A research team led by Professor Rong-Kung Tsai at Hualien Tzu Chi Hospital has developed a novel intravitreal injection therapy, GP-01, as a potential treatment for traumatic optic neuropathy (TON). The therapy has demonstrated neuroprotective effects in preclinical animal studies and has successfully completed Phase 1/2a clinical trials, showing significant improvements in both visual acuity and field of vision with strong safety and efficacy profiles.

Addressing the Limitations of Current Treatments

Traumatic optic neuropathy is a severe condition caused by head trauma leading to optic nerve damage, often resulting in sudden vision loss. Due to the limited regenerative capacity of the optic nerve, the therapeutic window for intervention is extremely narrow. Without timely treatment, irreversible blindness may occur. Conventional treatments primarily focus on supportive care, which is often ineffective in preventing progressive nerve degeneration and may carry substantial side effects.

Multi-Mechanism Approach to Neuroprotection

GP-01 is a long-acting protein-based drug administered via intravitreal injection. It exerts neuroprotective effects through multiple mechanisms:

• Preserving the Optic Nerve Blood-Brain Barrier: Strengthens barrier function to reduce inflammatory cell infiltration and vascular leakage, thereby minimizing optic nerve edema and pressure.
• Regulating Immune Response: Modulates microglial and macrophage activity to decrease pro-inflammatory cytokine release and enhance anti-inflammatory factors, mitigating neuroinflammation.
• Stimulating Optic Nerve Repair and Regeneration: Activates the PI3K/AKT signaling pathway to promote the survival and regeneration of damaged retinal ganglion cells (RGCs).
• Controlling Intraocular Pressure: Utilizes anterior chamber decompression technology to prevent post-injection intraocular pressure elevation.

Clinical Trial Success and Future Applications

Preclinical studies have demonstrated that GP-01 effectively protects retinal ganglion cells, enhances visual function, and reduces neuroinflammation. Clinical trial results further confirmed that a single intravitreal injection led to significant vision improvement in 73% of patients and measurable visual field enhancement in 64%, with a favorable safety profile.

Professor Tsai emphasized that beyond TON, GP-01 may have therapeutic potential for other optic neuropathies, including ischemic optic neuropathy and normal-tension glaucoma. Moving forward, the research team aims to conduct larger-scale clinical trials, optimize the drug formulation, and accelerate regulatory approvals, with the ultimate goal of bringing GP-01 to clinical practice and improving the quality of life for patients with vision impairment.

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