ALS, standing for amyotrophic lateral sclerosis, also known as Lou Gehrig's disease, is a progressive neurodegenerative disease affecting the nerve cells of the brain and the spinal cord. Current treatments for ALS work by slowing the progression of the disease however by only a few months. Now, research at the University of Alberta shows a promising drug that not only significantly slows the progression of the disease but can revolutionize treatment for ALS patients by extending and improving their quality of life.
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The drug is called telbivudine and is used as a treatment for patients with hepatitis making it particularly safe as a promising therapeutic for ALS. "It is already proven safe to use in patients, and it has very good potential for repurposing to use in a new clinical setting against ALS," said Ted Allison, co-author of the study and associate professor in the Department of Biological Sciences.
In the research study published in Neurobiology of Disease, telbivudine was selected using computer simulations of most likely drug candidates before being tested on animal models. Specifically, the works by targeting a protein called SOD1. The protein was found to misfold and function improperly in ALS producing toxic effects that characterizes much of the phenotypic symptoms of the disease.
"SOD1 is a protein that is known to misfold and misbehave in most cases of patients with ALS," says Allison. "We showed that telbivudine can greatly reduce the toxic properties of SOD1, including improving the health of the subject's motor neurons and improving movement."
However, because ALS is still not well understood by researchers and clinicians—promising therapeutics may take a while before being sent to clinical trials. But, this study provides one step closer.
“We don't yet know exactly what goes wrong first in the motor neurons or how the misbehaving SOD1 causes toxicity. Because there is still much to learn about the disease, the ALS research community focuses on both understanding ALS and on developing promising therapies,” explains lead author of the study—Michele DuVal.
Source: University of Alberta