UW-Madison scientists have successfully slowed the progression of Lou Gehrig's disease in rats by using modified adult stem cells to deliver a growth hormone to atrophied muscles, according to the current issue of Molecular Therapy.
This result may provide the first hint of finding an effective therapy for treating Lou Gehrig's disease, also known as amyotrophic lateral sclerosis (ALS), which is currently untreatable. The scientists involved do not expect a cure yet.
The effect is not so big '¦ We have to improve,"" said Masatoshi Suzuki, the UW-Madison associate scientist who led the study. ""There are many steps we have to do.""
Scientists now know it's possible to use adult stem cells as a vehicle to release glial cell line-derived neurotrophic factor (GDNF), the growth hormone, directly into the damaged muscle.
Suzuki said he and his team altered the stem cells, taken from bone marrow, in order to produce this ferrying behavior. While the existence of GDNF and its positive effects have been known for some time, Suzuki's team is the first to combine it with the stem-cell delivery method to bring it right to the affected tissue.
The discovery represents a substantial step forward in ALS research and brings scientists closer to developing a cure. ""This paper's [result] is kind of surprising,"" Suzuki said, and it represents ""significant improvement.""
The last step in the ALS research is fine-tuning for maximum efficiency. Years of animal and human testing remain before any product can hit the market.
Grants from the ALS Association, the National Institutes of Health, the UW Foundation and the Les Turner ALS Foundation funded the research.
