Is There Any Treatment for ALS? Approved Options

There is no cure for ALS, but several treatments can slow the disease, manage symptoms, and meaningfully extend survival. The landscape has changed in recent years, with FDA-approved medications now joined by breathing support, nutritional strategies, and coordinated team-based care that together can add months or even years of life.

Riluzole: The First-Line Medication

Riluzole has been the standard ALS medication since 1995. It works by reducing the activity of a chemical messenger called glutamate, which in excess can damage motor neurons. The survival benefit is modest: roughly 2 to 3 months on average, translating to about a 9% increase in one-year survival. That may sound small, but riluzole remains the most widely prescribed ALS drug because it’s taken as a simple oral tablet, has a well-understood safety profile, and offers a consistent, proven benefit across most patients regardless of disease stage.

Edaravone: Slowing Functional Decline

Edaravone (sold as Radicava) was approved in 2017 and works differently from riluzole. It acts as an antioxidant, reducing a type of cellular damage that accelerates motor neuron death. In clinical trials, patients on edaravone lost about 5 points on a 48-point functional scale over six months, compared to 7.5 points in the placebo group. That 2.49-point difference was statistically significant and represents a roughly 33% reduction in the rate of functional decline.

Edaravone was originally given as an intravenous infusion in treatment cycles, but an oral formulation is now available, which makes it far more practical for daily use. The drug appears to work best in people who are still relatively early in the disease and whose breathing function hasn’t declined severely.

Tofersen: A Treatment for Genetic ALS

About 2% of ALS cases are caused by mutations in a gene called SOD1, and in 2023 the FDA approved tofersen (Qalsody) specifically for these patients. This is a fundamentally different kind of therapy. Instead of broadly protecting neurons, tofersen targets the genetic instructions that produce the harmful SOD1 protein and reduces its production.

The drug is given as a spinal injection: three initial doses two weeks apart, then a maintenance dose every 28 days. In a 28-week trial of 108 patients with confirmed SOD1 mutations, those receiving tofersen showed significant reductions in a blood marker of nerve damage called neurofilament light chain, which tracks closely with disease progression. The most common side effects include pain, fatigue, joint pain, and muscle pain. Tofersen was approved under the FDA’s accelerated approval pathway, meaning its continued approval depends on confirmatory trials showing clinical benefit.

Genetic testing is necessary to determine whether someone carries a SOD1 mutation and qualifies for this treatment. If you have a family history of ALS, this is worth discussing early.

Relyvrio: No Longer Available

You may have heard of Relyvrio, a combination drug that received FDA approval in 2022. It has since been withdrawn. A large Phase 3 confirmatory trial called PHOENIX failed to meet its primary and secondary endpoints, meaning the drug did not demonstrate a meaningful benefit in the larger study. The manufacturer voluntarily requested withdrawal, and the FDA formally pulled its approval as of August 29, 2025. Relyvrio can no longer be legally distributed.

Breathing Support Extends Survival Significantly

Noninvasive ventilation, typically delivered through a BiPAP machine, is one of the most impactful interventions in ALS care. It doesn’t treat the disease itself, but it compensates for weakening respiratory muscles, which is the primary cause of death in ALS.

The timing of when you start matters enormously. Research published in Frontiers in Neurology found that patients who began BiPAP early (when their lung capacity was still at 80% or above) survived a median of 25.4 months, compared to 20.3 months for those who started at the more traditional threshold of below 50% lung capacity. That’s a 25% increase in survival just from starting sooner.

The difference becomes even more dramatic with an optimized protocol. Patients who started BiPAP early, used it more than 8 hours per day, and added daily cough-assist therapy had a median survival of 30.8 months, double the 15.4 months seen with a standard protocol. Overall, BiPAP users on the optimized approach gained an average of 17.1 additional months compared to non-users. Few pharmaceutical treatments for ALS come close to this magnitude of benefit.

Nutritional Support and Feeding Tubes

Maintaining body weight is directly linked to survival in ALS. As swallowing muscles weaken, people with ALS are at risk of malnutrition and aspiration (food entering the lungs). A gastrostomy tube, placed through the abdomen directly into the stomach, bypasses the swallowing problem entirely and allows reliable nutrition and hydration.

Current guidelines recommend placing the tube while lung capacity is still at or above 50% of predicted values. Waiting too long makes the procedure riskier because it typically requires sedation, and weakened breathing muscles tolerate sedation poorly. The tube doesn’t prevent you from eating by mouth if you’re still able to. It serves as a supplement or backup, ensuring you get enough calories even on days when swallowing is difficult.

Managing Specific Symptoms

ALS produces a range of symptoms beyond muscle weakness, and many of them are treatable. Pseudobulbar affect, a condition causing sudden, uncontrollable laughing or crying that doesn’t match how you actually feel, affects a significant proportion of people with ALS. An FDA-approved medication combining dextromethorphan and quinidine (Nuedexta) specifically treats this. It’s taken as one capsule daily for the first week, then one capsule every 12 hours afterward.

Muscle cramps and spasticity can be managed with medications that relax muscle tone. Excessive saliva, which becomes a problem when swallowing weakens, can be reduced with certain medications or, in more severe cases, with targeted treatments to the salivary glands. Pain is common, particularly from joint strain as muscles weaken and limbs fall into awkward positions. Physical therapy, bracing, and standard pain medications all play a role. Depression and anxiety, understandably frequent, respond to conventional treatments including medication and counseling.

Multidisciplinary Clinics Make a Measurable Difference

Where you receive care matters. ALS multidisciplinary clinics bring neurologists, respiratory therapists, physical therapists, occupational therapists, speech-language pathologists, dietitians, and social workers together in coordinated visits. This isn’t just convenient. It consistently produces better survival outcomes than receiving the same services piecemeal through general neurology practices.

A study at a U.S. Veterans Affairs medical center found that patients seen in their multidisciplinary ALS clinic had a median survival of about 24 months from diagnosis, comparable to European multidisciplinary centers and meaningfully better than general neurology clinics. The advantage likely comes from catching problems earlier: starting breathing support before a crisis, placing a feeding tube at the right window, adjusting medications proactively, and coordinating assistive devices as needs change. If a specialized ALS clinic is within reach, it’s one of the most important decisions you can make.

Combining Treatments for Maximum Benefit

No single treatment transforms the ALS trajectory on its own, but the combination of multiple approaches adds up. A typical comprehensive plan might include riluzole and edaravone to slow disease progression, early BiPAP with cough-assist therapy for respiratory support, nutritional management with a feeding tube placed at the right time, symptom-specific medications, and regular visits to a multidisciplinary clinic. Each piece contributes its own margin, and together they can substantially extend both survival and quality of life compared to no treatment at all.

For the small percentage of patients with SOD1 mutations, tofersen adds a genetically targeted layer. Clinical trials for other genetic subtypes and novel drug targets are actively enrolling, and people with ALS are encouraged to explore trial registries like ClinicalTrials.gov to see what options may be available for their specific situation.