Amyotrophic lateral sclerosis treatment options

Last updated April 11, 2024, by Marisa Wexler, MS
Fact-checked by Ines Martins, PhD

A number of treatment options exist for amyotrophic lateral sclerosis (ALS), a disease marked by the progressive loss of motor neurons — specialized nerve cells that control muscle movements — and symptoms like muscle weakness and wasting.

Also known as Lou Gehrig’s disease, ALS is fatal, usually leading to death within five years of symptom onset. While no available therapy can cure the disease or reverse motor neuron death after it occurs, there are several treatments that have been proven to slow disease progression and extend survival in ALS patients.

There also are a number of symptomatic and supportive ALS treatments that can minimize disease complications and ease discomfort, as well as help patients to maintain functionality for as long as possible.

Approved ALS treatments

There are three medications approved by the U.S. Food and Drug Administration (FDA) to treat ALS. Some formulations also are approved by regulators in other countries.

In clinical trials, these medicines have been proven to slow the progression of ALS, allowing patients to maintain functional abilities for longer. They also improved ALS survival rates. For some of these therapies, multiple formulations are available.

Riluzole formulations

Riluzole was the first treatment to be approved for ALS by the FDA in 1995. The therapy is thought to work by reducing molecular signaling, which can overstimulate and damage nerve cells.

The original formulation of riluzole is an oral tablet marketed under the brand name Rilutek by Covis Pharma. Generic versions of Rilutek have been available since the early 2000s.

Two other formulations of riluzole have recently become available:

• Tiglutik (Teglutik in Europe) is a liquid suspension formulation of riluzole that can be taken by mouth or given through a feeding tube.
• Exservan also is an oral formulation of riluzole, administered in a film that dissolves on top of the tongue.

Edaravone formulations

Edaravone was approved by the FDA in 2017, marking the first new approval for an ALS therapy in more than two decades. The medicine works by reducing oxidative stress, a type of cell damage thought to drive nerve cell death in ALS.

The first approved formulation of edaravone, which is administered via infusion into the bloodstream (intravenously), is marketed under the brand name Radicava by Mitsubishi Tanabe Pharma America. It was approved in Canada in 2018.

An equivalent liquid suspension formulation that can be taken by mouth or feeding tube, marketed as Radicava ORS, was approved by the FDA and by Canadian regulators in 2022.

Relyvrio

Relyvrio (sodium phenylbutyrate and taurursodiol) is a fixed-dose combination of two compounds that work together to reduce cellular stress pathways and promote nerve cell survival. Developed by Amylyx Pharmaceuticals, the therapy was approved in the U.S. to treat ALS in late 2022, but was removed from the market in April 2024 following negative results in a confirmatory Phase 3 clinical trial. Still, patients who were receiving the medication prior to its discontinuation could, in consultation with their treating physician, transition to a free drug program.

Qalsody

Qalsody (tofersen) is an RNA-based therapy designed to reduce the amount of toxic SOD1, a protein that builds up and forms toxic clumps in some ALS patients. Marketed by Biogen, the injection therapy was approved in the U.S. in April 2023 to treat people with ALS associated with mutations in the SOD1 gene.

Treating ALS symptoms

In addition to treatments that target the underlying causes of the disease, a number of therapies are available to help manage specific symptoms of ALS. The disease’s manifestations can include: 

• muscular symptoms like weakness, wasting, and tremor-like movements due to muscle exhaustion
• muscle stiffness (spasticity) and cramps
• difficulty speaking and swallowing
• trouble breathing
• constipation or other digestive complaints
• cognitive and behavioral abnormalities
• mental health issues.

Despite the variety of symptoms experienced by patients, there are limited data focused on treatments that may help ease these complaints specifically in people living with ALS. For most symptoms, ALS patients receive generally the same standard management offered to individuals with other medical conditions.

Treatments for spasms and cramps

Treatment of spasticity and cramps generally involves medicines that prompt muscles to relax. A number of therapies are approved to treat spasticity including:

• baclofen
• tizanidine
• formulations of botulinum toxin, such as Dysport
• dantrolene.

Other approved medicines are sometimes used off-label, meaning they are not specifically approved for this indication, in the management of ALS-related spasticity. These medications, which include the anti-seizure medications gabapentin and clonazepam, have been shown to help manage this symptom in people with ALS.

Some recent research has suggested cannabis, also known as medical marijuana, may help to ease spasticity in ALS. However, the available data is mostly from anecdotal reports.

Treatments for excess saliva production

Many people with ALS develop weakness in the muscles around the mouth and throat which can make it difficult to swallow. As a result, saliva can pool in the mouth, which may increase the risk of choking or getting fluid in the lungs.

Medications used to manage saliva production in ALS are mainly anticholinergic therapies, which block signals from nerves that promote saliva production in the salivary glands. Examples of these medicines that are commonly used in ALS include:

• scopolamine
• amitriptyline
• carbocisteine
• atropine.

Botulinum toxin injections into the salivary glands or radiation of these glands also may be used to help manage excess saliva production in ALS, particularly in instances where anticholinergic therapies are insufficient.

Treatment for pseudobulbar affect

Pseudobulbar affect, which occurs in some people with ALS and is often referred to as PBA, is characterized by sudden bouts of laughing or crying that are disconnected from the individual’s emotional state.

Nuedexta (dextromethorphan and quinidine) is the only medication that is FDA-approved to treat pseudobulbar affect. Specific antidepressants also may be used off-label for managing this symptom.

Treatment for other symptoms

Due to the significant impact of ALS on patients’ lives, anxiety and depression are common among people with this disease. These patients may benefit from antidepressants like amitriptyline and anti-anxiety medications, such as diazepam or lorazepam. Antidepressant drugs also can be helpful for those experiencing pain and fatigue.

Some people with ALS accumulate mucus in their lungs that’s oftentimes very thick and sticky (viscous). These patients may not be able to properly clear such secretions due to weakness in the muscles involved in coughing. Individuals who experience this complication can benefit from medications that help thin mucus (mucolytics) or others that widen the airways (bronchodilators). Drinking enough liquids also may make the mucus less viscous and improve airway clearance.

Stem cell treatment for ALS

Stem cells are undifferentiated cells in the body that are capable of self-renewing and maturing into other types of cells. They also can secrete signaling molecules that provide a supportive and protective environment to nearby cells.

Scientists have explored many treatment strategies for ALS that utilize stem cells, though so far none has been approved by the FDA.

Such ALS stem cell treatment is not the same thing as stem cell transplants, which are procedures used to manage certain cancers and autoimmune diseases. Stem cell transplants work by replacing the blood stem cells that live in the bone marrow.

NurOwn

NurOwn (debamestrocel) is an experimental treatment that uses mesenchymal stem cells (MSCs) engineered to secrete signaling molecules — called neurotrophic factors — that promote nerve health. These cells are injected into the spinal canal with the aim of improving motor neuron survival.

A Phase 3 clinical trial (NCT03280056), sponsored by NurOwn’s developer BrainStorm Cell Therapeutics, failed to meet its main goal of demonstrating that the treatment could significantly slow ALS disease progression. But analyses done after the trial was completed indicated a treatment benefit for patients with less severe ALS at the trial’s start.

In late 2022, the FDA declined to consider an application from BrainStorm that sought approval of NurOwn, on the basis that currently available data are insufficient to support an approval. The company still decided to proceed with the filling over the FDA’s objection, but it later withdrew its application after an FDA advisory committee echoed the FDA’s earlier sentiment. A Phase 3 clinical trial is now being planned to investigate the cell-based therapy in people with early ALS.

Other stem cell-based therapies

Several other stem cell-based therapies are in development for ALS.

• NeuroNata-R (lenzumestrocel) is an MSC-based therapy that’s conditionally approved for ALS in South Korea. It is now being tested in a Phase 3 clinical trial (NCT04745299) that may support a potential approval in the U.S.
• CNS10-NPC-GDNF uses neural progenitor cells, or stem cells that can grow into nerve cells, engineered to secrete a signaling molecule that promotes nerve health. The therapy is in early clinical testing.
• AstroRx contains nervous system support cells called astrocytes engineered from human embryonic stem cells, which are found in embryos during prenatal development. Findings from a small Phase 1/2 study (NCT03482050) suggested this experimental therapy may help slow ALS progression, and further testing is in the works.

Supportive therapies for ALS

In addition to medication, most people with ALS benefit from supportive forms of therapy, which can help to maximize comfort and quality of life. These can include:

• physical therapy, also called physiotherapy, which works to maintain physical function
• occupational therapy, to find strategies for navigating daily life with a chronic condition
• speech therapy, which can help with speech problems as well as difficulty swallowing
• respiratory therapy, with an aim toward maintaining lung function
• psychotherapy and counseling, to help navigate emotional complexities and mental health challenges, including psuedobulbar affect.

Lifestyle changes, such as adjusting a person’s diet to include foods that are easier to chew and swallow, also may be helpful for people with ALS.

As the disease progresses, many patients rely on adaptive equipment such as:

• wheelchairs or other mobility aids to make it easier to get around
• respirators and other devices to support breathing function and mucus clearance
• feeding tubes to ensure adequate nutrition when swallowing is a challenge.

What type of doctors treat ALS?

ALS is a disease of the nervous system. Patients may first consult with their primary care doctor about signs and symptoms of ALS, but most will be referred to a neurologist — a doctor who specializes in nervous system disorders — for diagnosis and treatment.

Diagnosing ALS usually takes several months of evaluation by a specialist, who will request tests checking for signs of ALS-like motor neuron degeneration and assessments to exclude other potential causes of symptoms.

While treatment of ALS is usually also led by a neurologist, optimal care for someone with the disorder typically involves clinicians across multiple medical specialties. This includes doctors, nurses, and therapists, all of whom can work together to provide the best possible care.

ALS News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.