By using a short, synthetic chain of chemically modified nucleotides, the new research has the potential to change treatment options for ALS. Researcher and lead author of the study Dr. Robert Brown said he and his colleagues have been able to suppress mutant forms of an ALS gene through their research.
“While other teams have documented that this gene can be suppressed in cells in culture, this is the first time this type of antisense oligonucleotide treatment for C9ORF72 ALS has been demonstrated in a person with ALS,” Brown said. “The results are very encouraging. It means this is a viable approach to suppressing the mutant C9ORF72 protein that causes most cases of familial ALS.”
The first ALS gene was found in 1993. Since then, researchers are now able to manipulate and reduce the toxicity of these genes.
“It’s been a long haul but I’m very excited,” Brown said. “I think these are inflection points in the work toward a real treatment of ALS.”
The next step of the research is to launch a multi-person clinical trial to see if this treatment can slow the progression of the disease.
“To do that, we’ve been required by the FDA to do some additional animal studies, which are now completed,” Brown explained. “So we do intend to expand the study as quick as possible.”
More information about the study can be found at the UMass Chan Medical School's website.
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