Mayo Clinic researchers report ALS find

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Mayo Clinic researchers in Jacksonville, Fla., say they have developed a new way to fight "the most common genetic risk factor" for ALS and frontotemporal dementia.

Their findings in the scientific journal Neuron say they've found a possible biomarker to track disease progression.

In collaboration with The Scripps Research Institute in Florida, the scientists have "developed a small-molecule drug compound to prevent abnormal cellular processes caused by a mutation in the C9ORF72 gene."

Mayo researchers say they previously showed the gene "produces an unusual repetitive genetic sequence that causes the buildup of abnormal RNA in brain cells and spinal cord."

They believe the toxic RNA in genetic ALS is a good target for development of drugs to fight the condition.


The abnormal RNA also produces proteins detectable in the spinal fluid of people with ALS.

"They are now evaluating whether these proteins are also present in spinal fluid of patients with frontotemporal demential," says the Mayo announcement. Developing a biomarker to detect the medical conditions earlier is a primary goal.

That can allow diagnosis and tracking of disease progression, which will make it easier to see if experimental treatments are working.

"These landmark findings offer the possibility of both improved diagnosis and treatment for up to 40 percent of all patients with familial (inherited) ALS and up to 25 percent of patients with familial frontotemporal demential," according to Dr. Kevin Boylan, medical director of the Mayo Jacksonville ALS Center.

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