It’s finally here: the first participant has been treated in a much-anticipated gene therapy trial aiming to encourage older cells to adopt a younger state.
This clinical trial is exploring a new method that involves activating three specific genes believed to “partially reprogram” aged cells, helping them act as if they were younger. Some experts believe that this kind of partial reprogramming could potentially restore old organs. However, this trial is focused on using the three genes as a treatment for a type of glaucoma, which can lead to blindness.
The idea is that the proteins produced by these genes will promote the regeneration of neurons in the optic nerve, connecting the eye to the brain—something that typically doesn’t happen in adults, especially in those with glaucoma. On June 9, Life Biosciences from Boston announced that it had successfully treated its first participant.
The stakes here are quite significant. This trial will also assess the safety of the reprogramming method, which is an ongoing concern in the field. Animal research has indicated that partial reprogramming can be done safely, but there are worries that it might push some cells toward becoming cancerous.
“Reprogramming could offer great benefits if it’s safe for humans,” says Matt Kaeberlein, a co-founder of Optispan, a preventative medicine firm in Seattle. “The technology is still in its early stages, and there’s potential for serious side effects.”
Kaeberlein mentions that the eye is probably a good initial target for this technique, as the likelihood of life-threatening side effects is lower compared to other organs.
Restoring Youth
The aim of partial reprogramming is to gently revert aged adult cells back to a more youthful state, restoring characteristics of young cells without completely stripping them of their specialized identities. Life Biosciences is utilizing three of the four genes that, in lab settings, can be manipulated to convert adult cells to a stem-cell-like state.
Back in 2020, researchers from David Sinclair’s lab at Harvard Medical School and their colleagues found that activating these genes in mice with impaired optic nerves resulted in neuron regeneration and even reversed vision loss in older mice and those with glaucoma. Since then, Life Biosciences has been studying this method in rodents and monkeys, and according to Sharon Rosenzweig-Lipson, the company’s chief scientific officer, there have not been any serious adverse effects detected.
