Announced the results of joint research on the development of a treatment for mitochondrial disease using 5-aminolevulinic acid and sodium ferrous citrate

SBI Pharma Co., Ltd.
Saitama Medical University
Chiba Children’s Hospital
Juntendo University

SBI Pharma Co., Ltd. (Location: Minato-ku, Tokyo, President: Yoshitaka Kitao, hereinafter “SBI Pharma”), a subsidiary of SBI Holdings Co., Ltd., is working with Saitama Medical University, Chiba Children’s Hospital , and Juntendo University. The results of the joint research were published in the scientific journal ” Scientific Reports ” of Nature Publishing Group .

Mitochondrial disease is a disease in which the body’s production of ATP, which is energy, is reduced. This is due to a genetic abnormality that reduces the activity of what is called the mitochondrial respiratory chain complex. However, until now, there was no radical cure for mitochondrial disease, only symptomatic treatment.

5-Aminolevulinic acid (*, hereinafter “5-ALA”) is metabolized intracellularly and becomes heme by binding to iron. Heme then becomes part of the mitochondrial respiratory chain complex and contributes to ATP production. In experiments conducted on animals, it was reported that it activates mitochondrial function, and the combination of 5-ALA and iron was expected to be effective as a therapeutic agent for mitochondrial disease.

In this joint study, we examined the effects of a combination of 5-ALA and iron (sodium ferrous citrate, hereinafter “SFC”) using skin fibroblasts from patients diagnosed with mitochondrial disease. As a result, significant increases in mitochondrial respiratory chain complex and ATP production occur, that is, 5-ALA and SFC improve mitochondrial function in skin fibroblasts of mitochondrial disease patients for the first time in the world. I made it clear.

The results of this study suggest that in mitochondrial disease, “enzyme-enhancing therapy” that improves mitochondrial function by strengthening the action of the remaining mitochondrial respiratory chain enzymes, that is, its usefulness as a radical treatment. Currently, in Japan, a doctor-initiated clinical trial (Phase III) using 5-ALA + SFC as an investigational drug is underway, centering on Leigh encephalopathy, which is one of the mitochondrial diseases, led by this research group. It is highly expected as a new therapeutic drug.

(*) What is 5-aminolevulinic acid (5-ALA): An amino acid made by mitochondria in the body. It is an important substance that is a raw material for functional molecules involved in energy production called heme and cytochrome, but it is known that productivity decreases with aging. 5-ALA is contained in foods such as shochu lees, red wine, and ginseng, and is also known as a raw material for plant chloroplasts.

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