New treatment is closer.
Amyotrophic lateral sclerosis (ALS) is a relatively rare neurodegenerative disease which represents a revelant economic and social burden. ALS has a late onset and is characterized by the loss of upper and lower neurons leading to muscle weakness and paralysis. From the point when symptoms start, most patients have about three years until the fatal end, which happens mostly due to respiratory failure.
There are potentially two forms of the disease: sporadic and familial. Even though patterns are mainly absent for distinguishing familial and sporadic ALS, family history is usually taken into consideration to set them apart. It is considered that 90% of the cases known today are sporadic and have no attributed cause, while the remaining 10% are supposedly hereditary and are said to have a genetic origin (these patients are usually carriers of one of 27 identified genes which are known as ALS causing).
These last years development of novel therapeutics for ALS has had a bump. It has now been almost 20 years since the last treatment was approved, mostly because of the difficulty of having an accurate model for testing such treatments on pre-clinical trials. However, recently a model was developed which is entirely derived from human elements and includes cells of the disease relevant tissue, capacitating the investigators to identify new targets and therefore successfully test new drugs.
Approving new drugs is closer in time too, as the investigators responsible for the model have already isolated a toxin which might be the cause of both types of ALS. The toxin is not only present in the model but also in patient removed cells, highlighting the importance of this model and this discovery. This finding is even more important for the sporadic type of ALS, from which there was less information and no defined cause. Even though investigators don’t know the function of the toxin yet, they already know how to control the upstream molecules (the molecules that control the toxin), which is already half way for the unraveling of the next approved drug.
Photo: Flickr, Oregon State University
als, Amyotrophic lateral sclerosis, brain, toxin, drug, neurodegenerative, disease