The FDA approved a combination stem cell-gene therapy clinical trial to test the treatment’s ability to slow the progression of amyotrophic lateral sclerosis. It’s the 1st clinical trial to use neural stem cells to deliver a particular protein in the hopes of slowing ALS progression, according to researchers at the ALS clinic at Cedars-Sinai Medical Center.
A progressive neurodegenerative disease, ALS affects nerve cells in the brain and spinal cord and patients eventually lose their ability to control muscle movement. The prognosis is dim – patients are totally paralyzed within 2 to 5 years of diagnosis and the disease is fatal. The ALS Assn. pointed out that there is no cure and only 1 FDA-approved drug that modestly extends survival.
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