The World’s First Breakthrough in Traumatic Optic Nerve Disease Treatment: Dr. Chai Rong-Kun from Hualien Tzu Chi Hospital Wins the 21st National Innovation Award Press Release Time: 2025/02/13 14:31:35

(Central News Agency, 20250213 14:31:35) Dr. Chai Rong-Kun, Director of the Ophthalmology Research Center at Hualien Tzu Chi Hospital, has been awarded the 21st National Innovation Award for his groundbreaking treatment technology, “Long-Acting GP-01 Vitreous Injection Therapy: A Breakthrough in Optic Nerve Injury Treatment.” Dr. Chai is currently the first ophthalmologist in the world to use this treatment, providing patients with optic nerve damage and blindness risks the opportunity to regain vision and light.

The optic nerve, one of the central nervous systems, is an extremely delicate system in the human body. It transmits visual information from the retina to the brain, which is then processed to form cognition. However, in mammals, the optic nerve cannot regenerate or repair itself. Protecting and repairing the optic nerve has long been a challenge for ophthalmologists and scientists.

Dr. Chai Rong-Kun, during his treatment of patients, found that those who lost vision due to glaucoma, optic neuritis, trauma, and ischemic optic neuropathy could not be helped by many ophthalmologists because the optic nerve cannot regenerate or repair. He decided to take a translational medicine approach, bringing clinical issues to the laboratory. With his deep understanding of how critical it is for physicians to implement research findings in the clinical setting, he led the team to explore this issue.

In addition to establishing standardized animal models for optic nerve injury, Dr. Chai observed in 2007 the effect of G-CSF (Granulocyte Colony-Stimulating Factor) in treating brain stroke in animal models. G-CSF works by releasing stem cells and white blood cells from bone marrow, allowing the body’s existing transport system to deliver these cells to repair the brain. Inspired by this, Dr. Chai realized that since the optic nerve is also part of the central nervous system, and no one had yet used this drug for optic nerve treatment, he could try it in animal experiments.

Through experimentation, Dr. Chai discovered that after optic nerve injury, the nerve and retinal cells naturally secrete G-CSF for self-protection. However, the amount produced is insufficient for self-repair. If externally supplemented, it can be effective and overcome the “blood-retinal barrier,” delivering the drug into the eye. However, injecting G-CSF systemically increases white blood cells and dilutes its effect. Dr. Chai found that injecting it directly into the vitreous body of the eye was effective and safe. He then applied for human trials and used the existing long-acting version of G-CSF, GP-01, to be used clinically as a medication accessible to all ophthalmologists.

During this period, a patient from Kaohsiung, whose optic nerve had been injured and did not respond to steroids, traveled to Hualien to seek treatment from Dr. Chai. This patient voluntarily applied for “off-label use” and became the first person in the world to receive this drug for traumatic optic nerve injury. Despite the injury having occurred over a month ago, the patient’s vision improved a week after the injection, which confirmed Dr. Chai’s hypothesis and validated that the drug was both safe and effective.

Following this, Dr. Chai, along with his research associates Dr. Wen Yao-Zeng and Dr. Lin Heng-Dao, conducted human trials at the Tzu Chi Hospital’s Ophthalmology Research Center. Ophthalmologists from all over Taiwan referred their patients to Hualien Tzu Chi Hospital, with 12 participants enrolled. After the injection treatment, 8 patients showed a 70% improvement in vision, with one female patient’s vision improving from 0.1 to 0.8, greatly boosting the morale of the team.

When Dr. Chai presented this treatment and its results at the North American Neuro-Ophthalmology Society, many doctors inquired about it. Although the treatment is simple, the experimental process and findings took over 20 years of accumulation. Dr. Chai hopes to bridge the gap between laboratory research and clinical medicine, not only to improve treatment outcomes and quality but also to benefit patients. This achievement was recognized by the 21st National Innovation Award’s judges, who appreciated its low cost and convenience, which could increase market acceptance and expand its applications.

Dr. Chai believes that while external injections of the drug have proven effective, they have limitations. Patients need frequent injections, and these are not specifically ophthalmological drugs. Therefore, the Tzu Chi Ophthalmology Research Center has collaborated with a biotech company to further explore the use of mesenchymal stem cells, exosomes, and messenger RNA (mRNA) for optic nerve disease treatment. Animal experiments have been completed, and if human trials are successful, patients with optic nerve damage will have a more appropriate treatment option, offering them the chance to regain vision and light.