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ID 117167
Title Alternative
To elucidate the pathophysiology and develop new treatments of amyotrophic lateral sclerosis
ALSの病態解明・治療法開発
Author
Keywords
amyotrophic lateral sclerosis
ALS
methylcobalamin
Content Type
Journal Article
Description
I graduated from Tokushima University in 1995 and trained at Hiroshima University and Sumitomo Hospital. In2000, Professor Ryuji Kaji was appointed as the first professor of the Department of Neurology, Tokushima University. In 2001, I graduated from Hiroshima University Graduate School and returned to Tokushima University. At the Department of Neurology, Tokushima University, we have been investigating natural history, neurophysiology including neuromuscular sonology, MRI, liquid biomarkers, genes, neuropathology, iPS cells, and new treatments as research on amyotrophic lateral sclerosis(ALS)(Tokushima ALS Research). We have examined the effect of methylcobalamin on ALS as a new therapeutic candidate. A phase Ⅲ study, Japanese Early-stage Trial of ultra-high dose methylcobalamin for ALS(JETALS), was carried out. Patients with ALS diagnosed within1year of onset by the updated Awaji criteria were initially enrolled. Of those, patients fulfilling the following criteria after12‐week observation were eligible for randomization :1- or 2-point decrease in ALS Functional Rating Scale Revised (ALSFRS-R)total score, a percent forced vital capacity over 60%, no history of noninvasive respiratory support and tracheostomy, and being ambulant. Methylcobalamin 50 mg or placebo was intramuscularly injected twice weekly for 16 weeks. The primary endpoint was change in ALSFRS-R total score from baseline to week 16. As a result, the least-squares mean difference in ALSFRS-R total score at week 16 of the randomized period was 1.97 points greater with methylcobalamin than placebo. The incidence of adverse events was similar between the two groups.
In the future, we plan to work on elucidating the pathophysiology of ALS, developing more powerful treatments for ALS, and dealing with non-motor symptoms of ALS.
Journal Title
Shikoku Acta Medica
ISSN
00373699
NCID
AN00102041
Publisher
徳島医学会
Volume
78
Issue
1-2
Start Page
45
End Page
50
Sort Key
45
Published Date
2022-04-25
FullText File
language
jpn
TextVersion
Publisher
departments
University Hospital