ALS Treatments and Trials

Current FDA-approved ALS treatments

You should discuss any treatment with your physician to determine if it is right for you or your loved one and to weigh the potential effectiveness or side effects.

Qalsody (Tofersen): This treatment is available for adults who have ALS due to a mutation in the superoxide dismutase 1 (SOD1) gene. Qalsody has shown to reduce neurofilament, a marker of neurodegeneration, resulting in slowed ALS progression. Qalsody is administered through recurring spinal injections by a healthcare professional. Learn more about Qalsody and talk to your doctor about whether this is right for you.

Relyvrio: This therapy for adults living with ALS can be administered orally or through a feeding tube. This drug has been shown to slow the rate of progression. Learn more about Relyvrio and talk to your doctor about whether this is right for you.  

Riluzole: Available in a pill form as Rilutek and in a liquid form as Tiglutik, this drug acts to slow the progression of ALS symptoms and prolong survival by inhibiting the release of the neurotransmitter glutamate. Neurotransmitters are molecules that nerve cells use to communicate, and excess levels of glutamate are thought to damage nerve cells in many instances in  ALS. Learn more about Riluzole or Tiglutik and talk to your doctor about whether this is right for you.

Radicava: Also known as Edaravone, there are two versions of this. Radicava IV is administered through a series of recurring intravenous infusions (meaning, into your veins through an IV), while Radicava ORS can be administered orally or through a feeding tube. This drug has been shown to slow the loss of physical function. Learn more about Radicava and talk to your doctor about whether this is right for you.

Nuedexta: Available as a capsule, this drug treats pseudobulbar affect, which is the occurrence of unusual laughing or crying in people with ALS or other neurodegenerative diseases. Learn more about Nuedexta and talk to your doctor about whether this is appropriate for you.

Clinical trials

Clinical trials are studies in which researchers investigate whether a new treatment or product works to treat or cure a health condition. There are an increasing number of ongoing clinical trials in ALS that have the potential to slow down disease progression and/or manage ALS symptoms. 

You may be considering whether you or your loved one should participate in an ALS clinical trial, and may have questions. Here’s your step-by-step guide to learning about and accessing clinical trials:

Step 1: Check out these frequently asked questions and answers, created by people living with ALS and caregivers, on what clinical trials are, why to consider participating, what the enrollment process is like and more. 

Step 2: We encourage you to explore the potential treatments and supplements that are currently being tested through clinical trials on ALS Signal: The Clinical Research Dashboard, which was designed for and by people living with ALS and caregivers. Make a list of the ones you want to learn more about. Pro tip: Contact an I AM ALS Support Specialist to help you find clinical trials. 

Step 3: Consider asking these Questions About A Specific Clinical Trial at your next visit with your care team to learn more about the trials on your list. Not sure about which trials you want to learn more about? Here are a few Questions to Ask About Clinical Trials that will help you with this. Pro tip: Don’t wait to talk about trials until your next in-person clinic visit. Email or call your care team to explore this as soon as possible!

Step 4: Ask your care team for your medical records and essential information. Once you have this, email the clinical trial coordinator to start the trial screening process. We encourage you to contact the I AM ALS Support Team for assistance if you experience challenges in getting connected with a coordinator.


Dr. Richard Bedlack and more than 120 colleagues from 11 countries have created a website called ALSUntangled through which they have assessed the effectiveness and safety of dozens of supplements. Explore their findings by clicking here.

Alternative pathways to accessing investigational therapies

Expanded access: Also known as compassionate use, this is a pathway established by the FDA for people with serious and/or life-threatening diseases to gain access to investigational medical products for treatment outside of a traditional clinical trial.  Expanded access is only available to people who do not meet the eligibility criteria for ongoing clinical trials, and access through this pathway must be requested by a licensed physician. Learn more about expanded access here and talk with your doctor about whether it might be a good option for you or your loved one. Please note that not all clinicians are familiar with this process. Additional resources for clinicians regarding how to request access can be found on the FDA’s website and here. As always, it is also important to discuss all possible risks of an investigational medical product. 

Right to Try: According to the FDA: “This law is another way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain unapproved treatments.” Learn more about Right to Try here and discuss with your doctor about whether this avenue is an option for you. Please note that the ultimate decision to provide the investigational therapy lies with the company that produces it.

We're here to help with the challenges of ALS

Connect with an ALS Support Specialist today

Have questions? Call us at 866.942.6257 between 9 a.m. - 5 p.m. or email us

Back to Top