UMass Chan trial first to demonstrate breakthrough ALS treatment in human
Photo | Courtesy | UMass Medical School
Dr. Robert Brown
A clinical trial for ALS research led by two UMass Chan Medical School doctors has shown significant progress in suppressing expression of the most commonly mutated ALS gene, according to an announcement from the Angel Fund for ALS Research in Wakefield, which funded the trial.
The research was initiated and led by Dr. Robert Brown and Dr. Jonathan Watts of UMass Chan in Worcester.
The study, which was conducted on one patient, uses newly developed antisense oligonucleotides (ASOs) to silence the C9ORF72 (C9) gene, which causes both familial ALS and frontotemporal dementia, per the release from Thursday.
While ASOs have been shown to suppress the C9 gene in previous studies, this is the first time it has been demonstrated in a human patient, according to the announcement. Similar trials are now being conducted by Biogen, Inc. in Cambridge.
Now, the team hopes to expand the testing to 10 patients in the coming months, pending further approval from the U.S. Food and Drug Administration.
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