Immune Response and Therapeutic Windows

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. If we can figure out when that switch happens, we can try to intervene before it does. This project is also testing drugs that might keep immune cells in their protective state longer, buying time for other treatments to work. The primary question is whether we can catch the immune switch early enough to change the clinical outcome for affected individuals.

Partners:

  • VIB-UAntwerp (Belgium)

What this project needs:

  • Funding and drugs to test.
  • We need immune-modulating compounds that are worth evaluating in our C9orf72 disease models, and we need funding to run those experiments. If you work in immunology or drug development and have candidates, this is a platform that can move them forward.
  • If you are a donor, this project offers a direct line from your contribution to drugs being tested in disease-relevant models.


Several of these projects require funding, and the project needs for funds fluctuate. To donate to these efforts, please give to an unrestricted fund that is split evenly between End the Legacy and research projects driven towards curing genetic ALS/FTD.