The protein, glial cell line derived neurotrophic factor (GDNF), supports glial cells, which help keep alive the motor neurons that control muscle movement. In ALS patients, these motor neurons die, causing progressive paralysis and ultimately death. There is growing evidence that GDNF is linked with protection of motor neurons and amelioration of ALS.
More than 12,000 people in the U.S. have ALS, commonly known as Lou Gehrig's disease, according to the National Institutes of Health (NIH). Although drugs and mechanical devices can help alleviate symptoms, there is no effective treatment, and most patients die within five years.
In the study, published June 5 in Stem Cell Reports, investigators harnessed a common antibiotic, doxycycline, to both stop and start production of GDNF by neural progenitor cells that had been genetically reprogrammed to produce GDNF. This ability, demonstrated in animal models, improves the potential usefulness of the combined gene and stem cell strategy in experimental treatments for a range of disorders, they said.