Stem cells and neurodegenerative diseases
Stem cells are remarkable cells that are highly active during the initial growth and development of the human body, but are also found in small numbers in adults, where they support the repair of damaged tissues. Stem cells are unique in that they remain in an uncommitted state from which they can reproduce and give rise to many different cell types; they are the building blocks of our body. For instance, a single neural stem cell has the ability to divide and produce neurons of many different types, supporting glial cells, or more stem cells. The potential of stem cells to help our body resist or recover from disease and injury is an exciting and active area of research.
Stem cell technology offers a unique opportunity to develop a therapeutic approach to support and replace damaged nerve cells in patients with ALS and other degenerative neurological diseases. The passage of Proposal 2 in Michigan allows scientists for the first time to create the stem cell lines needed to develop these therapies, and PNR&D investigators have begun to dramatically increase research efforts in this area.
Preliminary research through the PNR&D shows that animals with ALS retain their ability to move and live much longer after they receive stem cell therapy. This has provided supporting evidence for the ongoing human ALS stem cell trial. Studies examining how stem cells provide protection in culture and in animal disease models are ongoing, and some of the questions these current PNR&D research efforts are trying to address include:
- What specific functions of the stem cells are providing benefit to damaged neural tissue?
- What is the best way to deliver cellular therapies to the nervous system?
- Can the protective ability of stem cells be enhanced in neurological diseases?
- Would this approach work in other neurological diseases, like Alzheimer’s or Parkinson’s disease?
PNR&D scientists are developing innovative approaches to answer these questions, model disease, comprehend pathogenic mechanisms, screen potential treatments, and develop cellular therapies. Details on the current experiments designed to answer these questions are described in the stem cell research projects section on the ALS page.