Current Treatments

Treatment of ALS is primarily a process of managing symptoms. As PALS get weaker, their symptoms change, their needs change, and consequently their treatments are always being modified. Treatment involves managing ALS symptoms through drugs, therapies, nutrition, dietary supplements, and adaptive equipment.

FDA approved drugs to slow ALS progression:

Rilutek (available by prescription) is the only drug approved by the Food and Drug Administration for treatment of ALS patients. Two randomized and placebo-controlled trials performed in both Europe and North America found a difference of about 2 to 3 months in the time to tracheostomy or death in favor of patients treated with Rilutek as compared to those receiving placebo. However, there was no statistical significant difference in mortality at the end of the trial. Measures of muscle strength and neurological function did not show improvement. Potential side-effects include fatigue, nausea, dizziness, diarrhea, anorexia, vertigo, and somnolence.. While the effect of Rilutek is modest, it is a significant development in that is the first ALS drug proven to be effective in over 130 years of research. Because the effect is modest, one must weigh the financial cost versus the benefit when electing to use Rilutek. Rilutek is expensive, over $600 for a 30 day supply, but it is covered under most health insurance policies. The National Organization for Rare Disorders (NORD) may be able to assist you with purchasing Rilutek if you do not have insurance.

Prescription and OTC Drugs for ALS Symptoms:

Condition	Potential Treatment Drugs - Generic (brand name)
Spasticity (stiffness)	Baclofen (Lioresal), Tizanidine (Zanaflex), Memantine, Tetrazepam, Carisoprodol (Soma), Dantrolene, Marinol
Fasciculations (twitching) and Muscle Cramping	Quinine Sulfate, Baclofen (Lioresal), Clonazepam (Klonopin), Carbamazepine (Tegretol), Phenytoin (Dilantin), Magnesium, Verapamil (Calan)
Depression	Fluoxetine (Prozac), Sertraline (Zoloft), Paroxetine (Paxil), Amitriptylene (Elavil), Imipramine (Tofranil), Nortriptyline (Pamelor), Fluvoxamine (Luvox)
Pseudobulbar Emotionalism (excessive laughing/crying)	Amitriptylene (Elavil), Fluvoxamine (Luvox), Lithium; L-DOPA
Gastric Reflux (heartburn)	Pepcid, Ranitidane (Zantac)
Thick Phlegm (mucus)	Guaifenesin, Robitussen, Propranolol (Inderal), Metoprolol (Toprol)
Sialorrhea (drooling or excessive salivation)	Amitriptylene (Elavil), Trihexyphenidyl Hydrochloride (Artane), Clonidine (Catapres), Propantheline (Pro-Banthine), Benztropine (Cogentin), Glycapyrrolate (Robinul), Transdermal Hyoscine (Scopolamine), Benadryl, Atropine (Sal-Tropine)
Constipation	Bulk-forming fiber laxative (FiberCon, Citrucel, Metamucil, etc.), Docusate Sodium (Correctol, Colace, Dulcolax), Lactulose (Constulose, Duphalac, Chronulac, Constilac)
Urinary Urgency	Toterodine (Detrol)
Breathing (early stages)	Flu vaccine, Pneumonia vaccine
Breathing (latter stages)	Morphine (Roxanol), Lorazepam (Ativan), Midazolam (Versed)

Experimental drugs to slow ALS progression:

The good news about ALS research is that there are a number of drugs currently being studied. Every ALS patient should consider participating in clinical research trials. All clinical trials have an experimental group and a control group. The experimental group receives the active medication and the control group receives the placebo, a pill with no active medication. Neither the doctor nor the patient knows whether or not they are receiving the active medication or the placebo. This is called a "double blind study." For more information on drug development and current clinical trials of experimental drugs contact the ALS Association, Muscular Dystrophy Association, ALS Therapy Development Foundation or Project ALS. Contact information is included in the National Government and Nonprofit Organizations section of this web site.

Antioxidants and Supplements:

Antioxidants - It is thought that antioxidants such as vitamin E, vitamin C, and beta-carotene may protect nerve cells from damage by toxic free radicals. However, the specific benefits of these compounds have not been tested scientifically in patients with ALS. A recent experiment using an animal model shows that adding vitamin E to their food delays onset of clinical disease and slows progression, but does not prolong survival. The Muscular Dystrophy Association gives ALS patients the following dosage recommendations for vitamins A, C, and E:

Vitamin C	1,000 mg 3 times a day ( 3 grams daily )
Vitamin E	800 units 3 times a day ( 2,400 units daily )
Beta-Carotene	10,000 units 3 times a day ( 30,000 units daily )

Foods rich in in antioxidants include alfalfa sprouts, beets, blueberries, brussels sprouts, broccoli, garlic, grapes, kale, spinach and strawberries. Foods rich in vitamin C include citrus fruits, green peppers, broccoli, tomatoes and strawberries. Other antioxidants which may be beneficial include coenzyme Q-10, grape seed extract, green tea, melatonin, NAC, DHEA, tocotrienol, garlic, alpha lipoic acid, and zinc. Patients should always seek the advice of their physician before making any dietary changes or taking any vitamins or supplements.

Coenzyme Q-10 - Researchers at Massachusetts General Hospital and Harvard Medical School in Boston have found that coenzyme Q10, a widely available over-the-counter compound, can combat nerve-cell degeneration in mice with ALS. The study is in the July 21 issue of Proceedings of the National Academy of Sciences.

M. Flint Beal of the Department of Neurology at Massachusetts General and also at Cornell University Medical Center in New York was part of the research team. "I'm not as yet recommending that people with ALS take coenzyme Q10," Beal said. "In order to make this recommendation, it will be necessary to do a clinical trial. I don't believe it would be harmful, but there is no evidence as yet that it will be beneficial."

Oral coenzyme Q10 significantly prolonged the lives of mice with mutated SOD1 genes and a disorder that closely resembles human ALS (which also sometimes results from mutations in this gene). Coenzyme Q10, even when given by mouth, was found to penetrate into brain tissue in general and into the mitochondria of brain cells.

"Both antioxidant effects and preservation of mitochondrial function may contribute to the observed neuroprotection," the authors say. "Although vitamin E supplementation delays disease onset in [such] mice, it has no effect on survival," the report continues. "This finding suggests that coenzyme Q10 may be more effective than vitamin E in the treatment of neurodegenerative diseases."

Word of Caution: The study was done on mice, so the results cannot be assumed to be the same for humans with Amyotrophic Lateral Sclerosis. As with any supplement or medication, please consult your physician before trying coenzyme Q10.

Creatine - Researchers have reported that Creatine, an over-the-counter supplement popular as a muscle builder among athletes, works twice as well in diseased mice as the only approved prescription drug available for treating Lou Gehrig's disease. The study,done by researchers at Harvard Medical School and Cornell University Medical College in Manhattan, was reported in March of 1999 in the science journal Nature Medicine.

The research studied mice with a genetic mutation that normally leads to the same type of motor neuron destruction as is seen in amyotrophic lateral sclerosis, which is also called Lou Gehrig's disease or ALS. The study found that animals given a diet high in creatine had the same amount of healthy muscle-controlling nerve cells as mice in the control group. The animals on the creatine supplements showed complete protection up to four months, when the disease would normally have begun to take hold, said Dr. Flint Beal, chairman of neurology at Cornell. And the creatine worked twice as well as Riluzole, the only federally approved drug for Lou Gehrig's disease.

Word of Caution: The study was done on mice, so the results cannot be assumed to be the same for humans with Amyotrophic Lateral Sclerosis. Also, this animal study showed neuroprotection before disease onset and did not study whether creatine extended life after disease progression was underway. As with any supplement or medication, please consult your physician before trying creatine.

NAC (N-acetyl-L-cysteine) - Researchers report that NAC (N-acetyl-L-cysteine), an agent that increases levels of the antioxidant glutathione, improves survival and delays onset of motor impairment in a mouse model of familial amyotrophic lateral sclerosis (ALS), according to US-based researchers.

Dr. Ashley I. Bush from Harvard Medical School in Boston and colleagues there and elsewhere explain that previous studies have shown that a mutation in copper/zinc superoxide dismutase (SOD1), which leads to increased levels of oxidative damage, has been implicated in the pathogenesis of ALS. In the present study, the authors investigated the effects of NAC in transgenic mice with a mutation in SOD1. Treatment with 1% NAC in drinking water significantly improved "both rotorod performance and survival," according to the paper published in the August 3, 2000 issue of NeuroReport.

The study team points out that previous research has shown that 50 mg/kg/day NAC has beneficial effects in patients with limb-onset sporadic ALS. In light of these results and their own findings Dr. Bush's team suggests that "further trials of oral NAC supplementation may be worth considering at higher doses, and possibly combining it with other safe over-the-counter supplements that specifically enhance the activity of the glutathione system."