The Les Turner ALS Research Laboratory at Northwestern University Feinberg School of Medicine opened in December 1979 with a staff of four. Studies at that time were focused on the possible viral and immunological causes of ALS. In 1992, the laboratory was rededicated in the Tarry Research Building, where both research space and staff quadrupled in size. The laboratory, led by Dr. Teepu Siddique, is now a world-renowned program with nearly 20 international medical researchers focused on determining the genetic causes of familial ALS and the genetic predisposition involved in sporadic ALS.

Research Updates

April 2008: What is Missing in ALS?  
February 2008: CYP7B1 Responsive Gene
2006: PON Clusters Associated with ALS  
July 2006: Variations in Detoxifying Genes Linked to ALS 
April 2006 : Discoveries Should Aid Research into Cause of ALS 

Important Discoveries
Teepu Siddique, M.D., director of the Neuromuscular Disorders Program is the Les Turner ALS Foundation/Herbert C. Wenske Foundation Professor in the Ken and Ruth Davee Department of Neurology and Clinical Neurological Sciences at Northwestern University. Dr. Siddique and his team are known throughout the worldwide ALS community for the discoveries of three genetic causes of ALS, as well as for encouraging use of the transgenic mouse model developed in 1994 by Northwestern scientists.

In 1993, Dr. Siddique co-discovered the very first known cause of ALS: mutations in the SOD1 (superoxide dismutase) gene on chromosome 21. In 2001, his lab learned that mutations in the ALSIN gene on chromosome 2 are responsible for juvenile onset ALS. Identifying these genetic causes and then producing mice with ALS-causing gene mutations permit scientists to observe the actual mechanisms and course of the disease on a molecular level. Recent advancements in technology and the Human Genome Project have made the identification of genetic causes a particularly promising field of research. In 2008, the Laboratory verified findings of the X-ALS gene, which appears to be involved in both ALS and ALS/dementia. This gene discovery allows investigation of how proteins are regulated in the nervous system, and can could also be useful in the study other neurological diseases, such as Parkinson's Disease, dementia and other degenerative conditions.

Get Involved
The Neurologic Diseases Registry at the Les Turner ALS Research Laboratory uses blood samples and information from patients and family members for their studies. For more information, please call (312) 503-2712 or
(312) 503-0154. Research is also conducted using post-mortem brain and spinal cord tissue. To read more on how to become involved with tissue donation, please click  here or call (312) 503-0160.

Current Investigations
The team of scientists at the Les Turner ALS Laboratory is focused on finding the causes of ALS and understanding the mechanisms involved in the onset and progression of symptoms. Their belief is that a basic understanding of how ALS operates on a molecular level will lead to more effective treatments and a cure. Investigative priorities include:

• Environmental-gene interaction:
  Identifying genetic risk factors to common but specific environmental factors. In 2006, found of association of sporadic ALS with variation in PON cluster of genes, which detoxify pesticides and indigenous toxins. Also published a second environmentally sensitive gene, CYP7B1 that is regulated by common environmental fungicides and affects the upper motor neuron.

• Genome wide Association Study - Phase II:
  Phase One of this study captured representative sets of DNA from 1,000 subject’s genome to help identify patterns within the samples, allowing researchers to focus on specific anomalies that are shared between various samples. The Second Phase is helping to verify the findings of Phase One and will allow the ALS community to move forward with targeted therapeutic interventions and, most significantly, present possible preventative strategies.

• Clinical Trials:
  The G93A SOD1 mouse model is in wide use, with over 300 publications. It was recently used in two independent studies to screen Lithium carbonate as a useful drug in ALS. Subsequent use in humans is encouraging and will lead to more trials. The Lab continues to screen drugs that would regulate SOD1, for uses in SOD1 linked ALS.

• Animal models of ALS:
  Develop animal models as new genes are identified. The immediate goal is the development of the ALSIN knockout models for ALS and primary lateral sclerosis (PLS).

For more information, visit the  Feinberg School of Medicine's Neuromuscular Disorders Program  web site.