ALS treatment options

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 such as muscle weakness and wasting.

Approved ALS treatments

There are three disease-modifying therapies approved by the U.S. Food and Drug Administration (FDA) to treat ALS, with some also approved by regulators in other countries. These include:

• riluzole
• edaravone
• Qalsody (tofersen)

In clinical trials, approved ALS treatments have shown benefits that include slowing disease progression, allowing patients to maintain functional abilities for longer. They also may extend survival.

Riluzole formulations

In 1995, riluzole became the first approved ALS treatment in the U.S. The therapy is thought to work by blocking the release of glutamate, a nerve signaling chemical that can overstimulate and damage nerve cells when present at high levels. Riluzole is available in the form of:

• oral tablets: only generic versions are now available; previously sold under the brand name Rilutek
• oral liquid suspension: sold as Tiglutik (Teglutik in Europe) with generics also available; can be taken by mouth or given through a feeding tube

Riluzole is generally taken twice a day. The ALS medication is generally well-tolerated, but patients should be monitored for signs of liver damage during treatment.

Edaravone formulations

Edaravone was approved by the FDA in 2017, marking the first new ALS therapy approval 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. Edaravone is available in the form of:

• into-the-vein (intravenous) infusions: only generic versions are now available; previously sold under the brand name Radicava
• oral liquid suspension: sold as Radicava ORS; can be taken by mouth or through a feeding tube

Both formulations are administered in monthlong cycles, with treatment occurring daily for the first 10 to 14 days of each cycle. The intravenous formulation is administered by a healthcare provider, while the oral version can be self-administered.

The medication carries a warning that it may cause allergic reactions, so patients will be monitored for signs of a reaction.

Qalsody

Qalsody (tofersen) is an RNA-based therapy that was approved under the FDA’s accelerated approval pathway in 2023 for the treatment of adults with ALS associated with mutations in the SOD1 gene. It reduces levels of toxic SOD1, a protein that builds up and damages nerve cells in this subset of patients.

The medication is administered by an injection directly into the spinal canal, called an intrathecal injection, with once-monthly maintenance doses. Intrathecal administration is a relatively invasive procedure that must be performed by a trained healthcare provider.

Patients will be monitored for possible complications that could arise from the injection procedure, including inflammation affecting the brain, spinal cord, and optic nerve.

Treating ALS symptoms

In addition to therapies that target the underlying causes of the disease, treatment for ALS may involve medications to help manage specific symptoms. These medications are a key component of palliative care, a branch of medicine focused on symptom relief and quality of life at any stage of a serious illness.

In general, people living with ALS are offered the same standard treatments that would be used for these symptoms in people without ALS. Examples of symptoms and treatments include:

• Muscle spasticity: Oral muscle relaxants (e.g. baclofen, tizanidine), anti-seizure medications (gabapentin), or cannabis; injections of botulinum toxin products (e.g. Dysport) if other treatments don’t provide relief.
• Muscle cramps: Sodium channel blockers (e.g. ranolazine, quinine sulfate), baclofen, or gabapentin.
• Excess saliva: Anticholinergic medications that block saliva-stimulating nerve signals, such as scopolamine, amitriptyline, or atropine; botulinum toxin injections into the salivary glands when other treatments don’t provide relief.
• Pseudobulbar affect: Nuedexta (dextromethorphan and quinidine) is the only medication that is FDA-approved to treat the sudden bouts of laughing and crying that characterize this symptom. Some antidepressants also may be used off-label.
• Depression or anxiety: Standard antidepressant or anti-anxiety medications.
• Thick mucus in the airways: Medications to thin mucus or widen the airways.

Treating ALS symptoms requires an individualized approach — other symptoms will be managed according to standard practice as they arise. Fatigue and sleep problems in ALS are often multifactorial and influenced by other symptoms. Managing these underlying factors is a core part of treatment.

Supportive therapies for ALS

In addition to medication, most people with ALS benefit from non-drug interventions that help maximize comfort and quality of life. These supportive therapies for ALS may include:

• physical therapy, which works to maintain physical function
• occupational therapy, to find strategies for navigating daily life with a chronic condition
• speech therapy, to help with speech and swallowing difficulties
• respiratory therapy, with an aim toward maintaining lung function
• psychotherapy and counseling, to help navigate emotional complexities and mental health challenges

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 mechanical ventilation devices to support breathing function and mucus clearance
• feeding tubes to ensure adequate nutrition when swallowing is a challenge

End-of-life care

When a person with ALS is in the advanced stages of the disease, they may be referred for hospice care. Like palliative care, hospice care is focused on improving comfort, but it is specifically intended for people expected to be in the final six months of life.

At this stage, disease-modifying therapies or other intensive interventions may be stopped, depending on the wishes of the person with ALS and their care partners. Supportive care to manage pain and other symptoms, along with emotional or spiritual counseling, are pillars of managing ALS during this time.

What type of doctors treat ALS?

While patients may first consult with their primary care doctor about signs and symptoms of ALS, most will be referred to a neurologist — a doctor who specializes in nervous system disorders — for diagnosis and treatment. Getting a second opinion can help patients feel more confident in their diagnosis and care plan.

While treatment is usually led by a neurologist, a multidisciplinary care team, including doctors, nurses, and therapists, is key to optimal ALS care. Healthcare professionals who may be members of an ALS care team include:

• pulmonologists (lung specialists)
• physical, occupational, and speech therapists
• dietitians or nutritionists
• mental health professionals or social workers
• palliative care specialists

A hospice team often includes family members, physicians, nurses, home health aides, social workers, clergy members, and therapists.