Drug to prevent C9orf72 FALS?

Does this mean that they’ve developed a drug that can prevent C9orf72-type ALS?

Extracted from a Mayo Clinic Press Release, August 14th, 2014:

JACKSONVILLE, Fla. — A team of researchers at Mayo Clinic and The Scripps Research Institute in Florida have developed a new therapeutic strategy to combat the most common genetic risk factor for the neurodegenerative disorders amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and frontotemporal dementia (FTD). In the Aug. 14 issue of Neuron, they also report discovery of a potential biomarker to track disease progression and the efficacy of therapies.

The scientists developed a small-molecule drug compound to prevent abnormal cellular processes caused by a mutation in the C9ORF72gene. The findings come on the heels of previous discoveries by Mayo investigators that the C9ORF72 mutation produces an unusual repetitive genetic sequence that causes the buildup of abnormal RNA in brain cells and spinal cord.

>potential biomarker to track disease progression and the efficacy of therapies.

foxp3?

Not ready for primetime Mike. I am not sure how long before that hits clinical trials. Going to ask about it next neurologist I talk to. But my MGH neuro did not bring it up. First up for c9 specific is antisense. Two companies are racing to be the first to trials almost certainly sometime in 2015. I was told both will likely have a clinical trial.
Down the road I think a lot of people have hope for small molecule in the future though. I firmly believe they will solve this for our children. If you have interest in who is doing what for c9 pm me.

Max No I believe this is something different and specific to c9 NIH talked about this when I first went there last year but as you know they are looking for biomarkers in both FALS and SALS. The difference with FALS is you can study people before they get symptoms. I know they were trying to figure out when the biomarker shows up, whether it was before symptoms or not. I am hoping my spinal fluid helps I had 3 spinal taps before symptoms.

Interesting dilemma I have. Shall I fund my kid's college education or instead fund research for the disease that they might possibly get?

I better not get too excited right now. Or drink too much beer.

Even I am not bankrupting myself to fund research but I do know where my charitable donations go!

>Shall I fund my kid's college education



greatest gift a parent can give!

I agree with Max, fund their education; they might be the ones to find the cure

BK

Hi Mike,

I so hope they're working on a medication to prevent the expression of FALS and I so hope this benefits you personally. Maybe all these buckets of water will bring us some much needed results! There is always hope.

I bet you've got your Google alerts set up already......


Peace,
Laurie