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Treatments for ALS

In December 1995 the U.S. Food and Drug Administration approved the drug riluzole (brand name Rilutek) for ALS. The drug seems to have a modest effect, prolonging survival by a few months. It’s the only prescription drug currently listed for ALS treatment.

Riluzole works by partially blocking glutamate, a nervous system chemical that carries signals between cells in the brain and between the brain and spinal cord. Excess glutamate or abnormal glutamate clearance may be involved in ALS.

Another glutamate-inhibiting drug is topiramate (Topamax), which is on the market to treat seizures. It’s now being tested for possible benefit in ALS.

Neurotrophic Factors

An additional theme in ALS drug development has been a group of substances known as neurotrophic ("nerve-nourishing") factors. In laboratory experiments, these natural nervous system chemicals have been shown to help nerve cells survive under adverse conditions. But results of trials of neurotrophic factors conducted in the 1990s in people with ALS were disappointing. As of 2003, the neurotrophic factor known as insulin-like growth factor 1 (IGF-1) is still under investigation.

A drug that increases the body’s own production of neurotrophic factors, xaliproden, has shown some positive trends with respect to survival and disease progression in ALS after preliminary analysis of data from a large study. Further analysis is under way.

Other Drugs

Other types of drugs being studied in ALS include celecoxib (Celebrex), which is mainly employed to treat arthritis. This drug may be able to interfere with glutamate and with the production of "free radicals," toxic chemical by-products of various cellular activities. It’s known as a COX-2 inhibitor and anti-inflammatory medication.

The antibiotic minocycline (Minocin, Dynacin) is another subject of study in ALS. This drug can block caspase enzymes, which are part of a "cell death" pathway that could be involved in ALS.

Other researchers are studying tamoxifen (Nolvadex), which is used in breast cancer therapy. Preliminary evidence suggests it may be helpful in ALS through its action against a substance called protein kinase C.

In addition to these prescription drugs, many physicians recommend various over-the-counter substances, including vitamin E, coenzyme Q10 and creatine. These compounds have shown potentially modest benefits in ALS, and studies are continuing.

Symptom Control

Although there are few drugs now available that substantially influence the course of ALS, doctors can prescribe several treatments to help control some of the symptoms of the
disease.

For example, there are medications to treat muscle cramps and spasms, help clear respiratory secretions from the bronchial tree, dry up excess saliva (a problem when mouth muscle weakness results in drooling), relieve constipation (a problem when abdominal muscles weaken), and minimize anxiety and depression.

One drug, a combination of dextromethorphan and quinidine, is being tested for its potential to reduce unwanted excessive crying and laughing. These reactions, essentially muscular in nature, are thought to occur in ALS because of some loss of neurologic control over these muscles.

Antibiotics can be prescribed to treat respiratory and other infections. Your doctor will guide you about flu and pneumonia immunizations.

Therapies and Nutrition

Physical therapy to maintain full range of motion of the joints and as much mobility as possible; occupational therapy to help with adaptive equipment and new methods for accomplishing daily activities; and respiratory therapy and the prescription of ventilation devices to assist breathing, may all be involved in the treatment of ALS at various stages.

Speech therapy and devices that substitute for speech can help compensate for loss of muscle control in the mouth and throat.

Maintaining optimal nutrition is also an important part of sustaining health for as long as possible. The advice of a nutritionist or dietician, as well as specialists in swallowing and sometimes a gastroenterologist, can be helpful for many patients and their families. In some situations, a feeding tube into the stomach is offered as an alternative to using weak swallowing muscles, which pose a choking hazard.

Psychotherapy or counseling to help the patient and family adjust to a very difficult situation may become part of the treatment program at different stages of the illness.

Practice Guidelines

The American Academy of Neurology has developed a "practice parameter" for physicians treating people with ALS.

Published in 1999, this set of guidelines lists standards, guidelines and options for a variety of treatment issues in ALS, ranging from diagnosis to respiratory care to pain management.

If you or your doctor would like to see the guidelines in detail, go to www.aan.com/professionals/practice/pdfs/g10058.pdf.


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