‘Cell Therapy’ Developed for Treating Damaged Spinal Cord

UNIST Professor Jeongbeom Kim's Team Develops Autologous Cell Therapy to Restore Motor Function
Potential Application for ALS and Other Diseases; Synergy Expected with Occupational Disease Specialized Public Hospital

Professor Kim Jungbeom and Researcher Lee Hyuna at UNIST.

[Asia Economy Yeongnam Reporting Headquarters Reporter Kim Yong-woo] Research on treating damaged ‘spinal cord’ using motor neurons derived from skin cells is attracting global attention.

The spinal cord, a nerve tissue inside the vertebrae that supports the body, is difficult to recover once damaged. According to this research, not only spinal cord injuries caused by accidents such as traffic accidents or industrial disasters but also diseases like Lou Gehrig’s disease, where motor neurons are destroyed, now have potential treatment options.

The research team led by Professor Kim Jung-beom of the Department of Life Sciences at Ulsan National Institute of Science and Technology (UNIST) succeeded in producing ‘motor neurons’ that make up the spinal cord by injecting two genetic factors into skin cells.

They confirmed the regenerative ability of the produced motor neurons in animal experiments, and it is known that the cells can be mass-produced for clinical application, indicating promising commercialization potential.

The spinal cord acts as a signal conduit, transmitting signals from the brain to the limbs and vice versa, conveying sensory information from the body to the brain. Therefore, damage to the spinal cord results in loss of motor function or sensation, causing serious aftereffects.

There are drug treatments and surgical methods to treat such spinal cord injuries, but their effects are limited and they have serious side effects. ‘Cell therapy’ using stem cells to regenerate damaged tissue is gaining attention.

In particular, motor neurons, which play an important role in controlling motor functions among the cells constituting the spinal cord, are strong candidates for cell therapy to treat spinal cord injuries.

However, obtaining motor neurons from embryonic stem cells or induced pluripotent stem cells posed a risk of cancer development, making direct application to patients difficult.

Schematic diagram of motor neuron production process and verification of therapeutic effects in spinal cord injury animal experiments.

To solve these problems, Professor Kim Jung-beom’s research team produced motor neurons using a direct transdifferentiation technique that obtains the desired target cells directly from skin cells.

By directly injecting two types of genes into patient skin cells, they created autologous motor neurons without passing through the ‘pluripotent cell stage,’ where cells could potentially transform into cancer cells. This approach resolved both immune rejection and cancer risk issues associated with conventional stem cell therapies.

Hyun-ah Lee, first author and integrated master’s and doctoral course researcher at UNIST’s Department of Life Sciences, explained, “We successfully created cells with motor neuron functions by sequentially injecting the gene ‘OCT4,’ which imparts stem cell properties, and the gene ‘LHX3,’ which imparts motor neuron characteristics, into patient skin cells.”

The developed motor neuron production method also has the advantage of mass production. A sufficient quantity of cells is required for patient clinical treatment, but existing direct differentiation methods were limited in the number of cells that could be obtained.

In contrast, the method developed by the research team passes through an intermediate cell stage capable of self-renewal, enabling mass production.

The team injected the produced cells into spinal cord injury model mice and confirmed recovery of lost motor functions and nerve regeneration within the damaged spinal cord tissue.

Professor Kim Jung-beom said, “We developed a direct transdifferentiation technology that overcomes the limitations of existing motor neuron production methods. Combining the produced motor neurons with ‘Spinegel,’ a therapeutic agent that protects spinal cord injuries and helps cells engraft well, could maximize therapeutic effects.”

He added, “Since spinal cord injuries have a high incidence rate due to industrial accidents, synergy effects with the specialized public hospital for industrial accidents planned to be established in Ulsan can also be expected.”

This research was conducted jointly with ‘Spine Therapeutics,’ a startup founded by Professor Kim Jung-beom, supported by the Ministry of SMEs and Startups. The research results were published online on June 23 in ‘eLife,’ a prestigious journal of the European Molecular Biology Organization.

This content was produced with the assistance of AI translation services.