Developed ALS Therapy Shows Early Trial Promise
Researchers in China have reported promising early results for RAG-17, an experimental siRNA-based therapy for a genetic form of ALS. The small-scale trial showed that the drug was well-tolerated and successfully reduced key disease-related biomarkers.
Why it matters
ALS remains an incurable neurodegenerative disease, and this therapy represents a potential breakthrough in targeting the biological drivers of genetic variants.
Chinese researchers have reported encouraging early results for a domestically developed experimental therapy targeting a genetic form of amyotrophic lateral sclerosis, or ALS. In a six-patient trial published this month in Nature Medicine, the drug was generally well tolerated and reduced key disease-related biomarkers.
Called RAG-17, the therapy uses small interfering RNA, or siRNA, to suppress SOD1, a gene whose mutations can cause ALS. By reducing production of the harmful protein associated with the mutation, the drug is designed to target a biological driver of the disease.
ALS is a progressive neurodegenerative disease that destroys the nerve cells controlling voluntary movement, gradually impairing a patient’s ability to move, speak, swallow, and breathe. The disease gained wider public attention through campaigns such as the Ice Bucket Challenge, but it remains incurable.
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