In July 2012, the DSMB for the NINDS-sponsored clinical trial of ceftriaxone in ALS recommended that based on existing data the trial be stopped because the study was unlikely to reach the pre-determined efficacy criteria. The NINDS leadership concurred. Pre-clinical research identified ceftriaxone as a promising treatment for ALS therefore it was important for people with ALS to find out if the drug could be beneficial in ameliorating the disease. The study used a novel seamless adaptive design. Final analysis and presentation of the results will occur after completion of site monitoring and database lock. The important contributions of patients, their families and the hard work of the investigators and their teams made it possible to implement the trial. While all had hoped for a more positive result, the trial has moved ALS research forward.
Amyotrophic lateral sclerosis (ALS), sometimes called Lou Gehrig's disease or classical motor neuron disease, is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles. In ALS, both the upper motor neurons and the lower motor neurons degenerate or die, ceasing to send messages to muscles. Unable to function, the muscles gradually weaken, waste away, and twitch. Eventually the ability of the brain to start and control voluntary movement is lost. Symptoms are usually first noticed in the arms and hands, legs, or swallowing muscles. Muscle weakness and atrophy occur on both sides of the body. Individuals with ALS lose their strength and the ability to move their arms and legs, and to hold the body upright. When muscles in the diaphragm and chest wall fail to function properly, individuals lose the ability to breathe without ventilatory support. The disease does not affect a person's ability to see, smell, taste, hear, or recognize touch. Although the disease does not usually impair a person's mind or personality, several recent studies suggest that some people with ALS may develop cognitive problems involving word fluency, decision-making, and memory. The cause of ALS is not known, and scientists do not yet know why ALS strikes some people and not others.
No cure has yet been found for ALS. However, the drug riluzole--the only prescribed drug approved by the Food and Drug Administration
to treat ALS--prolongs life by 2-3 months but does not relieve symptoms. Other treatments are designed to relieve symptoms
and improve the quality of life for people with ALS. Drugs are available to help individuals with spasticity, pain, panic
attacks, and depression. Physical therapy, occupational therapy, and rehabilitation may help to prevent joint immobility
and slow muscle weakness and atrophy. Individuals with ALS may eventually consider forms of mechanical ventilation (respirators).
The National Institute of Neurological Disorders and Stroke (NINDS) conducts research in its laboratories at the National
Institutes of Health (NIH) and also supports additional research through grants to major medical institutions across the country.
The goals of this research are to find the cause or causes of ALS, understand the mechanisms involved in the progression of
the disease, and develop effective treatments.
National ALS Registry Both the NINDS and the Centers of Disease Control and Prevention (CDC) are committed to studies of disease patterns or risk
factors among persons with ALS in order to better understand the causes of ALS, the mechanisms involved in the progression
of the disease, and to develop effective treatments. In keeping with this goal, the CDC has launched the National ALS Registry,
a program to collect, manage and analyze data about persons with ALS. The Registry includes data from national databases as
well as de-identified information provided by persons with ALS. Persons living with ALS who choose to participate can add
their information to the Registry by clicking the button below.
ALS Association 1275 K Street, N.W. Suite 1050 Washington, DC 20005 advocacy@alsa-national.org http://www.alsa.org Tel: 202-407-8580 Fax: 202-289-6801 |
Les Turner ALS Foundation 5550 W. Touhy Avenue Suite 302 Skokie, IL 60077-3254 info@lesturnerals.org http://www.lesturnerals.org Tel: 888-ALS-1107 847-679-3311 Fax: 847-679-9109 |
Muscular Dystrophy Association 3300 East Sunrise Drive Tucson, AZ 85718-3208 mda@mdausa.org http://www.mda.org Tel: 520-529-2000 800-572-1717 Fax: 520-529-5300 |
Project ALS 3960 Broadway Suite 420 New York, NY 10032 info@projectals.org http://www.projectals.org Tel: 212-420-7382 800-603-0270 Fax: 212-420-7387 |
ALS Therapy Development Institute 300 Technology Square Suite 400 Cambridge, MA 02139 info@als.net http://www.als.net Tel: 617-441-7200 Fax: 617-441-7299 |
Prize4Life P.O. Box 425783 Cambridge, MA 02142 contact@prize4life.org http://www.prize4life.org Tel: 617-500-7527 |
Prepared by:
Office of Communications and Public Liaison
National Institute of Neurological Disorders and Stroke
National Institutes of Health
Bethesda, MD 20892
NINDS health-related material is provided for information purposes only and does not necessarily represent endorsement by or an official position of the National Institute of Neurological Disorders and Stroke or any other Federal agency. Advice on the treatment or care of an individual patient should be obtained through consultation with a physician who has examined that patient or is familiar with that patient's medical history.
All NINDS-prepared information is in the public domain and may be freely copied. Credit to the NINDS or the NIH is appreciated.
Last updated December 20, 2012