large sums of capital, but it is also known to trigger tumours in animals if pushed too far.

The eye offers a relatively contained testing ground, limiting systemic exposure and making complications easier to detect.

Even supporters stress that the trial should be seen as a proof of concept rather than a step toward whole body rejuvenation.

If successful, however, it would provide the first human evidence that ageing-related damage can be reversed at the cellular level, potentially reshaping how degenerative diseases are treated.