ALS Treatments and Trials

FDA-approved treatments

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

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: Administered through a series of recurring intravenous infusions (meaning, into your veins through an IV), 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.

Alternative pathways to accessing drugs in testing

Expanded access: Also known as compassionate use, this is a “potential pathway for a patient(s) with an immediately life-threatening condition or serious disease or condition to gain access to an  investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are  available.” Learn more about expanded access here and talk with your doctor about whether there is an investigational therapy that may deserve your consideration. It’s important to discuss all possible risks of an investigational medical product.  See updates here on how I AM ALS is working to ensure access to ALS investigational therapies is available via expanded access or other mechanisms.

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 treatment lies with the company who produces it.

Clinical trials

Researchers and biopharmaceutical companies (“drug companies”) are currently exploring potential new treatments for ALS through clinical trials. There are more than 30 potential therapies in clinical trial testing.

To learn more about the potential treatments and supplements that are currently being tested take a look at ALS Signal: The Clinical Research Dashboard designed for and by ALS patients and caregivers.

You may consider whether you or your loved one should participate in an ALS clinical trial. For more information on what a clinical trial is, click here for a clinical trial 101 tutorial from the Northeast ALS Consortium.

If you are interested in searching for a clinical trial, you’ll find that many websites offer search engines to help you identify potential trials. We’ve included below some of the leading search engines in the hopes that you’ll find one that is the most intuitive for you:

Consider asking these Questions About Clinical Trials at your next visit with your care team to learn more about your trial options. Have you found a trial you’re interested in learning more about? Here are a few Questions to Ask About a Specific Clinical Trial.


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.

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