• GNE myopathy, a type of distal myopathy, is a rare disease and a designated intractable disease in which muscles atrophy and degenerate starting from areas distant from the trunk, gradually depriving the body of freedom. Patients with this disease have mutations in the gene for an enzyme called GNE and cannot synthesize sialic acid, including aceneuramic acid. At the Department of Neuromuscular Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry, a model mouse was created, and preventive effects were obtained through oral administration of aceneuramic acid.
• In response to this, the field of neurology conducted the world's first physician-initiated Phase I clinical trial in 2010-2011, establishing safety. After physician-initiated Phase II/III trials, extension trials, and efficacy confirmation trials, Nobel Pharma obtained manufacturing and marketing approval for the product named Acenobel® in March 2024. In the future, it is expected that the know-how gained from this seed in creating registries and protocols will be utilized to advance the development of treatments targeting other sialic acid supplements, viral vectors, and oxidative stress.

• AIMS: The invention is to analyze metabolic condition, especially in oxygen consumption and energy production in the adipose tissues of human (Patent: JP 3848818).
• PROBLEM TO BE SOLVED: To provide an instrument and method for analyzing the metabolism condition of a living body which is constructed in such a manner that it can measure the metabolism condition of a living body correctly and easily, and a recording medium.
• SOLUTION: A metabolism condition analyzer is provided with an input means for inputting information about the body of a subject, a control means for processing this information and an output means for outputting results of the processing. The information consists of name, age, sex, race, height, weight, bioelectric resistance value and the date and time of measurement. A data file consisting the control means stores evaluation data of a metabolic condition which is previously determined by medical judgment based on a combination of an internal respiration index and oxygen consumption and energy production in adipose tissues. These are computed by calculating the value obtained by subtracting one from a body density calculated from the height, weight of the subject and the bioelectric resistance value and then multiplying the value. Welcome to your investment or co-operation.

• Primary aldosteronism is a leading cause of secondary hypertension accounting for approximately 10 % of hypertensive patients; 4 million patients potentially exist in Japan. We aim to optimize the method of ablating aldosterone producing adenoma using newly developed bipolar radiofrequency ablation system with 300-400kHz high-frequency current and electric power of 40W (Celon Prosurge Applicator), and establish a less invasive interventional radiological treatment of primary aldosteronism.

Collaboration with medical equipment manufacture and universities are expected to develop new devices enabling ablation of aldosterone producing adenoma. Flexible guide-wire-type ablation system and rapid aldosterone assay system should be co-developed to tread aldosterone producing microadenoma which is difficult to detect due to its small diameter of less than 5mm.

• Dr. Katsunori Nonogaki has developed the novel ultrasound irradiation device, which can improve the autonomic nervous system activity and peripheral circulation. In addition, the ultrasoud device can improve hypertension and hyperglycemia within 20 min in subjects with drug-resistant hypertension and diabetes. Our initial device was approved in Japan (226AIBZX00028000). This device will be avaliable for the treatment of 1) muscle pain, 2) the autonomic neural dysfunction and stress-related disorders, 3) hypertention, and 4) diabetes. Moreover, the device will be usefull for your healthy life and aging care.

Our aims are to export the device internationally. We seek the investment and international business partners.

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.

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.

• 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

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.