Announcement of the Commencement of a Specific Clinical Study for ALS
-Towards ALS Treatment Using Deciduous Tooth Pulp Stem Cell Culture Supernatant “U-Factor® Solution”-

U-Factor Co., Ltd. (Headquarters: Chiyoda-ku, Tokyo; CEO: Hidehiro Ijima, hereinafter referred to as “U-Factor”) and Hitonowa Medical (Headquarters: Chiyoda-ku, Tokyo; Director: Yasuhiro Seta) have jointly commenced a specific clinical study targeting ALS (Amyotrophic Lateral Sclerosis) patients in March 2024. This study will involve the administration of “U-Factor® Solution,” a deciduous tooth pulp stem cell culture supernatant developed by U-Factor, across multiple medical institutions to assess its safety and efficacy in treating ALS.

1. Background

ALS is a disease where the muscles in the limbs, throat, tongue, and those necessary for breathing gradually weaken and lose function. This condition primarily affects motor neurons, leading to the loss of communication between the brain and muscles. While sensory functions, vision, hearing, and internal organs remain unaffected, ALS progresses relentlessly, often leading to death within 3 to 5 years after onset. It is estimated that there are approximately 456,000 ALS patients worldwide, with about 10,000 in Japan (according to the Japan ALS Association). Every year, between 1,000 and 2,000 new cases are diagnosed in Japan. With no effective treatment available, ALS is designated as an intractable disease, representing a significant unmet medical need. The ALS Ice Bucket Challenge, a global phenomenon around 2014, brought considerable attention to this disease. Before launching this specific clinical study, a retrospective study conducted at Hitonowa Medical demonstrated the safety and efficacy of U-Factor® Solution, leading to the decision to proceed with this clinical study.

2. Overview of the Specific Clinical Study

This clinical study will evaluate the safety and efficacy of the investigational drug “U-Factor® Solution” in ALS patients.

3. What is Deciduous Tooth Pulp Stem Cell Culture Supernatant “U-Factor® Solution”?

U-Factor® Solution is a highly purified supernatant obtained through our proprietary technology during the cultivation of stem cells. It contains a large quantity of cytokines (proteins with physiological effects) secreted by the stem cells. Its efficacy was first discovered by U-Factor’s Director and Professor Emeritus Minoru Ueda of Nagoya University Graduate School of Medicine and colleagues (M Ueda, et al. Neurosci 2015). Through further refinement by our company, U-Factor® Solution has shown significant potential in terms of efficacy.

Comments from the Representatives

Hidehiro Ijima, CEO of U-Factor Co., Ltd.:

I am confident that U-Factor® Solution, which has been developed through extensive research, will meet unmet medical needs. As the first step, we are initiating a specific clinical study targeting ALS patients. By maximizing “quality” and ensuring “quantity” through the development of automated culture systems, we are committed to making U-Factor® Solution a beacon of hope for ALS patients through this clinical study.

Yasuhiro Seta, Director of Hitonowa Medical:

We are undertaking clinical research on ALS with the aim of sharing our findings with the world, providing new treatment options for patients suffering from intractable diseases, and contributing to the advancement of medicine. As a medical institution that walks alongside our patients, Hitonowa Medical is dedicated to furthering the field of regenerative medicine and making a global impact through both research and treatment.