ALS - Lou Gehrig’s Disease

Amyotrophic Lateral Sclerosis is a disease of the brain and spinal cord which causes progressive muscle weakness. Amyotrophic means that motor neurons--nerves that nourish and signal muscles--wither and die, causing the muscles themselves to shrink and atrophy. Both upper motor neurons, in the brain, and lower motor neurons, die in the disease. Lateral refers to the outer part of the spinal cord tracts where nerve fibers are injured. Sclerosis means that these tracts harden. ALS is also called Lou Gehrig’s disease, after the famous baseball player who died from the disease in 1941, at a time when little was known about it.

Hope Center Scientists and Clinicians who Work on ALS

Robert H. Baloh M.D., Ph.D.
Assistant Professor of Neurology

Neurodegenerative diseases of the peripheral nervous system

Nigel Cairns Ph.D., MRCPath
Research Associate Professor of Neurology and Pathology & Immunology; Director, Betty Martz Laboratory for Neurodegenerative Research

Protein misfolding, inclusion formation and neurodegeneration in Frontotemporal Dementias

Valeria Cavalli Ph.D.
Assistant Professor of Anatomy and Neurobiology

Axonal Transport, Injury Signaling and Nerve Regeneration

Carl Frieden Ph.D.
Professor of Biochemistry & Molecular Biophysics

Protein folding, structure

Eugene M. Johnson Ph.D.
Professor of Neurology and Developmental Biology; Hope Center Steering Committee

Biological function and pharmacological potential of GFL neurotrophic factors

Jeffrey D. Milbrandt M.D., Ph.D.
James S. McDonnell Professor and Head of the Department of Genetics, Professor of Neurology; Professor of Pathology & Immunology and Internal Medicine; Hope Center Steering Committee

Axonal degeneration, regulation of myelination, neuronal energetics and mitochondrial function in neuropathy and neurodegenerative disease

Timothy M. Miller M.D., Ph.D.
Assistant Professor of Neurology

ALS therapeutics

Rohit V. Pappu Ph.D.
Associate Professor of Biomedical Engineering

Alzheimer's disease; biophysics; mathematical modeling; thermodynamics

Alan Pestronk M.D.
Professor of Neurology and Pathology; Director of the Neuromuscular Division

Diagnosis, pathogenesis and treatment of immune, hereditary and degenerative neuromuscular disorders

B. Joy Snider M.D., Ph.D.
Assistant Professor of Neurology

Mechanisms of protein degradation and intracellular calcium homeostasis in neurodegenerative disorders

Read about Hope Center research on ALS

New ALS Mouse Model Closely Resembles "Typical" ALS

Like humans with ALS, the new mouse engineered by Hope Center investigator Robert Baloh, MD, PhD, develops progressive paralysis; loses muscle mass and motor neurons, and eventually dies of the disorder. These similarities to human ALS make it an exceptional model for developing and testing new treatments for human ALS. Read More >

Dr. Jeffrey Milbrandt Becomes Chair of Genetics Department

Jeffery Milbrandt, MD, Ph.D, will succeed interim head of Genetics Susan Dutcher, Ph.D., who has held the position since June 2006. In addition to being a Hope Center investigator, Milbrandt serves on the Hope Center's steering committee. Read More >

Meet Tim Miller: Director of the Hobler Lab for ALS Research

Elisa Reeves of Hope Happens interviewed Dr. Timothy Miller, MD/PhD, Assistant Professor of Neurology at Washington University School of Medicine, about joining the Hope Center faculty and his ALS research. Miller is the first director of the Christopher Wells Hobler Laboratory for ALS Research. Read More >

Hope Center profiled in Washington University Magazine

Spring 2006 — Research at the Hope Center for Neurological Disorders, supported by Hope Happens (formerly ALS Hope,) provides new hope for patients suffering from Amyotrophic Lateral Sclerosis (Lou Gehrig's Disease,) Alzheimer's, Multiple Sclerosis, and other degenerative neurological disorders through multi-disciplinary, team-oriented approach. Read More >

The Hope Center for Neurological Disorders helps ensure forward motion through groundbreaking research and treatments

Research shows that diseases of the brain, spinal cord and nerves have common threads that can only be discovered on a fundamental level. When we understand these shared disease pathways, we can begin to find cures. Read More >

Alzheimer's genetics expert Goate turns to ALS

A member of the Departments of Psychiatry and Genetics at Washington University School of Medicine since 1992, Alison Goate, Ph.D. is the Samuel and Mae S. Ludwig Professor of Genetics in Psychiatry and professor of genetics and of neurology. Her 16-person lab is devoted to genetic research on Alzheimer's disease (AD), tauopathies, and alcoholism—and since January 2005, the molecular underpinnings of amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease. Read More >

Tracking a gene mutation to treat its effects

Robert H. Baloh, MD, PhD, deciphers the molecular mechanics of neurodegenerative disease. Two of his research focuses are amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, and Charcot- Marie-Tooth disease (CMT). Read More >

Anneliese Schaefer gets ALS research funding

The Hope Center's Annaliese Schaefer has won a one-year grant for basic research in amyotrophic lateral sclerosis (ALS). Read More >

Hope Center investigators identify new ALS gene

Research conducted at the Hope Center for Neurological Disorders has linked a mutation in the TDP-43 gene to ALS. Senior researcher Nigel Cairns, Ph.D, says the discovery has implications for research and treatment of both inherited and sporadic forms of ALS. Read More >

Robert Willson regularly travels from Overland Park, KS, to receive treatment from neurologist Tim Miller, MD. Willson's Amyotrophic Lateral Sclerosis (ALS) progressively robs him of mobility and strength.

Information about ALS

We don’t know what causes ALS. Between five and ten percent of ALS patients have a family history of the disease, implying a genetic component. While ALS can strike at any age, it usually occurs in people between 40 and 70 years old. About 5,000 people in the United States are diagnosed with ALS each year; about 20,000 live with it.

There is no known cure for ALS, which eventually results in paralysis—often, but not always, between three and five years after diagnosis (10% of patients live longer). ALS does not affect the mind or senses. Its progress can be sporadic. Most ALS patients eventually require a respirator to breathe. The drug Riluzole® seems to modestly extend survival time, and has been approved by the FDA.

How is ALS diagnosed?

The first signs of ALS are often arm or leg weakness, problems with speaking or swallowing, loss of fine motor control, muscle twitches or cramps, loss of muscle bulk, and/or tight or stiff muscles. Some people laugh or cry more easily.

No specific test diagnoses ALS. Nerve conduction studies and electromyography (EMG) can measure declines in nerve function. Tests of blood tests and spinal fluid, along with brain imaging, can rule out other conditions.

What are some research directions in ALS?

Many promising leads are being pursued. One line of research teases out possible genetic causative factors from genetic susceptibility (i.e., some people might inherit an increased susceptibility to certain “triggers” for ALS).

Some studies suggest that night-time breathing assistance in the early stages can provide benefits. Current treatments can manage symptoms, and ALS patients work with speech pathologists, physical therapists, and other specialists, usually coordinated by a neurologist as the primary physician.

U.S. Government Sites

NINDS ALS Information page