therapy to make cells young again given to a person
Life Biosciences, a company based in Boston, Massachusetts, has treated the first participant in a clinical trial testing a gene therapy designed to rejuvenate aged cells. The trial aims to partially reprogram cells to restore them to a younger state, focusing on treating glaucoma, a condition that causes blindness by damaging the optic nerve.
How the Gene Therapy Works
The trial utilizes a novel approach that involves activating three specific genes within the body. According to the company, these genes function to “partially reprogram” aged cells, allowing them to regain the characteristics of younger cells without losing their specialized identity or function. By doing so, the therapy aims to stimulate the regeneration of neurons in the optic nerve, which connects the eye to the brain and does not typically regenerate in adults.
Did You Know? The research is built upon findings from 2020, where scientists in David Sinclair’s lab at Harvard Medical School demonstrated that activating these three genes could promote neuron regeneration and reverse vision loss in mice with damaged optic nerves.
Clinical Stakes and Safety Concerns
While the potential for cellular rejuvenation is significant, the trial faces scrutiny regarding the safety of genetic reprogramming. Matt Kaeberlein, co-founder of the longevity-focused company Optispan, notes that while reprogramming offers a potential upside, the technology remains in an early stage. There are concerns within the scientific community that such interventions could cause cells to shift into a cancerous state.
Expert Insight: The choice of the eye as a testing site reflects a strategic effort to manage risk. Because the eye is a localized, contained organ, researchers believe the probability of life-threatening, systemic side effects is lower compared to interventions involving other organs, making it a logical starting point for human trials.
What May Happen Next
Following the administration of the treatment to the first participant on June 9, the trial will continue to monitor for safety and efficacy. Life Biosciences has previously conducted studies on the approach in rodents and monkeys, reporting no serious adverse effects. Future outcomes depend on whether the therapy can safely replicate these results in human subjects. If the trial proves successful, the approach could potentially serve as a foundation for treating other diseases by rejuvenating aged tissues, though the field remains cautious about the high potential for catastrophic side effects.
Frequently Asked Questions
What is the primary goal of the trial?
The trial aims to test whether activating three specific genes can partially reprogram aged cells to behave like younger cells, potentially regenerating optic nerve neurons damaged by glaucoma.
Why is the trial being conducted on the eye?
The eye is considered a favorable site for this initial human trial because the risk of life-threatening side effects is lower than it would be for other organs.
Has this therapy been tested before?
Yes, researchers previously found that activating these genes promoted neuron regeneration in mice with glaucoma and damaged optic nerves; Life Biosciences has also tested the method in rodents and monkeys.
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