The ALS Association, a nonprofit organization of global research and support for people with ALS and the Clinical Research in ALS and related disorders for Therapeutic development (CReATe) consortium, recently announced that two new amyotrophic lateral sclerosis (ALS) research projects have been selected for funding. The studies will advance the discovery and validation of new biomarkers vital for the development of ALS therapies.
ALS, or amyotrophic lateral sclerosis, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of motor neurons in ALS eventually leads to their demise. When motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, people start to lose their ability to speak, eat, move and breathe. For reasons that remain unknown, veterans are twice more prone to develop ALS than the general population. There is currently no cure for ALS and the only FDA approved drug merely extends patients survival.
The first study, which will be led by Dr. Adrian Isaacs, a Reader (Associate Professor) in the Department of Neurodegenerative Disease at University College London aims to develop a single molecule detection test for the dipeptide repeat proteins (DPRs) generated by ALS- and frontotemporal degeneration (FTD)-causing repeat expansion in the C9orf72 gene, known to be an underlying cause of ALS. During the study, the team will use a strategy able to detect a single molecule and that has a proven sensitivity of up to 1000-fold higher than the current enzyme-linked immunosorbent assay (ELISA) methods. This will hopefully allow researchers to detect DPRs in the blood of patients carrying the C9orf72 ALS/FTD genetic mutation. If this study is successful, only a blood test will be necessary to understand if a medication is working. It could also simplify the evaluation of new treatments for ALS or FTD that are caused by mutations in the C9orf72 gene.
The second study will be led by Dr. Benjamin Murdock, a Research Investigator in the Department of Neurology at the University of Michigan, and is based on prior knowledge that innate lymphoid cell populations are augmented in the blood of patients with ALS. The research team wants to better understand the role of innate lymphoid cells in ALS by tracking them for a year in ALS patients to observe if the cells express higher levels of pro-inflammatory signals, which may intensify the condition. If this study is successful and researchers are able to demonstrate the association between innate lymphoid cells and ALS progression, these cells could be tracked to predict disease progression.
“The ALS Association is extremely excited to partner with the CReATe Consortium in supporting these efforts to develop biomarkers that will be so essential to our collective efforts to identify effective therapies for this group of disorders” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., M.B.A.