For years, scientists have wondered whether aging cells could be restored to a younger, healthier condition rather than just continuing to decline. A Boston-based biotechnology company has taken what is believed to be the first step toward getting an answer.
Life Biosciences, Inc. (Life Bio) announced that its first patient has been dosed with ER-100, an experimental treatment intended to reverse age-related vision loss. In this instance, the injection went into one eye of an individual with glaucoma. Over the next six months, scientists will monitor the treatment’s effectiveness while doctors will watch for any safety issues.
Life Bios says its ER-100 is designed to treat optic neuropathies, such as glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION), a common cause of sudden, irreversible vision loss in adults 50 and over.
This first phase of the company’s clinical trial is described as a “pivotal moment” for longevity science because it is believed to be the first human trial of epigenetic reprogramming, a technique designed to make aging cells behave more like younger cells.
While the technique is sometimes referred to as “reverse-aging” therapy, researchers are not trying to make people biologically younger but to restore the function of damaged cells in the optic nerve and potentially improve vision in patients with diseases such as glaucoma.
“This is an important moment for Life Bio and for the field of aging biology,” David Sinclair, co‑founder of Life Biosciences and Professor of Genetics at Harvard Medical School, said in a news release. “Our research has suggested that aging is driven in large part by the loss of epigenetic information, not irreversible damage. This clinical study represents the first opportunity to test whether restoring that information can ameliorate human disease.”
Clinics in Boston, New York City, Los Angeles, and Charleston are recruiting patients with glaucoma or NAION to participate in the clinical trial for ER-100. The company expects fewer than 20 people to participate in the initial trial. After the participants receive the single injection into the eye, Life Bio researchers will give them doxycycline, an antibiotic, every day. The antibiotic acts as an on/off switch that will activate the therapy. Should there be any safety concerns, the participants could stop taking the pill, and the cellular reprogramming will stop.
“To me, it represents a potential transformational moment, not only for the company, not only for the field of aging biology, but I think potentially, and I don’t say this lightly, for medicine,” Life Biosciences CEO Jerry McLaughlin told Business Insider. “We’re really looking at the ability to restore function, to reverse disease at a very fundamental level in the body.”
What is Cellular Reprogramming?
Cellular reprogramming is a complex process of reverting a mature, specialized cell to a completely different cell type. Scientists have been exploring this concept for nearly two decades.
In 2007, scientist and surgeon Shinya Yamanaka was the first to successfully reprogram mature adult human cells into immature cells capable of developing into any cell type in the body. The scientist discovered four specific proteins, now called the “Yamanaka Factors,” that can reprogram cells, essentially making old cells act like new again. This breakthrough earned him the 2012 Nobel Prize in Physiology or Medicine.
Some critics are concerned about the potential for cancer since two of the four factors Yamanaka identifies are oncogenes, which are mutated genes that have the potential to cause cancer. Cells driven by oncogenes can divide indefinitely. In some cellular reprogramming studies involving mice, some developed cancerous tumors.
Life Bio is using three of the four Yamanaka proteins in its therapy, except for the one most closely associated with cancer development.
Other Biotech Companies Launching Similar Research Projects
Life Bio isn’t the only biotech company focusing on cellular reprogramming. Many other companies are exploring the best way to safely and effectively test cellular reprogramming without reverting cells to their original state. And these companies are getting funding to support their efforts.
For instance, New Limit, another biotech company, received $435 million in funding from Eli Lilly for its work on epigenetic reprogramming. Silicon Valley startups Altos Labs and Retro Biosciences received funding from Sam Altman, co-founder and CEO of OpenAI, and Jeff Bezos, founder of Amazon and Blue Origin, a spaceflight services company.
And novel therapies aren’t just focused on humans. Rejuvenate Bio, a biotech company, and Merck Animal Health have formed a collaboration to develop gene therapy programs targeting chronic and age-related diseases in companion animals.
Longevity Experts Hopeful and Cautious
Longevity researchers hope to find new ways to make cells act more youthful and resilient while maintaining their original identity and roles in the body. Although Life Bio is focusing on eye diseases, scientists are also investigating liver cells and muscle cells. The goal is to improve how the cells in these tissues perform, which could help people maintain their strength and energy as they age.
The idea of reprogramming cells to change how doctors diagnose and treat diseases is incredibly promising. If cells could be transformed to function better during the aging process, longevity experts say the possibilities would go far beyond just improving eyesight or building stronger muscles.
“I think we’re in the early days of showing the technology works, and it absolutely makes sense to start in the eye, but I think it gets truly exciting when we start getting towards these ideas of systemic changes,” Daniel Oliver, the CEO and co-founder of the cellular reprogramming startup Rejuvenate Bio, told Business Insider. “These technologies get really, really exciting when you go into things like muscle, possibly liver, neuronal tissue, where we absolutely know that when you have positive effects there, it tends to have positive effects across the body.”
Brian Kennedy, who directs the Centre for Healthy Longevity at the National University Health System in Singapore, expressed some caution in a recent episode of a podcast hosted by longevity researcher Matt Kaeberlein.
“I am supportive of the concept of epigenetic reprogramming, partly because I think that it has, conceptually, the potential to have a big effect on aging if it can be done correctly,” Kennedy said on the podcast. “I’ve always been skeptical that we have enough knowledge to do that safely and effectively in a human. Having said that, this is an interesting place to look.” Since the current human trial involves the eye, which is semi-protected and sheltered from the rest of the body, Kennedy believes it will have fewer side effects.
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