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Researcher to study Lou Gehrig’s Disease

Sept. 14, 2012 College News - The Medical College of Wisconsin has received a $40,000 grant from the ALS Association’s Jeff Kaufman Fund to study the impact of skeletal muscle on the progression of ALS, or Lou Gehrig’s Disease, as it’s commonly known.

Allison Ebert, PhD, Assistant Professor of Cell Biology, Neurobiology and Anatomy, is the primary investigator of the grant.

ALS is an incurable and fatal disease in which muscle progressively weakens, resulting in paralysis.  The nerve cells which control the body’s movements deteriorate and die; the neurons in the brain and spine follow. In the United States, 14 cases of ALS are diagnosed each day. The average expected survival time is three to five years, and at any given time, approximately 30,000 people in the U.S. are living with ALS.

In her research project, Dr. Ebert plans to develop a novel system to generate and characterize the skeletal muscle from ALS patient’s induced pluripotent stem cells (iPSCs), which are stem cells generated from adult tissue.  Studies suggest that the loss of neurons and skeletal muscle connection may occur very early in patients with ALS, which presents a potential therapeutic target. 

Dr. Ebert will use iPSCs from patients with ALS and health adults to create skeletal muscle.  That muscle tissue will be combined in the lab with iPSC derived neurons to observe the interactions between the two in ALS patients versus the healthy controls.

“Understanding the progression of this disease at a molecular level will help us target areas for early detection,” said Dr. Ebert. “I am grateful to the ALS Association for its generous support of our research.”

The ALS Association’s Jeff Kaufman Fund, established to fund cutting-edge research from proceeds of the ALS Association-Wisconsin Chapter’s Evening of Hope.

“We are very pleased to announce these new research grants to some of the most promising ALS researchers in the U.S. and around the world,” said Lucie Bruijn, Ph.D., Chief Scientist for The ALS Association. “The ALS Association looks forward to sharing the results of these diverse studies, which we hope will provide pieces to the complex puzzle of this devastating disease.”

The studies will help understand the disease process of ALS, and could lead to potential targeted therapies in the future.

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