ALS may be the cruelest, most severe neurological disease,” says Clive Svendsen, director of the Regenerative Medicine Institute at the Cedars-Sinai Medical Center in Los Angeles. Fortunately, Svendsen and his colleagues appear to have found a potential treatment for ALS, using stem cells from patients’ own bodies.
“In these studies, we turned skin cells of patients who have ALS into motor neurons that retained the genetic defects of the disease,” says Robert H. Baloh, lead researcher and lead author of the article, published in the October 23, 2013 issue of Science Translational Medicine. “We focused on a gene, C9ORF72, that two years ago was found to be the most common cause of familial ALS and frontotemporal lobar degeneration, and even causes some cases of Alzheimer’s and Parkinson’s disease. What we needed to know, however, was how the defect triggered the disease so we could find a way to treat it.”
“In frontotemporal dementia, portions of these lobes atrophy or shrink. Signs and symptoms vary, depending upon the portion of the brain affected. Some people with frontotemporal dementia undergo dramatic changes in their personality and become socially inappropriate, impulsive or emotionally indifferent, while others lose the ability to use and understand language.”
Researchers led by Baloh found a genetic defect that may cause ALS and were then able to ‘knock it out’ with genetic material, with no evidence of detrimental effects. This laboratory research, all conducted in a lab dish, paves the way for clinical trials.
Svendsen concludes: “I believe the stem cell approach used in this collaborative effort holds the key to unlocking the mysteries of this and other devastating disorders. Within the Regenerative Medicine Institute, we are exploring several other stem cell-based strategies in search of treatments and cures.”