ALS medications & treatments

Amyotrophic Lateral Sclerosis (ALS), once commonly known as Lou Gehrig’s disease, is a progressive neuromuscular disease that affects as many as 30,000 Americans, and 200,000 people worldwide. In the US, 1 to 3 new cases of ALS per 100,000 people are diagnosed every year. It is believed to be the same worldwide. ALS can occur at any age but most people develop it between 40 to 70 years of age. It is estimated that 4 out of 5 people with ALS die within 2 to 5 years of being diagnosed.

What is ALS?

Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease, is a progressive disease characterized by degeneration of nerve cells in the brain (upper motor neurons) and spinal cord (lower motor neurons) that control voluntary muscle movements. When these cells die off, it causes the muscle tissues to atrophy (waste away). This leads to muscle weakness and loss of muscle control, which can make it difficult to walk, talk, swallow, and breathe. ALS does not seem to affect your mental function, vision, hearing, or sense of taste, smell, or touch.

Most people with ALS die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear.

Causes of ALS

ALS is a common neuromuscular disease that affects people of all races and ethnicities, although Caucasians and non-Hispanics are most likely to develop the disease. Only about 10% of cases are considered familial ALS, where there is a known family history of the disease. If you have familial ALS, there is a 50% chance that your children will inherit the gene mutation and may develop the disease. For the other 90% of cases, they have sporadic ALS, where there is no family history or genetic mutation linked to ALS.

While experts are not sure what causes ALS, some other risk factors include:

• Age. ALS risk increases with age and is most common after age 60.
• Gender. Men are slightly more likely than women to develop ALS. This difference disappears after age 70.
• Smoking. In a case-control study in New England, cigarette smoking was associated with a significant 70% increase in ALS risk.
• Military service. Studies suggest that military veterans are about twice as likely to develop ALS. The reason for this increased risk is not clear, but it may be due to exposure to environmental toxins such as lead and pesticides.

While there is currently no cure for ALS, the right combination of treatments can slow the progression of the disease and help people with ALS live a good quality of life.

How is ALS diagnosed?

ALS is a difficult disease to diagnose as there is no single test for ALS. It is typically diagnosed based on your history of the symptoms, a thorough neurological exam, a review of your medical history, and a series of tests to rule out other diseases.

ALS symptoms

The initial symptoms of ALS can vary significantly between people. Some symptoms you may notice include:

• Muscle weakness in your arms or legs (limb onset ALS)
• Difficulty with speech or swallowing (bulbar onset ALS)
• Muscle twitching
• Muscle cramps
• Dropping things
• Slurred or thick speech and problems projecting your voice
• Difficulty walking
• Tripping and falling
• Inappropriate or uncontrolled crying or laughing (pseudobulbar affect or PBA)

As ALS progresses, it can cause most voluntary muscles to become paralyzed. This leads to shortness of breath and difficulty in breathing and swallowing, which is what eventually leads to a person’s death.

Along with your history of symptoms, your doctor may include some of the following tests and procedures:

• Electromyography (EMG). This test uses electrodes that are inserted into a muscle, or placed on the skin over a muscle or muscle group to detect and evaluate the electrical activity of your muscles when contracting and at rest to help diagnose ALS.
• Nerve conduction study (NCS). This test is similar to an EMG but it measures your nerves’ ability to send signals along the nerve or to muscles in different areas of your body.
• Magnetic resonance imaging (MRI). This non-invasive procedure produces detailed images that can be used to look for any damage to the brain and spinal cord.
• Blood and urine tests. These tests may look at your thyroid and parathyroid levels, as well as look for any heavy metals or environmental toxins in your blood. They are typically used to rule out the possibility of other causes for your symptoms.
• Spinal tap. This procedure is used to rule out other diseases and examines fluid withdrawn from the spinal column.
• Muscle biopsy. This test uses a small tissue sample taken from your muscle to rule out other muscle diseases other than ALS.

What are some ALS treatment options?

Treatments will not reverse the damage that has already occurred in ALS patients, but they can slow down the progression of ALS, prevent complications and help improve your quality of life.

Medications

The U.S. Food and Drug Administration (FDA) currently has approved 2 drugs to treat ALS:

• Rilutek (riluzole). This oral medication reduces damage to your motor neurons by decreasing levels of glutamate, which transports messages between nerve cells and motor neurons. Clinical trials with this medication showed that it prolonged survival by a few months in patients with the bulbar form of the disease. Patients with swallowing difficulties may have better success with the thickened liquid form (Tiglutik) or the dissolving tablet (Exservan).
• Radicava (edaravone). This injectable medication is typically given via an IV infusion. Edaravone is believed to work by reducing the effects of oxidative stress, which has been thought to play a role in the death of nerve cells. This can help preserve motor neuron health, which would keep muscles functioning for a longer period of time.
• Nuedexta (dextromethorphan/quinidine). This oral medication is used to treat pseudobulbar affect (PBA), which is a medical condition that causes involuntary, sudden, and frequent episodes of crying and/or laughing in people living with certain neurologic conditions (ALS, stroke, multiple sclerosis, Alzheimer’s disease) or brain injury.

Physical therapy

Physical therapy can help you stay mobile and keep some independence if you have ALS. This typically involves using low-impact aerobic exercises such as walking, swimming, or riding a stationary bicycle. Physical therapy can with muscle strength and help ease discomfort from stiff muscles, cramps, and fluid retention.

Occupational therapy

An occupational therapist can help you find equipment such as ramps, braces, wheelchairs, and electric beds that can help you perform daily activities such as getting dressed, eating, using the toilet, and bathing.

Speech therapy

If you have difficulty speaking due to ALS, a speech therapist can teach you techniques to speak louder and be easier to understand. They can also help find other ways to communicate such as an alphabet board, computer-based speech synthesizers, and other non-verbal techniques.

Nutritional counseling

Nutritionists can give you and your caregivers suggestions for foods that are easy to swallow and provide you with enough calories, fiber, nutrients, and fluids you need. They may recommend a feeding tube once swallowing becomes too difficult.

What is the best medication for ALS?

The best medication for the treatment of ALS will depend on the individual’s specific medical ALS, medical history, medications that the individual is already taking that may potentially interact with ALS medications, and the individual’s potential response to the treatment. It is advisable to always speak with your healthcare provider about the best medication for you. The table below includes a list of the most prescribed or over-the-counter ALS medications approved by the Food and Drug Administration (FDA).

Best medications for ALS

Your healthcare provider will determine the right dosage based on your response to the treatment, medical ALS, weight, and age. Other possible side effects may exist; this is not a complete list.

What are the most common side effects of ALS medications?

As with all medicines, those used for ALS will have some side effects, depending on the class you are taking:

• Rilutek, Tiglutik, and Exservan (riluzole) can all cause a loss of sensation in your mouth, weakness, nausea, decreased lung function, high blood pressure (hypertension), and dry mouth.
• Radicava and Radicava ORS can cause bruising, headache, skin irritation, eczema, and respiratory failure.
• Nuedexta (dextromethorphan/quinidine) can cause diarrhea, dizziness, cough, vomiting, and weakness.

What are some home remedies for ALS?

There is no cure or natural way to treat ALS. Diet and lifestyle changes have a minimal effect on the course of the disease but can make you or your loved one feel more independent. Some aids to help manage your condition and prevent falls and other complications include:

• Electronic communication devices
• Wheelchairs
• Ramps
• Wrist and leg braces
• Lift chairs
• Hospital-style beds
• Devices and supports to help you walk
• Special grips for writing and eating utensils
• Button hooks and zipper pulls to help get dressed or undressed

Frequently asked questions about ALS

Why is ALS called Lou Gehrig’s disease?

Lou Gehrig was a famous baseball player in the 1920s and 1930s. ALS ended his career. He died of the disease a few years after getting diagnosed.

What are the first signs and symptoms of ALS?

Symptoms will vary from person to person and in the early stages of ALS, they may be overlooked. Some common early symptoms include:

• Muscle weakness in your arms or legs
• Difficulty with speech or swallowing
• Muscle twitching
• Muscle cramps
• Dropping things
• Slurred or thick speech and problems projecting your voice
• Difficulty walking

Are there any new drugs coming out for ALS?

There has been a lot of progress in ALS research and several new treatments are in various stages of development. Amylyx, also known as AMX0035, submitted a New Drug Application (NDA) to the FDA in November 2021. The FDA gave September 29, 2022, as the target date by which the FDA intends to complete its review and decide whether or not to approve the drug.

One other treatment in the pipeline is Tofersen. Researchers have completed a phase 3 clinical trial using this medication in people with ALS with SOD1 mutations.

What is the life expectancy of someone with ALS?

It is estimated that 80% of people with ALS die within 2 to 5 years of being diagnosed, while about 10% live for more than 10 years.

Related resources for ALS

• ALS Statistics. John Hopkins Medicine
• ALS Overview. Muscular Dystrophy Association
• ALS symptoms and causes. Cleveland Clinic
• ALS Overview. Mayo Clinic
• What is ALS? ALS Association
• ALS Fact sheet. National Institute of Neurological Disorders and Stroke
• Smoking and the risk of ALS. Jama Network
• Emerging Drugs. ALS.org