The Les Turner ALS Research Laboratory led by Teepu Siddique, MD, is focused on understanding the processes by which ALS develops so that effective treatments can be developed. The lab was first dedicated in 1979 and has been directed by Dr. Siddique since 1991.
Dr. Siddique, the Les Turner ALS Foundation/Herbert C. Wenske Foundation Professor, is a physician-investigator with expertise in the neurology, molecular genetics, biochemistry and cell biology of neurodegenerative disease. He is the director of the Division of Neuromuscular Medicine at Northwestern, where he leads the Les Turner ALS Research Laboratory and is co-director of the Les Turner/Lois Insolia ALS Center. His research in neurogenetics has been generously funded by the NIH since 1985.
Dr. Siddique and his colleagues in the laboratory and clinic carry out a wide range of molecular genetic experiments to understand the causes of ALS, identify biomarkers to diagnose and track ALS progression, and conduct drug trials to treat ALS. Dr Siddique initiated the molecular genetic approach to ALS in 1984 which lead to the successful identification of many genetic causes for ALS including SOD1, ALSIN, PON, FUS, TDP-43, SQSTM1 and UBQLN2. Recently he and his colleagues identified a common pathology for all of ALS involving ubiquilin2 and SQSTM1. His group has engineered several models for ALS including the first genetic model for neurodegeneration. His current research includes discovery of novel causes for ALS, the gene-environmental interaction in ALS and a common mechanism of disease amenable to therapeutic intervention. A mechanism involving the ubiquitin-proteasome pathway and autophagosome machinery, involved in recycling of cellular contents, was discovered by his team in 2011 and found to be modulated by ubiquilin2/SQSTM1 class of proteins . This molecular pathway appears to underlie all types of ALS irrespective of cause, and provides an attractive target for treatment. Dr Siddique's group has made an animal model for this pathway and are developing cell-based assays to screen drugs to treat ALS.
The Les Turner Laboratory maintains a Neurologic Diseases Registry that contains over 16,000 blood samples and 120 autopsied ALS cases.
The underlying disease process of ALS (also known as Lou Gehrig's disease), a fatal neurodegenerative disease that paralyzes its victims, has long eluded scientists and prevented development of effective therapies. However, a new study from Foundation researcher Dr. Teepu Siddique and his team has identified a common cause of all forms of ALS for the first time.
Please visit our full coverage page for further explanation of this discovery and its impact for patients and families worldwide, a short video, and a selection of comments from experts, media, patients and families.