Turning off “Bad” Gene in ALS

By Heather Kohn, Ivanhoe Health Correspondent

ORLANDO, Fla. (Ivanhoe Newswire) -- There is a new approach to treating ALS, and it may be the first significantly effective therapy for this neurodegenerative disease.

Each year nearly 10,000 people in the United States are told they have ALS, a fatal disease made famous by the late baseball player Lou Gehrig. ALS attacks the nerve cells in the brain and spinal cord, eventually preventing a person from moving, speaking and breathing, which ultimately leads to death several years after diagnosis.

Researchers from the University of California, San Diego have developed a method to silence the bad gene throughout the nervous system in animals with ALS. Some ALS cases are inherited by a mutation in a specific gene, and this method protects the neurons from premature death. There are also “sporadic” cases of ALS, and researcher Don Cleveland, Ph.D., told Ivanhoe, “If we are successful in the initial therapy in the inherited form, then the therapy would be directly applicable overall to all cases of ALS.”

In their tests on rodents, researchers found they could “dramatically slow disease progression in those animals by introducing this gene silencing therapy into their nervous system.” Cleveland adds, “This is the only method so far that has really robustly slowed disease progression, and it is from that basis that we are intending to go to human therapy next year.” His goal is to add years onto patients’ lives, perhaps doubling the amount of time they have after diagnosis.

The therapy involves directly infusing into the spinal cord the gene silencing drug through a catheter. Patients must have a surgically implanted pump for the delivery of the drug. Cleveland says, “That sounds complex, but this is a routine surgical practice done every day in hospitals across the country, primarily as a method for delivering pain medicine for those people with chronic back pain.”

This type of gene silencing therapy could also be applied to other neurodegenerative disease, like Alzheimer’s, Parkinson’s and Huntington’s.

This article was reported by Ivanhoe.com, which offers Medical Alerts by e-mail every day of the week. To subscribe, click on: http://www.ivanhoe.com/newsalert/.

SOURCE: Ivanhoe Interview with Don Cleveland, Ph.D.

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