What was the key question behind this study, and why is it so important?

We're really trying to understand why it is that we've had so little success in developing therapies that really work for patients with ALS.

And one of our key concerns is that experimental therapeutics are being brought to bear too late in the course of disease.

And so this led us to ask the question: when does the biology of disease begin, and can we predict when symptoms are likely to arise? And if we can do those things, might we think about intervening therapeutically much earlier in the course of disease? And so in order to be able to answer that question, that series of questions, we had to study people at elevated risk for ALS.

And so we set upon the idea of studying people who carry a genetic risk that puts them at a high lifetime risk of developing ALS, and to study them longitudinally before symptoms ever develop.

And then once they've developed symptoms, we can go back and look at the data that we've collected, in this case biofluids and proteomic data that we've generated from those biofluids, and ask if there was something measurable that could have told us that disease was coming.

What makes these proteomic findings promising as biomarkers?

It's a good question.