What is ALS?

Amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's disease, is a progressive neurological disease affecting 30,000 Americans with about 5,000 new cases occurring in the United States each year.

ALS belongs to a class of disorders known as motor neuron diseases. Motor neurons are nerve cells located in the brain, brainstem, and spinal cord that serve as controlling units and vital communication links between the nervous system and the voluntary muscles of the body. The loss of these cells causes the muscles under their control to weaken and waste away, leading to paralysis. ALS is usually fatal within five years of diagnosis.

Symptoms of ALS

ALS manifests itself in different ways, depending on which muscles weaken first. Symptoms may include:

  • Tripping and falling
  • Loss of control in hands and arms
  • Difficulty speaking, swallowing and/or breathing
  • Persistent fatigue
  • Twitching and cramping.

ALS strikes in mid-life, and men are about one-and-a-half times more likely to have the disease as women. More information on ALS symptoms listed below.

Because ALS affects only motor neurons, the disease does not impair a person's mind, personality, intelligence, or memory. Additionally, it does not affect a person's ability to see, smell, taste, hear, or recognize touch. Patients usually maintain control of eye muscles and bladder and bowel functions.

Is there a cure for ALS?

There is no known cure for ALS, nor is there a therapy to prevent or reverse its course.

Riluzole is the only FDA-approved drug shown to prolong the survival of people with ALS – but only for a few extra months. Riluzole is believed to minimize damage to motor neurons due to the release of the neurotransmitter glutamate. ALS patients have raised levels of glutamate in the fluid bathing the brain and spinal cord.

Riluzole may also extend the time before a person needs ventilation support. Riluzole does not reverse the damage already done to motor neurons however, and people taking the drug must be monitored for liver damage and other possible side effects.

In 2011, the FDA approved the NeuRx Diaphragm Pacing System® (DPS) for ALS patients experiencing breathing issues. Clinical trials demonstrated that DPS neurostimulation helped ALS patients live longer and sleep better than with standard care.

ALS experts have identified numerous compounds that show promise for treating the disease. Several drugs and cell therapies are currently being tested in patients. A company called Neuralstem has enrolled several dozen patients in a clinical trial testing neural stem cells; there have been no safety issues and some indication that the cells are beneficial.

There is strong evidence that trophic factors, molecules that nurture and protect cells, can rescue dying neurons in animal models of ALS. Indeed, targeted delivery to a vulnerable cell may be beneficial. So far, human trials have failed to follow up on success in animals. This work is still in progress.

A drug called arimoclomol, originally developed to treat diabetic complications, inhibits progression of ALS in a mouse model of the disease. Arimoclomol is thought to amplify "molecular chaperone" proteins, normally found in all cells of the body; these cells may protect a motor nerve cell against toxic proteins, repairing those that are believed to cause diseases such as ALS.

Arimoclomol appears to accelerate the regeneration of previously damaged nerves in animals. Early phase clinical trials have shown the drug to be safe in humans; more tests are ongoing for dose and treatment.

Drug cocktails

Recent mouse model studies of ALS showed dramatic benefits using a combination of drugs, including riluzole, nimodipine (a calcium channel blocker used in the treatment of acute stroke and migraine headache) and minocycline (an antibiotic that may block inflammation). The compounds given together appear to delay cell death, prevent nerve cell loss, and reduce inflammation. Read more on ALS clinical trials.

Physical or occupational therapy and special equipment can enhance independence and safety throughout the course of ALS. Low-impact aerobic exercise such as walking, swimming, and stationary bicycling can strengthen unaffected muscles, prevent deconditioning, improve cardiovascular health, and help patients fight fatigue and depression.

Range-of-motion and stretching exercises can help prevent painful spasticity and muscle contractures (shortening of muscles, limits joint movement).

Occupational therapists can suggest devices such as ramps, braces, walkers, and wheelchairs that help people conserve energy and remain mobile, while making it easier to perform activities of daily living.

Research holds great promise for treatments for ALS, including drugs, cell transplants, gene therapy, and immune system modulation.

Respiratory weakness and excess saliva

People living with ALS are at risk for pneumonia and pulmonary embolism. Indicators of deteriorating respiratory status can include:

  • Difficulty breathing, especially when lying down or after meals
  • Lethargy, fatigue and drowsiness
  • Confusion
  • Anxiety and irritability
  • Loss of appetite
  • Morning headaches
  • Depression

When the muscles that assist in breathing weaken, use of ventilatory assistance (intermittent positive pressure ventilation, IPPV; or bi-level positive airway pressure, BiPAP) may be used to aid breathing during sleep. When muscles are no longer able to maintain oxygen and carbon dioxide levels, these devices may be required full-time.

Another problem common to many people with ALS is the inability to cough forcefully enough to clear away even normal amounts of mucus. People are advised to make sure their fluid intake is sufficient to keep the secretions thin. Some take an over-the-counter cough medicine containing the expectorant guaifenesin, a mucus thinner.

A weak cough can be made more effective by quad coughing (assisting a cough by applying a sort of Heimlich-like maneuver as the patient coughs), supplying fuller breaths with an ambu-bag to improve the cough, or using a device such as a "cofflator" or "in-exsufflator" (delivers deep breaths through a mask and then quickly reverses to negative pressure to simulate a cough).

While people with ALS do not overproduce saliva, their swallowing problems can create sialorrhea, or excessive salivation and drooling. Sialorrhea can be undertreated -- it may take trials of several medications until one provides relief without undesirable side effects.

Muscle problems and spasticity

Spasticity is present in some people with ALS. It causes a tightening of muscles and a stiffening of the arms, legs, back, abdomen, or neck.

Spasms can be triggered by a simple touch and can be painful especially if it sets off muscle cramps, common in ALS because of muscle fatigue. Cramps can be very painful but become less severe with time – weakening muscles can't tighten into a cramp anymore.

Fasciculation (muscle twitching) is common, too, though these are not painful so much as annoying.

Loss of communication

While the loss of the ability to communicate is not life-threatening or painful, being "locked-in" is a very frustrating aspect of ALS.

Although assistive technology offers many solutions, it may be underutilized because people lack information about their options.

Assistive devices range from simple call buttons and sensitive switches to small communication boards that speak pre-recorded words and messages. Also, equipment is available to magnify a weak whisper into audible speech. If a person can move nearly any body part, there is potential for some basic communication.

In experiments using brain waves, people who are locked-in due to ALS have learned to communicate by way of a computer using only their thoughts. For example, trials of the BrainGate System, which implants a sensor in the brain to transmit, have shown that neural signals associated with the intent to move a limb can be "decoded" by a computer in real-time and used to operate external devices, including robot arms. Trials are ongoing.

Numerous communication devices are on the market and can be found in many home health dealers or shopping sites. The ALS Association supports a list of products and vendors.

ALS support and resources

Social workers, home care and hospice nurses help patients, families, and other caregivers with the medical, emotional, and financial challenges of coping with ALS, particularly during the final stages of the disease.

Social workers provide support such as assistance in obtaining financial aid, arranging durable power of attorney, preparing a living will, and finding support groups for patients and caregivers.

If you are looking for more information on ALS or have a specific question, our information specialists are available business weekdays, Monday through Friday, toll-free at 800-539-7309 from 9:00 am to 5:00 pm ET.

Additionally, the Reeve Foundation maintains an ALS fact sheet with additional resources from trusted Reeve Foundation sources. Check out our repository of fact sheets on hundreds of topics ranging from state resources to secondary complications of paralysis.

We encourage you to reach out to ALS support groups and organizations, including:

  • ALS Association features news, research support, and resources; it offers a national network of support groups, clinics, and specialty hospitals.
  • The ALS Therapy Development Institute is a nonprofit biotechnology company working to discover treatments.
  • Project ALS aligns researchers and doctors from many disciplines to collaborate and share data openly in four main areas: basic research, genetics, stem cells, and drug screening.

Sources: The National Institute of Neurological Disorders and Stroke, ALS Association

This project was supported, in part by grant number 90PR3002, from the U.S. Administration for Community Living, Department of Health and Human Services, Washington, D.C. 20201. Grantees undertaking projects under government sponsorship are encouraged to express freely their findings and conclusions. Points of view or opinions do not, therefore, necessarily represent official Administration for Community Living policy.