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ALS

Listen: Could a daily pill one day treat ALS?

More breakthroughs in the basic biology of amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) are on the way and a cure is possible, says Robert Kalb, director of the Les Turner ALS Center at Northwestern University Medicine.

In this episode of the Breakthroughs podcast, Kalb explains how ALS diagnosis works and how patients go about getting treatment.

Listen to the episode here:

(Episode transcript.)

“ALS is a clinical diagnosis. There is no test, there’s no blood test. There are no imaging tests. There’s no electrophysiologic test that makes the diagnosis. It’s a constellation of signs and symptoms,” he says. “Once a person gets to our clinic, and we assemble all of the pieces of information, and include the positive and the negative results, we’re pretty confident that if a person has ALS, that we can give them that diagnosis.”

Kalb says he’s optimistic that a treatment can be found. “I’m quite sure, that at some point, there will be a pill, you take the pill in the morning, once a day, and the disease never progresses.”

Kalb is investigating the use of antisense oligonucleotides or ASOs in the treatment of ALS. This technology is already a treatment for children with spinal muscular atrophy.

“We are basically curing or having a huge impact on children and infants with spinal muscular atrophy. So, this is a template. This is a pathway I know works,” Kalb says. “Identify the mutant gene, devise therapies that use antisense oligos, give them to patients. The patients will get better. So with that pathway ahead of me, I think that it’s overwhelmingly likely that antisense oligos technologies will turn out to be useful for patients with ALS.”

ALS-linked protein clusters may actually protect neurons

Most people who have ALS have a sporadic form of disease, which means that there’s no clear genetic cause. In about 10 or 15 percent of cases there is a single gene, which is mutated, that causes the disease, and it can be passed along in families.

“We think that a major problem in ALS is the recognition of damaged proteins and disposal of them. And because this disposal process, or the recognition and the disposal process, is impaired, what ends up happening is an accumulation of damaged proteins and cells don’t like that. Cells are very unhappy when misfolded damaged proteins accumulate,” Kalb explains.

Source: Northwestern University

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