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๐Ÿ‡ฐ๐Ÿ‡ท South Korea /Health & Science

Cellular Rejuvenation Therapy Enters First Human Trial for Eye Disease

From Hankyoreh · () Korean

Translated from Korean, summarized and contextualized by DistantNews.

At a glance

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  • A biotechnology company, Life Biosciences, has begun the first human clinical trial for a 'cellular reprogramming' therapy aimed at treating age-related diseases.
  • The initial trial involves patients with glaucoma, a condition where aging eye cells are targeted to revert to a younger state, potentially restoring vision.
  • While promising, experts express caution about potential side effects and the need for further research, though the company aims for broader applications including organ regeneration and life extension.

Humanity's age-old dream of rejuvenation is now entering the realm of clinical application with the commencement of 'cellular reprogramming' therapy trials. This innovative approach aims to treat diseases caused by aging by turning back the cellular clock. The first human patient to receive this 'anti-aging' treatment is a glaucoma patient facing vision loss.

This method allows us to control the expression of the genes to ensure they do not express beyond the level needed for cell regeneration.

โ€” Sharon Rosenzweig-LipsonThe Chief Scientific Officer of Life Biosciences explained the safety mechanism of the gene therapy.

Life Biosciences, a Boston-based biotechnology firm, announced it has administered its gene therapy to the first patient in a clinical trial. The therapy is designed to revert damaged ocular cells in aging eyes to a younger state. Success in reversing ocular aging could not only restore vision but also pave the way for organ regeneration and life extension research.

The core of this trial lies in safely returning retinal ganglion cells to their 'youthful' state before they become damaged. Life Biosciences uses a harmless virus to deliver three specific genes, known as Yamanaka factors, into the patient's retinal ganglion cells. This method, based on the Nobel Prize-winning work of Shinya Yamanaka, induces partial cellular reprogramming, rejuvenating the cells' biological age without fully resetting them to a primitive stem cell state. Previous studies in mice and non-human primates demonstrated the safety and efficacy of this technique, leading to FDA approval for human trials.

This technology is still in its early stages, so there is a high possibility of serious side effects.

โ€” Matt KaeberleinA co-founder of Optispan expressed concerns about the potential risks of the new therapy.

The trial will enroll 18 patients with optic neuropathy, including those with open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy. Glaucoma, which damages the optic nerve cells connecting the retina to the brain, can lead to blindness, and damaged optic nerve cells are currently considered irreversible. A unique safety mechanism is built into the therapy: the reprogramming genes are activated only when the patient takes the antibiotic doxycycline. Discontinuing the medication immediately halts the cellular regeneration process, preventing uncontrolled gene expression and potential cancer development.

There is currently too much expectation placed on this technology. If the trial fails, it could put all of our futures in jeopardy.

โ€” Peter WilliamsDr. Peter Williams of the Australian Institute for Medical Research voiced concerns about the hype surrounding the trial.

Despite the potential, some experts urge caution. Matt Kaeberlein of Optispan noted that the technology is in its early stages and carries a significant risk of side effects, though the eye is considered a relatively safe target for initial trials due to its lower risk of life-threatening complications. Peter Williams of the Australian Institute for Medical Research expressed concern about excessive expectations, warning that a trial failure could jeopardize future research. Life Biosciences CSO, Sharon Rosenzweig-Lipson, stated that while the ultimate goal is whole-body rejuvenation, the current focus is on addressing age-related diseases one by one, with ongoing research in animal models for conditions like liver disease. Meanwhile, David Sinclair, a co-founder of Life Biosciences, is reportedly pursuing whole-body rejuvenation, planning trials with volunteers using his gene reprogramming technology in a bid to win the XPrize Healthspan competition.

We hope to apply this technology to whole-body rejuvenation someday, but for now, we are in the stage of solving age-related diseases one by one.

โ€” Sharon Rosenzweig-LipsonThe CSO of Life Biosciences discussed the company's long-term vision and current research focus.
DistantNews Editorial

Originally published by Hankyoreh in Korean. Translated, summarized, and contextualized by our editorial team with added local perspective. Read our editorial standards.