TOKYO: The health ministry Tuesday approved the production and sale of a new medicine for amyotrophic lateral sclerosis in Japan, raising hopes among patients.
In a doctor-led clinical trial, the drug, mecobalamin, extended the survival period of patients within a year of the onset of ALS by over 500 days, surpassing existing drugs.
It is the third treatment in Japan for ALS, a severe neurological disease that gradually causes full-body muscle paralysis, making late-stage patients unable to breathe by themselves and require respiratory support. It affects about 10,000 people in Japan.
While research using induced pluripotent stem, or iPS, cells is going on for potential ALS drugs, no fundamental treatment has been established.
Mecobalamin is a form of vitamin B12 sold as a remedy for peripheral neuropathy and other conditions. It was initially proposed for pharmaceutical approval as an ALS treatment in 2015 by drugmaker Eisai Co. after its clinical trial found a certain level of efficacy.
But the application was withdrawn due to the need for further tests, and a research team including members from Tokushima University conducted the doctor-led clinical trial.
The trial covered patients within one year of the onset of ALS at 25 facilities in Japan. It found that patients administered mecobalamin four months earlier than another group of patients lived longer by over 500 days. The effects were more significant than those of riluzole, an existing treatment that extends patients’ survival by an average of around 90 days.
“We are extremely surprised,” Ryuji Kaji, specially appointed professor at Tokushima University and a member of the research team, told a press conference last week. “A synergistic effect has been confirmed among those also given an existing treatment, so it may be possible to halt disease progression by using a mix of effective drugs.”
Eisai reapplied for government approval in January this year. The health ministry’s expert panel gave its green light late last month.
According to the research team, the extension of patients’ survival was confirmed in analyses conducted after the application to the health ministry.
“Patients have strong hopes over the emergence of a new drug to slow the (ALS) progression,” said Koichiro Miho, 57, an ALS patient who attended last week’s press conference. “I believe the disease will be curable.”
JIJI Press