Amyotrophic Lateral Sclerosis (ALS)

#EndALSwithMDA

MDA takes a big-picture perspective on neuromuscular diseases, including amyotrophic lateral sclerosis (ALS), so we can work across conditions to find effective treatments and cures.

With our collective strength, we encourage early diagnosis and action, support families in hometowns across the country, and uncover research breakthroughs to help everyone with ALS live longer, stronger lives.

What is amyotrophic lateral sclerosis (ALS)? ALS is a disease of the parts of the nervous system that control voluntary muscle movement. In ALS, motor neurons (nerve cells that control muscle cells) are gradually lost. As these motor neurons are lost, the muscles they control become weak and then nonfunctional, thus leading to muscle weakness, disability, and eventually death. ALS is the most common form of motor neuron disease.

The word amyotrophic comes from Greek roots that mean without nourishment to muscles and refers to the loss of signals nerve cells normally send to muscle cells. Lateral means to the side and refers to the location of the damage in the spinal cord. Sclerosis means hardened and refers to the hardened nature of the spinal cord in advanced ALS.

In the United States, ALS also is called Lou Gehrig's disease, named after the New York Yankee baseball player who lived with it until his death in 1941. This disease was first described by Dr. Jean-Martin Charcot in the 19th century.

Who gets ALS?

ALS usually strikes in late middle age (the late 50s is average) or later, although it can occur in young adults as well as in very elderly people. Some forms of ALS have their onset in youth. Men prior to the age of 65 or 70 are slightly more likely to develop ALS than are women. In the US, 1 to 3 new cases of ALS per 100,000 people are diagnosed every year; this is believed to be the same worldwide.

MDA partners with the Centers for Disease Control and Prevention to promote the National ALS Registry, the only national population-based registry in the U.S. that collects information to help scientists learn more about who gets ALS and what may cause it. Learn more by clicking below.

What causes ALS The causes of the vast majority of ALS cases are still unknown. Investigators theorize that some individuals may be genetically predisposed to developing the disease but do so only after coming into contact with an environmental trigger. The interaction of genetics and environment may hold clues as to why some individuals develop ALS.

Although the majority of ALS cases are sporadic, meaning there is no family history of the disease, about 10 percent of cases are familial, meaning the disease runs in the family. A common misconception is that only familial ALS is "genetic." Actually, both familial and sporadic ALS can stem from genetic causes, and some people who have a diagnosis of sporadic ALS may carry ALS-causing genetic mutations that can be passed on to offspring. A genetic counselor can help people with ALS understand inheritance and any associated risks for family members.

For a more detailed discussion of possible causes of sporadic ALS and the genetics of familial ALS, please see Causes/Inheritance.

What are the symptoms of ALS?

ALS results in muscles that are reduced in size (atrophic), weak, and soft, or muscles that are stiff, tight, and spastic. Muscle twitches and cramps are common; they occur because degenerating axons (long fibers extending from nerve-cell bodies) become irritable. 1 Symptoms may be limited to a single body region, or mild symptoms may affect more than one region. When ALS begins in the bulbar motor neurons, localized in the brainstem, the muscles used for swallowing and speaking are affected first. Rarely, symptoms begin in the respiratory muscles. 2. As ALS progresses, symptoms become more widespread, and some muscles become paralyzed while others are weakened or unaffected. In late-stage ALS, most voluntary muscles are paralyzed. The involuntary muscles, such as those that control the heartbeat, gastrointestinal tract and bowel, bladder, and sexual functions are not directly affected in ALS. Sensations, such as vision, hearing, and touch, are also unaffected. In many cases, ALS does not affect a person's thinking ability. However, as many as 50 percent of people with ALS develop some degree of cognitive (thinking) or behavioral abnormalities.3,4 For more information on ALS symptoms, see Signs and Symptoms and Medical Management.

What is the life expectancy in ALS?

Each person's disease course is unique.

From the time of diagnosis, most patients die within three to five years. Around 30 percent of ALS patients are alive beyond five years after diagnosis, and 10 to 20 percent of patients survive 10 years or more. Survival beyond 20 years is possible but rare. Factors associated with more favorable survival rates include a younger age, male gender, and limb rather than bulbar symptom onset. 5,6
For more information on the disease course, see Medical Management.

What can be done about ALS?

Medical interventions and technology have vastly improved the quality of life for people with ALS by assisting with breathing, nutrition, mobility, and communication. Proper management of symptoms, and proactive use of medical interventions and equipment, can make a positive difference in day-to-day living and potentially may lengthen life. The FDA-approved drug riluzole (brand name Rilutek) has been shown to slightly increase longevity.7

The U.S. Food and Drug Administration (FDA) on May 5, 2017, approved Edaravone (brand name Radicava) for the treatment of ALS. Radicava is thought to work by relieving the effects of oxidative stress, which has been suspected to play a role in the death of motor neurons in people with ALS. (Oxidative stress is an imbalance between the production of free radicals and the ability of the body to counteract or detoxify their harmful effects with antioxidants.) Targeting this pathway could potentially preserve motor neuron health, which could in turn keep muscles functional for a longer period of time. For more, see FDA Approves Radicava to Treat ALS and Questions and Answers About Newly FDA-Approved Radicava to Treat ALS.

What is the status of ALS research?

A number of strategies and approaches are being tested around the world, both in the laboratory and in human clinical trials. MDA's basic science program is constantly pursuing new avenues of research to understand the underlying causes of ALS, with a sharp focus on developing treatments.

One of the most significant breakthroughs in the last decade has been the discovery of a number of genes that, when flawed, cause ALS. In fact, the genes responsible for causing the majority of the familial forms of ALS are now known with some of these genes also having been found to be involved in sporadic ALS. Identification of these genes is crucial to moving research forward because it allows researchers to better understand the causes of ALS and design therapies to target them. For details about current ALS research, go to Research and Clinical Trials.

References

Chi, A., Mora, G. & Lauria, G. Pain in amyotrophic lateral sclerosis. The Lancet Neurology (2017). doi:10.1016/S1474-4422(16)30358-1 Hardiman, O., Van Den Berg, L. H. & Kiernan, M. C. Clinical diagnosis and management of amyotrophic lateral sclerosis. Nature Reviews Neurology (2011). doi:10.1038/nrneurol.2011.153 Strong, M. J., Lomen-Hoerth, C., Caselli, R. J., Bigio, E. H. & Yang, W. Cognitive impairment, frontotemporal dementia, and the motor neuron diseases. in Annals of Neurology (2003). doi:10.1002/ana.10574 Lomen-Hoerth, C. et al. Are amyotrophic lateral sclerosis patients cognitively normal Neurology (2003). doi:10.1212/01.WNL.0000055861.95202.8D Kim, W. K. et al. Study of 962 patients indicates progressive muscular atrophy is a form of ALS. Neurology (2009). doi:10.1212/WNL.0b013e3181c1dea3 Visser, J. et al. Disease course and prognostic factors of progressive muscular atrophy. Arch. Neurol. (2007). doi:10.1001/archneur.64.4.522 Miller, R. G., Mitchell, J. D., Lyon, M. & Moore, D. H. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders (2003). doi:10.1080/14660820310002601

MDA History

MDA's legacy of progress has always started with families at the heart of all we do. For more than 65 years, we have been committed to saving and improving the lives of kids and adults living with muscular dystrophy and related life-threatening diseases. We are proud of our rich history and grateful to the dedicated families and supporters who have made today's progress possible. The victories of our past will lead to tomorrow's treatments and cures.

How MDA Started

In June 1950, Paul Cohen, a prominent New York business leader living with muscular dystrophy, invited a group of individuals to meet in his Rye, New York, office. Each had a personal connection to muscular dystrophy, and the gathering focused on the urgent need to raise funds to advance research seeking treatments and cures for muscular dystrophy. The group — so vested in the fight against neuromuscular diseases — formed the organization that became the Muscular Dystrophy Association. That year, MDA’s first research grant for $1,500 was awarded to neuromuscular disease pioneer Ade. T. Milhorat, M.D.

Celebrities & Our Historic Telethon

MDA Families in the Spotlight

One of MDA’s first goals when it started in 1950 was to recruit celebrities who could help promote the newly created Muscular Dystrophy Association to the American public. Founder Paul Cohen met with renowned comedians and entertainers Dean Martin and Jerry Lewis to urge them to become champions for the cause. They agreed.
On Dec. 28, 1951, Martin and Lewis concluded their popular NBC network television show with a special appeal to support muscular dystrophy research. The comedic duo made a second national appeal the following month during its Jan. 4, 1952, network radio program.
Early to recognize the power of television to bolster awareness and raise income for MDA, Cohen pioneered the establishment of local telethons featuring a variety of stars to raise money. Thanks to early hosting commitments from top stars, including Robert Alda, Dick Van Dyke, Captain Video and Virginia Graham, MDA successfully broadcast five local telethons in two years.

The MDA Telethon Begins

On June 29-30, 1956, Martin and Lewis co-hosted their first MDA telethon from New York City’s famed Carnegie Hall. Jerry Lewis hosted subsequent telethons in 1957 and 1959. In 1966, the first MDA Jerry Lewis Labor Day Telethon was broadcast by a single station in New York WNEW-TV. The telecast was so successful that MDA selected Labor Day weekend for all future telethons. Then, with help from broadcasting icons Sylvester “Pat” Weaver and Robert M. Bennett, MDA created a “Love Network” of stations that in 1971 facilitated the nation’s first networked telethon.
Throughout the decades, the legendary Labor Day broadcast originated from different locations including New York, Las Vegas and Hollywood. The most successful fundraising event in the history of television, the show, with Lewis as its iconic host and with help from a legion of top celebrities and entertainers, raised nearly $2 billion during the years.
Thanks to unwavering public support of MDA's research and service programs through the telethon, pledges and other fundraising efforts, MDA became the first nonprofit organization to receive a Lifetime Achievement Award from the American Medical Association “for significant and lasting contributions to the health and welfare of humanity.”

Biggest Names in Show Business Show Their Support

Thousands of the biggest names in show business have appeared on the MDA telethon, including: Alan Alda, Jason Alexander, Woody Allen, Jack Benny, Milton Berle, Carol Burnett, Richard Burton, Johnny Carson, Johnny Cash, Billy Crystal, Doris Day, Robert DeNiro, Patty Duke, Jimmy Fallon, Don Francisco of “Sabado Gigante,” Jackie Gleason, Whoopi Goldberg, Woody Harrelson, Neil Patrick Harris, Larry King, Burt Lancaster, Jack Lemmon, Howie Mandel, Dr. Phil McGraw, Eddie Murphy, Bob Newhart, Paul Newman, Rosie O’Donnell, Gregory Peck, Regis Philbin, Ray Romano, Mickey Rooney, Adam Sandler, Jerry Seinfeld, William Shatner, Martin Short, Ed Sullivan, Barbara Walters, Betty White, Robin Williams and Oprah Winfrey.
Consider musical acts, too, and it’s easy to see why the telethon is so much a part of American history. The telethon audience has enjoyed thousands of hit performances by a diverse range of artists, including: Alabama, Count Basie, The Bee Gees, Tony Bennett, Clint Black, Jon Bon Jovi, Garth Brooks, Mariah Carey, Ray Charles, Cher, Kenny Chesney, Chicago, Phil Collins, The Commodores, Perry Como, Sammy Davis Jr., Gloria Estefan, Aretha Franklin, Josh Groban, Faith Hill, Enrique Iglesias, Julio Iglesias, Alan Jackson, The Jackson 5, Billy Joel, Elton John, Quincy Jones, Tom Jones, KISS, Eartha Kitt, Cyndi Lauper, John Lennon, Tracy Lawrence, Paul McCartney, Reba McEntire, Tim McGraw, John Mellencamp, Willie Nelson, Oak Ridge Boys, Donny and Marie Osmond, Dolly Parton, Tom Petty, Queen, Debbie Reynolds, LeAnn Rimes, Kid Rock, Kenny Rogers, Carlos Santana, Spice Girls, Ringo Starr, Rolling Stones, Sugarland, Randy Travis, Tina Turner, Eddie Van Halen, Clay Walker, Dionne Warwick, Stevie Wonder and Wynonna. Even the King of Pop, Michael Jackson, Paula Abdul, American Authors, Backstreet Boys, Luke Bryan, Carole King, Kenny Loggins, Bret Michaels, Pitbull, Rascal Flatts, Darius Rucker, Ryan Seacrest, Carrie Underwood, will.i.am. and many others performed on the MDA Telethon.
Despite the many celebrities that graced the telethon stage, some of the most riveting telethon moments came from courageous individuals and families taking the stage. From MDA National Goodwill Ambassadors, including Ben Teraberry, Mike Neufeldt, Kelly Mahoney, Ben Cumbo, Sarah Schwegel, Mattie Stepanek, Luke Christie, Abbey Umali, Bryson Foster and Reagan Imhoff; to other families sharing how MDA makes concrete differences in their lives; to MDA-funded researchers and clinicians describing why public support of MDA is so vital. A big part of the telethon’s success was letting people speak from the heart about the importance of the fight against muscular dystrophy and related diseases.

The End of an Era

On May 16, 2011, Jerry Lewis announced his retirement as host of the annual MDA Labor Day Telethon. MDA will be forever grateful to Jerry Lewis, a world-class humanitarian, for his indefatigable and inspiring work on behalf of kids and families with neuromuscular diseases, and for the countless dollars his commitment helped raise for critical research and services.
Throughout the decades, the broadcast industry evolved, bringing major changes in demographics and viewer habits, as well as rising production costs for live televised events. That meant the telethon needed to evolve too. In 2011, the 21½-hour show was streamlined to a more viewer friendly six-hour format. The following year, the show was streamlined further as a three-hour prime-time broadcast special that included performances and celebrity appearances from Hollywood, Nashville and New York. It also proudly featured a new name, the MDA Show of Strength Telethon.
In 2013 and 2014, the MDA Show of Strength Telethon became a two-hour entertainment-packed event carried exclusively on the ABC Television Network. The Show of Strength conveyed the same message of hope and progress as always while reaching out to younger viewers and supporters to strengthen MDA’s mission going forward.
On May 1, 2015, MDA made the difficult decision to end its historic telethon tradition. After careful consideration and analysis and as families and supporters began looking for new ways to support and get involved with the organization, MDA once again evolved with the times to create new opportunities through social media and other digital channels to inspire the nation in support of the fight against muscular dystrophy.

Transforming Lives

MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS and related neuromuscular diseases through innovations in science and innovations in care.
As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA’s MOVR is the first and only data hub that aggregates clinical, genetic and patient reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development.
MDA supports the largest network of multidisciplinary clinics providing best in class care at more than 150 of the nation’s top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families.

Pioneering Partners

In the early 1950s, several important sponsors became long-standing allies of MDA, including the Tall Cedars of Lebanon of North America, the National Association of Letter Carriers (NALC) and the International Association of Fire Fighters (IAFF).
The NALC quickly established a nationwide door-to-door campaign for MDA and continues to support MDA as a national sponsor today.
In the same time frame, a group of families affected by muscular dystrophy approached Local 718 fire station in Boston to ask professional fire fighters to help fight muscular dystrophy. Responding enthusiastically, the fire fighters took to the streets with their boots in hand to ask greater Boston to make donations that would be used to fight muscular dystrophy.
The Fill the Boot campaign was an instant success, with the IAFF membership passing a resolution to support MDA's fight against muscular dystrophy until treatments and cures are found. More than 60 years later, IAFF continues its tradition as the top fundraising organization for MDA today, raising more than $26 million each year to help MDA families live longer and grow stronger.