Specializes in clinical neurology, particularly neurodegenerative diseases and skeletal muscle diseases, and clinical genetics, and engaged in drug discovery and translational research for patients with intractable neuromuscular diseases.
- Overview of Technology
Amyotrophic lateral sclerosis (ALS) is a representative intractable neurodegenerative disease, and the development of effective therapies is desired. A novel phase I trial of intrathecal administration of recombinant HGF protein was conducted at Tohoku University Hospital. Based on the results, a phase II trial (investigator-initiated clinical trial) to confirm efficacy and safety for ALS was conducted and completed at the Tohoku and Osaka University Hospital from 2016 to 2022. Although there was no statistically significant difference in the primary endpoint, additional analyses are currently ongoing. Clinical development of the same formulation for acute spinal cord injury is also underway.
- Comparison with Conventional Technology
Simultaneous development of a novel intrathecal formulation of recombinant HGF protein, and a medical device (a subcutaneous port with an intrathecal catheter) that enable repeated intrathecal administration for efficient and selective delivery to the central nervous system.
- Features and Uniqueness
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- We developed a novel rat model of ALS model (Nagai M, et al. 2001)
- Intrathecal continuous administration of HGF protein was confirmed to have a slowing effect on progression in this model
- Phase I and II clinical trials have been completed, and the efficacy is currently being verified through additional analysis
- Practical Application
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We have been working with Kringle Pharma, Inc., Keio University (Prof. Hideyuki Okano and Prof. Masaya Nakamura), and Asahikawa Medical University (Prof. Hiroshi Funakoshi) to develop this drug. Phase I and II clinical trials for ALS have been completed. In addition, a phase III clinical trial for acute spinal cord injury is near completion. Additional collaboration with pharmaceutical companies is planned.
- Keywords
Researchers
Graduate School of Medicine
Masashi Aoki, Professor
Doctor of Medicine