12/19/2023 0 Comments Als new findings![]() ![]() Early studies exploring the potential of a blood pressure medication as a treatment for ALS Vincent Picher-Martel, the recipient of a 2021 ALS Canada Clinical Research Fellowship. The potential for STMN2 to act as a biomarker for ALS is also being explored by Dr. QRL-201 is a genetic therapy targeted towards STMN2 that is expected to enter clinical trials later this year. An experimental treatment currently under development by QurAlis, called QRL-201, aims to do just that. These findings support the idea that a reduction in STMN2 resulting from TDP-43 dysfunction contributes to ALS and suggest that methods to preserve the levels of STMN2 within motor neurons may have a therapeutic benefit in people living with ALS. Further analyses revealed that the functional impairments likely resulted from changes at the neuromuscular junction, the place where motor neurons connect to muscle fibers, which is often considered an early feature of ALS pathology. Using genetic engineering techniques, researchers developed mice that made lower levels of the STMN2 or were unable to produce the protein entirely.Īfter observing the mice, they found that reduced STMN2 led to functional impairments like those seen in ALS. ![]() In this new study, researchers at Washington University sought to answer this question. The results from the previous studies demonstrated a clear relationship between TDP-43 and STMN2 but left researchers uncertain whether loss of STMN2 directly contributes to disease progression. Patient tissues analyzed by researchers also showed that STMN2 levels are lower than expected specifically in motor neurons. Two landmark studies in 2019 ( 1, 2) revealed that when the amount of functional TDP-43 is decreased within cells, the level of another protein, STMN2, is substantially decreased. Normally, TDP-43 is found in the nucleus of a cell (a central compartment where our DNA is located) however, in people living with ALS it is often found in the cytoplasm (the area outside of the nucleus) where it tends to form clumps, or aggregate, and is no longer able to function properly. New insight into cellular processes that may be affected at early stages of diseaseĪdvances in identifying a novel biomarker for ALSĪ new study provides additional confirmation that reduced levels of a protein called stathmin-2 (STMN2), caused by TDP-43 dysfunction, likely contributes to the progression of ALS.Ībnormalities in a protein called TDP-43 are present in approximately 97 per cent of all ALS cases. Harnessing the power of artificial intelligence (AI) to uncover new therapeutic targets for ALS Researchers identify another piece of the ALS puzzleĮarly studies exploring the potential of a blood pressure medication as a treatment for ALS To help provide clarity around terminology that may be unfamiliar to our readers, we have developed a research glossary, which contains a list of scientific and medical terms and definitions relevant to ALS. In the September 2022 Research Update, you’ll learn about the progress researchers have made in untangling the complex biology that contributes to ALS, harnessing the power of artificial intelligence to help identify new therapeutic targets, exploring already approved medications as potential treatments for the disease, uncovering new biomarkers, and more. ALS researchers from around the world continue to build upon existing work and make new discoveries in the hopes of realizing a future without ALS. ![]()
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