A new therapeutic approach blocks ALS degeneration and saves motor function – in mice

Martin Marsala, MD

Researchers at the University of San Diego School of Medicine describe a new way to treat adult amyotrophic lateral sclerosis in mice. The result is the long-term suppression of the degenerative motor neuron disorder if the treatment is delivered prior to disease onset .

ALS is a neurodegenerative disease that affects nerve cells in the brain and spinal cord. More than 5,000 Americans are diagnosed with ALS each year and there are an estimated 30000 persons currently living with the disease. There is currently no cure and the majority of patients live between two to five years after diagnosis .

Of the two types of ALS, Sporadic is the most common form and accounts for 90 to 95% of all cases. It can affect anyone. Familial ALS accounts for 5 to 10% of all cases in the US and it is inherited.

This therapeutic approach provides the most potent therapy ever demonstrated in mouse models of the mutated gene linked to ALS. It is also likely to be effective in treatment of other hereditary forms of ALS  and possibly other spinal neurodegenerative disorders.

Researchers also tested the injection approach in adult pigs using an injection device developed for use in adult humans and found the procedure could be performed reliably and without surgical complications.

Learn more: Injection of Virus-Delivered Gene Silencer Blocks ALS Degeneration, Saves Motor Function