Blood metabolite links ALS progression rate to potential treatment target
Researchers in Japan have identified a blood metabolite, N-acyl taurines, that correlates with the progression rate of ALS. The study suggests that increasing these levels could potentially slow motor decline, offering a new target for drug discovery.
Why it matters
This discovery provides a novel biomarker and therapeutic pathway for treating ALS, a disease with limited current treatment options.
A metabolic signal found in the blood of people with amyotrophic lateral sclerosis (ALS) could help researchers track how quickly the disease is progressing and identify new treatment strategies, according to a study led by Nagoya University researchers in Japan.
The team found that blood levels of molecules called N-acyl taurines (NATs) were higher in people with rapidly progressing ALS and that patients with the highest levels had shorter survival times. In experiments using patient-derived cells and ALS mouse models, the researchers also found that increasing NAT levels with the compound PF-04457845 slowed motor decline and extended survival.
The findings point to a new route into ALS drug discovery by starting with metabolic changes observed directly in patients rather than relying primarily on laboratory models.
“We therefore began by analysing patient blood samples to map metabolic changes and identify treatments suggested by the results,” said Professor Masahisa Katsuno.
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