A new discovery made by researchers at UNC School of Medicine and NC State could lead to treatment and prevention for Lou Gehrig’s Disease.
Lou Gehrig’s disease or ALS is a disease that gained more awareness in the past five years, which many say is thanks to the viral online Ice Bucket Challenge.
Senior author of new ALS research at UNC School of Medicine, Dr. Todd Cohen agrees.
“The ice bucket challenge tapped into something that I would have not predicted, so I think that social awareness about this disease has this amazing power to put it on everyone’s radar”, said Cohen.
According to Cohen, the protein TDP-43 is believed to be a factor that causes ALS and sporadic inclusion body myositis or sIBM, another muscle related weakness.
The protein TDP-43 clumps together in the cells of patients with ALS and sIBM, and many scientists believe this is the major reason why those cells die.
In ALS, this death of motor neurons causes a patient’s brain to be unable to control voluntary muscles throughout the body. In sIBM, the death of cells in muscles causes weakness and impaired strength.
“What we have found with this study is that we can demonstrate that there are proteins that we understand and that we are tinkering with and that we are starting to get a handle on how they do harm in the brain and in the spinal cord of these patients. And once we can understand that process then we can start to develop therapies that we think will be really effective in these patients,” said Cohen.
A discovery made in 2015 by Cohen and colleagues found that clumping of TDP-43 is caused by a specific chemical modification called acetylation.
Once this discovery was made, researchers were then able to successfully come up with and test an anti-clumping method on mice subjects, preventing sIBM in the mice.
Although the study, published in Nature Commons, only studied this effect in muscle cells for sIBM, they believe the same reversal of clumping in the brain and spinal cords of ALS patients could be achieved in the future.
“The key is going to be how quickly can we take this knowledge and develop it into an FDA approved drug that then is going to be able to be given to patients. And, of course, that process can take some time as well, but it is my firm belief that a lot of it starts in the lab,” said Cohen.
The next step for Cohen’s team is to try to identify compounds that can be used in oral drugs for human patients that have the same anti-clumping effect.
“Of course those studies would have to progress into…clinical trials with patients and of course those studies could take years, but I think that once you see positive effects I think things speed up quickly because it’s a devastating disease. And I think that there’s going to be a lot of interest in moving that along as quickly as possible,” said Cohen.
The UNC School of Medicine is the state’s largest medical school, is ranked among the top medical schools in the US and is among the most well funded for its research efforts.
I am excited about this research. Could you do me a big favor and add some some more information regarding myositis. I believe the study look at treatment for IBM and they hope they can apply it to ALS as well. Your headline and bulk of the article speaks to ALS. I happen to have polymyositis and know many people with IBM. We are a rare disease and any mention would really help to spread awareness. I really appreciate your understanding.
Regards,
Rose