Interesting report of a biotech sponsored clinical study at Emory using stem cells to treat ALS victims. While this is obviously very exciting if you know someone who has ALS (also known as Lou Gehrig’s Disease), the study design is important. A quick analysis will help patients considering ALS stem cell therapy figure out the difference between a likely sham treatment and a cell therapy that may be effective.
The study will involve taking neural progentor cells (stem cells whose next step in the differentiation pathway is to become nerve cells) and will place them into the spinal cord (called intrathecal-meaning placing the cells in the fluid that circulates through the spinal cord and brain). Note two important things here: 1. the cells aren’t random, concentrated fat or adipose cells, but cells selected based on the fact that they like to become nerve cells 2. the cells aren’t injected IV (where 99.95% would get trapped in the lungs and only 1 in 500,000 would ever make it to the brain), but they’re placed directly in the space where they can act locally on the diseased brain and spinal cord tissue.
While this is all I could glean from the press release, this study is important for patients to grasp key concepts about stem cell therapy. First, type of stem cell matters for an effective orthopedic stem cell treatment, as I have bogged before. Second, where and how you place the stem cells can mean the difference between a sham treatment (for this condition, an IV therapy wouldn’t be credible) and a real attempt at therapy (in this case placing the cells where they can do some good). These same principles apply whether ALS is being treated or knee, shoulder, hand, or ankle arthritis. In the case of arthritis, placing cells blindly into a joint without advanced imaging guidance also won’t do, they need to be placed into very specific target areas in need of treatment.