Amyotrophic Lateral Sclerosis (ALS) Treatment Options

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive motor neuron disease. ALS affects the nerve cells that help control voluntary muscle movement. As the disease progresses, patients may experience symptoms such as:

• Muscle twitching or cramps
• Worsening muscle weakness
• Mobility loss
• Speech changes
• Swallowing difficulty
• Breathing problems

There is currently no cure for ALS. Instead, standard treatment focuses on slowing progression where possible, managing symptoms and supporting quality of life through a multidisciplinary treatment plan.

Medications, like other ALS treatments, do not cure ALS or reverse motor neuron damage. Instead, modern treatment plans may combine medications that slow disease progression with drugs that relieve specific symptoms.

The exact plan depends on physician review, including the patient’s diagnosis, disease stage, genetic subtype, symptoms, overall health and local medication availability.

Medication availability can change over time. For example, Relyvrio/Albrioza was voluntarily discontinued in the U.S. and Canada in 2024 after the Phase 3 PHOENIX trial did not confirm benefit. Patients should ask their neurologist which medications are currently available and appropriate in their country.

 

 

Riluzole (Rilutek®, Exservan® and Tiglutik®)

After receiving FDA approval in 1995 and EU marketing authorization in 1996, riluzole became one of the main medications used in ALS treatment. It is available in different oral forms, including tablets, oral film, and oral suspension, depending on the specific product and country.

Riluzole has shown the potential to modestly slow ALS disease progression and may help extend life or the time to mechanical ventilation.

Common side effects may include:

• Dizziness
• Gastrointestinal side effects
• Liver problems

As a result, patients typically require physician monitoring while taking the medication.

Tofersen (Qalsody®)

Sold under the brand name Qalsody®, tofersen was granted accelerated FDA approval in 2023 and EU marketing authorization under exceptional circumstances in 2024 to treat ALS associated with SOD1 mutations. As a result, it is not relevant for most ALS patients, and confirmation of an SOD1 mutation is needed to determine eligibility.

Tofersen should be discussed with a neurologist familiar with genetic ALS treatment.

Edaravone (Approved in Some Countries, Not Authorized in the EU)

Edaravone is an antioxidant treatment that received FDA approval for ALS in 2017. However, it is not currently authorized as a standard ALS treatment in the EU.

Edaravone can be delivered through a vein in the arm or taken orally, depending on the specific product and country. It is intended to reduce oxidative stress, a process being studied in ALS-related motor neuron damage. In selected ALS patients, it may help slow functional decline.

Potential benefits for lifespan are currently less clear, while possible side effects include:

• Bruising
• Headache
• Problems walking

Suitability depends on physician review, disease stage, overall health, local medication availability, and treatment goals.

Symptom Management Medications

Alongside medications intended to slow ALS progression, physicians may also recommend symptom-focused medications to manage complications and improve comfort.

These medications may help with ALS-related symptoms such as:

• Muscle cramps
• Spasticity
• Pain
• Excess saliva
• Sleep problems
• Constipation
• Depression or anxiety
• Pseudobulbar affect

While symptom management medications can help improve quality of life, they do not stop ALS progression or reverse established motor neuron damage.

Treatment often involves more than medication alone. Supportive therapies do not cure ALS or modify the disease itself, but they can help people with ALS preserve daily function, independence, and quality of life.

The sections below explain how different supportive therapies may help ALS patients adapt as their symptoms change.

 

 

Physical Therapy

Physical therapy can help ALS patients adapt to their symptoms and preserve function for as long as possible.

Some potential benefits of physical therapy include:

• Maintaining mobility, flexibility, posture, comfort, and remaining muscle strength
• Helping manage pain and discomfort
• Supporting cardiovascular fitness in appropriate patients

Because ALS-related weakness and fatigue can worsen function, physical therapy should always be adapted to the patient’s disease stage and fatigue level, with activity plans developed in coordination with a physical therapist.

Occupational Therapy

Occupational therapy can help ALS patients maintain independence with daily activities as hand, arm, and mobility function changes.

An occupational therapist may recommend practical supports such as adaptive equipment, assistive technology and home safety adjustments to make daily routines safer and reduce caregiver strain.

Speech Therapy and Communication Support

Speech therapy can help ALS patients maintain communication as speech, voice, and swallowing symptoms progress.

A speech-language pathologist can help with speech clarity, swallowing assessment and communication planning. In many cases, this may include voice banking or technology such as speech-generating devices, text-to-speech tools and eye-tracking systems.

Early planning can help patients preserve communication options before speech becomes severely affected.

Nutritional Support

As symptoms progress, swallowing difficulties can make it harder to eat, contributing to weight loss in ALS patients. Proper nutritional support from a dietitian can help patients maintain weight, which may be associated with better outcomes.

Nutritional support from a dietitian may include:

• Higher-calorie meals
• Texture-modified foods
• Thickened liquids
• Meal timing adjustments
• Supplements, when appropriate

When swallowing becomes too difficult, nutritional support may also include the use of feeding tubes. These can help reduce risks associated with difficult swallowing while supporting hydration, nutrition, and medication intake.

The decision to use a feeding tube should be made collaboratively with the patient, family, neurologist, dietitian, and speech-language pathologist.

Breathing Support

ALS can weaken the muscles involved in breathing, making respiratory support important as symptoms progress.

Respiratory support may include:

• Noninvasive ventilation, such as BiPAP
• Assisted coughing devices
• Suction devices to help clear secretions
• Tracheostomy ventilation in selected advanced cases

In some cases, tracheostomy ventilation may be considered when noninvasive ventilation is no longer enough or when patients choose long-term invasive ventilation.

A tracheostomy is a major decision involving quality of life, planning and caregiver support. Patients and families should discuss breathing support early with their ALS clinical team to understand the medical, practical, emotional and home-care implications.

Psychological, Social, and Palliative Support

ALS can place emotional, financial and caregiving strain on patients and families. Psychological and social support may include counselling, social work, caregiver support and advance care planning.

Palliative care does not mean stopping ALS treatment. Instead, it can help with symptom management, decision-making, emotional support and quality of life.

Social support can also help patients access equipment, home care, benefits, transportation and community resources.

Beyond current medications and supportive therapies, ALS research continues to evolve as scientists learn more about motor neuron degeneration, genetic subtypes of ALS, disease biomarkers, and the biological environment surrounding affected nerve cells.

Emerging areas of ALS research include investigational drugs, gene-targeted approaches, biomarker research, precision medicine, cell-based therapies,stem cell secretome and extracellular vesicle research, and regenerative medicine research.

Investigational therapies are usually studied through clinical trials before they become part of standard ALS care. Eligibility varies by study, and benefit is not guaranteed.

Experimental ALS therapies are not cures and should never replace standard neurological care, respiratory support, nutritional support, or multidisciplinary ALS management.

Investigational Drug Trials

Investigational drug trials study potential ALS medications that are not yet established as standard care. These trials may evaluate whether a therapy can slow functional decline, affect disease biomarkers, improve survival-related outcomes, or support better symptom control.

Some examples of ALS drug trials include:

• HEALEY ALS Platform Trial: Began in 2020 and has studied multiple investigational ALS treatments through a shared platform trial design. More than 1,300 people have participated in the platform trial.
• PREVAiLS Phase 3 trial: Began enrolling participants in 2026 to study pridopidine, an investigational drug being evaluated for ALS progression.

Participation in an ALS clinical trial depends on study criteria, disease stage, diagnosis, location, prior treatments, breathing function, and overall health. Eligibility varies, and benefit is not guaranteed.

Gene-Targeted and Precision Medicine Research

ALS research is also moving toward more individualized treatment approaches based on genetics, biomarkers, and disease subtype.

Gene-targeted and precision medicine research may focus on:

• Specific ALS-related mutations
• Biological pathways involved in disease progression
• Measurable disease markers
• Treatment approaches for defined patient subgroups

Tofersen is one example of howgenetic testing can affect ALS treatment decisions, since it is only relevant for patients with confirmed SOD1-associated ALS. Other gene-targeted and precision medicine approaches remain under investigation.

These approaches are still highly individualized and should be reviewed with a neurologist or ALS specialist.

Stem Cell and Regenerative Medicine Research

Stem cell-based therapy is another emerging area being explored for ALS. The current rationale is not based on replacing lost motor neurons or curing the disease. Instead, research has focused on whether these approaches may influence the biological environment around affected nerve cells.

Rather than claiming to rebuild damaged motor neurons, researchers are exploring possible effects on:

• Neuroprotection
• Inflammatory signalling
• Cellular stress
• Paracrine signalling
• Extracellular vesicle and secretome activity

 

 

At ANOVA-IRM,experimental regenerative medicine for ALS is reviewed through individualized medical assessment and case-by-case discussion. This may include stem cell secretome or extracellular vesicle/exosome-related approaches when medically appropriate.

Clinical use of stem cell-based therapy for ALS remains experimental. Suitability depends on diagnosis, disease stage, breathing and swallowing function, overall health, travel suitability, treatment goals, and benefit-risk assessment. Stem cell-based therapy cannot guarantee ALS improvement, disease stabilization, motor recovery, or survival extension.

Stem Cell Therapy Contraindications

Experimental regenerative therapy is not suitable for every ALS patient. ANOVA-IRM does not treat patients when certain risk factors are present, including:

• Active cancer within the last two years
• Being under the legal age for treatment
• Pregnancy or breastfeeding
• Ventilator dependence or inability to breathe independently
• Difficulty breathing while lying down
• Severe swallowing difficulty
• Severe or uncontrolled psychiatric conditions
• Active infectious disease, including hepatitis, HIV, syphilis, or other infections

If you or a family member has ALS, you can contact ANOVA-IRM to ask whether an individual medical review may be appropriate.

Before any experimental option is considered, ANOVA reviews each case individually. This may include diagnosis, disease stage, progression rate, breathing and swallowing function, current medications, overall health, travel suitability, and treatment goals.

Regenerative therapy cannot replace standard ALS care. Patients should continue working with their neurologist or ALS care team while exploring additional options.

Contact ANOVA to request an individual medical review.