C9orf72 research data is scattered across hospitals, universities, and countries. The most valuable samples in the field come from carriers who were tracked before and after their disease began — blood, spinal fluid, and clinical records from when they were still healthy, and again after symptoms started.
Drug companies and researchers need cells from C9orf72 carriers to test potential treatments. Until now, getting those cells has been difficult, expensive, and inconsistent.
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There are drugs that have been tested in humans for other conditions and shown to be safe that could work for ALS, but that have stalled on the way there.
The goal of this project is to develop a new kind of blood test for ALS. Most diagnostic tests look for one molecule at a time. Like listening to an entire orchestra instead of one instrument, this platform uses nanosensor technology to read patterns across many molecules simultaneously.
Right now, doctors cannot reliably predict how fast ALS will progress in a given patient. This project is building algorithms that predict disease trajectory using clinical and genetic data.
The brain has its own immune system. In C9orf72 disease, immune cells called microglia appear to start out protecting neurons and then, at some point, we think they turn harmful.
Some C9orf72 carriers get ALS, others get FTD. No one knows why. This project uses one of the most powerful imaging facilities in the world