The Power of Clinical Trials in ALS: Past, Present, and Future

Joumana Fawaz-Baroody, RN, BSN
Vice President of Care Services, ALS United Greater Chicago

For decades, people living with ALS and their families have courageously contributed to the fight against this devastating disease. Thanks to their participation in clinical trials, every new discovery and treatment option has been made possible. While ALS remains without a cure, the history of clinical research proves that progress is real—and accelerating.

Clinical trials are the bridge between laboratory science and real-world treatments. They have led to the approval of medications that help manage symptoms, extend survival, and improve quality of life. More importantly, they continue to fuel hope for breakthroughs that could one day stop or even reverse the course of ALS.

A Legacy of Progress: How Clinical Trials Have Moved ALS Research Forward

Clinical trials have been essential in advancing our understanding of ALS. Early breakthroughs led to therapies like riluzole, the first FDA-approved drug to modestly extend survival, and more recently, edaravone (Radicava), which can slow functional decline in some patients.

These successes were only possible because of the brave individuals who chose to participate in research. Every person who joins a trial helps answer critical questions:

• Is this treatment safe?
• Does it improve symptoms or slow progression?
• How does it compare to existing options?

Even trials that don’t result in approved therapies provide essential knowledge for future studies. Every trial matters. Each step builds toward better treatments and, ultimately, a cure.

Observational & Natural History Studies: Quiet but Critical

Not all studies test new treatments. Observational and natural history studies follow people with ALS over time, without changing their current care.

These studies help researchers:

• Understand how ALS progresses
• Improve how treatments are measured
• Discover biomarkers for earlier diagnosis and targeted therapies

These studies are typically low-risk and offer a meaningful way to contribute to ALS research, even if you are not ready or eligible for clinical trials.

Understanding the Journey: How Clinical Trials Work

Preclinical Research: Where It Begins

• Before human testing, scientists study new therapies in the lab and with animal models to ensure safety and potential effectiveness.

Phase 1: Testing Safety

• Participants: 20–100 people (sometimes including individuals living with ALS)
• Goal: Ensure the treatment is safe and determine the correct dosage
• Focus: Monitor side effects and how the body processes the treatment

Phase 2: Testing Effectiveness

• Participants: Several hundred individuals living with ALS
• Goal: Assess if the treatment slows progression or improves symptoms
• Focus: Continue safety monitoring and evaluate effectiveness

Phase 3: Confirming Results

• Participants: Hundreds to thousands
• Goal: Compare to standard care or placebo to confirm benefits
• Outcome: Data used for FDA or other regulatory approval

Phase 4: Monitoring Long-Term Impact (Post-Marketing Studies)

• Participants: Thousands of people using the treatment in real-world settings
• Goal: Continue monitoring the treatment’s long-term safety and effectiveness after FDA approval and broad use for ALS
• Focus: Detect rare or long-term side effects, gather insights from everyday clinical use, and inform future treatment guidelines

Why Your Participation Matters More Than Ever

The ALS research landscape has never been more promising. Innovative designs like the HEALEY ALS Platform Trial test multiple therapies at once, reducing placebo use and speeding up discoveries.
When you participate in a trial or study, you:

• Gain potential early access to new treatments
• Advance global ALS research
• Leave a legacy of hope for future generations

Your care team can help you understand risks, benefits, and whether participation is right for you.

The Evolution of Research and Smarter Clinical Trial Designs

Today’s clinical trials are smarter, faster, and more inclusive than ever before. Researchers have learned valuable lessons from past studies, leading to innovations that make trials more efficient and patient-centered.

New approaches like platform trials allow multiple therapies to be tested under a single study framework, reducing the time it takes to bring promising treatments to people with ALS. Adaptive trial designs enable researchers to adjust the study as results emerge, meaning fewer participants receive placebos and more gain access to potential therapies sooner.

Additionally, there is a growing focus on biomarkers—biological indicators of disease progression—that help measure whether a treatment is working more accurately and earlier in the trial process.

These advancements are accelerating discoveries and giving people living with ALS more opportunities to participate in research that truly makes a difference.

Finding Clinical Trials and Studies

Explore these trusted resources to find trials and studies that match your needs:

• ClinicalTrials.gov: clinicaltrials.gov
• NEALS Consortium: neals.org

For natural history studies, explore:

• Answer ALS: answerals.org
• CDC National ALS Registry: cdc.gov/als

Your ALS Clinic Team or Care Services Coordinator is here to help — from answering your questions to connecting you with local studies at UIC, Rush, and UChicago, and helping you explore clinical trials and research studies that align with your needs and journey.

Together, we honor the past, embrace the present, and build a future without ALS.