Amyotrophic lateral sclerosis (ALS)

Question 1

When does symptoms of amyotrophic lateral sclerosis typically start?

Answer 1

Around 50-60 years of age

Question 2

What are the symptoms of ALS?

Answer 2

Symptoms and disease progression varies individually.
Subtle cognitive abnormalities before onset (depression)
ALS is the progressive degeneration of motor neurons which leads to death - when motor neurons die, the brain ability to initiate and control muscle movement is lost.

Question 3

What is the outlook for a patient with ALS?

Answer 3

There is no cure, and only two FDA approved drugs.
Patients typically die 2-4 years after diagnosis.

Question 4

Is ALS genetic?

Answer 4

Around 10% of ALS cases are familial, and some genes have been shown to increase risk of ALS

Question 5

What is the mechanism of action behind ALS?

Answer 5

It is still unknown. We know that motor neurons die, but not why.

Question 6

What is the best characterized animal model for ALS?

Answer 6

The SOD1-G93A mouse model.
The mutation is related to oxidative stress.

Question 7

What is the only available treatment option for ALS in Finland?

Answer 7

Riluze -> it decreases glutamate toxicity.
New studies show it has no survival promoting activity.

Question 8

What are the possible future therapies for ALS?

Answer 8

Neurotrophic factor
Stem cell therapies
Antisense-therapies
Viral vector therapies

Question 9

Why is CDNF studied in relation to ALS?

Answer 9

CDNF has been shown to be protective and restorative in models of parkinsons.
It suppresses neuroinflammation.
It diffuses better than GDNF in brain tissue.