Tissue engineered skin model for ALS

Most of the attention around making ALS treatable is placed on development of therapies that can slow down disease progression. Equally important is the ability to diagnose people earlier so that such therapies can be applied at a time when motor neuron loss is not already so significant. Researchers have long sought after something called biomarkers, which are substances that can be detected through imaging or in body fluids (blood, cerebrospinal fluid, etc.) that can indicate if someone has ALS at a time point earlier than diagnosis by exclusion. Ideally, these tests will be available at symptom onset, and possibly someday, before physical manifestations of ALS ever occur.

For decades, ALS clinicians and researchers have known that the skin of people with the disease has unique characteristics, but until recently no one had created a way to study these effectively. In recent years, Dr. François Gros-Louis utilized expertise of collaborators at Université Laval to a develop tissue-engineered skin model derived from people living with ALS. PhD student Bastien Paré has received a 2016 Doctoral Research Award from ALS Canada to study these models for biomarkers that may be detectable at or before the onset of neurological symptoms. In preliminary work, Bastien has already discovered intriguing abnormalities in a region called the extracellular matrix, and perhaps even more promising, clumps of TDP-43, a well-known marker of ALS, in the skin. Furthermore, Bastien will examine the possibility of tissue-engineered skin to be a model for monitoring the effectiveness of drug treatments, giving the model even more value. Ultimately, the potential to use a simple skin sample for diagnosis and/or monitoring of clinical trials is a very attractive notion and would be a very important part of making ALS a treatable. It will certainly be exciting to observe the progress of Bastien’s work in the years ahead.