ICYMI: Potential Treatment in the Works for Familial Amyotrophic Lateral Sclerosis

According to a story from telegram.com, a group of researchers from the University of Massachusetts medical school recently announced that they may have reached a major treatment breakthrough for genetically linked amyotrophic lateral sclerosis (ALS). The scientists have been testing a possible gene therapy approach that has shown promise in animal models so far. This therapy could be a useful treatment in about ten percent of disease cases. Most patients with amyotrophic lateral sclerosis do not have a genetic basis for their disease.

About Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease, is a rare, degenerative disease that causes the death of nerve cells associated with the voluntary muscles. Little is known about the origins of amyotrophic lateral sclerosis, with no definitive cause in about 95 percent of cases. The remaining five percent appear to inherit the disease from their parents. Symptoms initially include loss of coordination, muscle weakness and atrophy, muscle stiffness and cramping, and trouble speaking, breathing, or swallowing. These symptoms worsen steadily over time; most patients die because of respiratory complications. Treatment is mostly symptomatic and the medication riluzole can prolong life. Life expectancy after diagnosis ranges from two to four years, but some patients can survive for substantially longer. To learn more about amyotrophic lateral sclerosis, click here.

Gene Therapy and Amyotrophic Lateral Sclerosis

The success of the gene therapy so far means that it will be moving to clinical trials with human patients soon. Researchers first discovered that genetic mutations were responsible for amyotrophic lateral sclerosis in a small number of cases around 25 years ago. These mutations affect a protein called SOD1. This gene therapy involves the use of microRNAs in order to switch off the activity of this protein.

Christian Mueller, a member of the faculty that has been involved in the research, says that the development of the therapy has progressed relatively quickly over a period of seven years. Many gene therapies can take decades of research before reaching the clinical trial stage.

The therapy involves a single injection into the spinal cord, which should effectively halt disease progression. It is still unclear when clinical trials will begin. Mr. Mueller hopes that further research will allow the gene therapy to be useful in some non-familial cases of amyotrophic lateral sclerosis as well, which make up the vast majority of cases.


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