ALS Clinical Trials Consortium (NEALS)

<table class="infobox infobox-institution">
<tr>
<th class="infobox-header" colspan="2">ALS Clinical Trials Consortium (NEALS)</th>
</tr>
<tr>
<td class="label">Location</td>
<td>Multiple sites across the United States and internationally</td>
</tr>
<tr>
<td class="label">Type</td>
<td>Clinical Trial Network</td>
</tr>
<tr>
<td class="label">Founded</td>
<td>1997 (NEALS); coordinated consortium formed 2010s</td>
</tr>
<tr>
<td class="label">Funding</td>
<td>National Institute of Neurological Disorders and Stroke (NINDS), ALS Association, MDA</td>
</tr>
<tr>
<td class="label">Website</td>
<td><a href="https://www.neals.org" target="_blank">neals.org</a></td>
</tr>
<tr>
<td class="label">Focus Areas</td>
<td>[Amyotrophic Lateral Sclerosis](/diseases/als), Clinical Trials, Therapeutic Development</td>
</tr>
<tr>
<td class="label">Parent Institution</td>
<td>[NIH - National Institutes of Health](/institutions/nih)</td>
</tr>
</table>

ALS Clinical Trials Consortium (NEALS)

The ALS Clinical Trials Consortium refers to the coordinated network of clinical trial sites and researchers dedicated to accelerating ALS therapeutic development. In the United States, this function is primarily served by NEALS (Network of Excellence for ALS), a global network of researchers, clinicians, and people with ALS working together to accelerate the discovery and delivery of effective treatments[@neals].

Overview

<table class="infobox infobox-institution">
<tr>
<th class="infobox-header" colspan="2">ALS Clinical Trials Consortium (NEALS)</th>
</tr>
<tr>
<td class="label">Location</td>
<td>Multiple sites across the United States and internationally</td>
</tr>
<tr>
<td class="label">Type</td>
<td>Clinical Trial Network</td>
</tr>
<tr>
<td class="label">Founded</td>
<td>1997 (NEALS); coordinated consortium formed 2010s</td>
</tr>
<tr>
<td class="label">Funding</td>
<td>National Institute of Neurological Disorders and Stroke (NINDS), ALS Association, MDA</td>
</tr>
<tr>
<td class="label">Website</td>
<td><a href="https://www.neals.org" target="_blank">neals.org</a></td>
</tr>
<tr>
<td class="label">Focus Areas</td>
<td>[Amyotrophic Lateral Sclerosis](/diseases/als), Clinical Trials, Therapeutic Development</td>
</tr>
<tr>
<td class="label">Parent Institution</td>
<td>[NIH - National Institutes of Health](/institutions/nih)</td>
</tr>
</table>

ALS Clinical Trials Consortium (NEALS)

The ALS Clinical Trials Consortium refers to the coordinated network of clinical trial sites and researchers dedicated to accelerating ALS therapeutic development. In the United States, this function is primarily served by NEALS (Network of Excellence for ALS), a global network of researchers, clinicians, and people with ALS working together to accelerate the discovery and delivery of effective treatments[@neals].

Overview

| Attribute | Details |
|-----------|---------|
| Full Name | NEALS (Network of Excellence for ALS) |
| Type | Clinical Trial Consortium |
| Founded | 1997 |
| Headquarters | Boston, MA (Massachusetts General Hospital) |
| Coverage | 100+ clinical sites globally |
| Focus | ALS and frontotemporal dementia (FTD) |

Mission and Objectives

NEALS' mission is to accelerate the development of effective treatments for ALS by:

History

Foundation and Growth

The Northeast ALS (NEALS) Consortium was established in 1997 by Dr. Michael Benatar and Dr. Matthew B. Harms at the Massachusetts General Hospital in Boston. What began as a regional network of academic medical centers in the northeastern United States has grown into one of the most influential ALS clinical trial networks globally[@nealsa].

Key milestones:

| Year | Milestone |
|------|-----------|
| 1997 | NEALS founded as regional consortium |
| 2000s | Expanded to national coverage |
| 2010 | Integrated with NIH ALS clinical research |
| 2015 | Launch of adaptive platform trials |
| 2020 | Remote trial capabilities expanded |

Structure and Leadership

Administrative Structure

• Executive Director: Dr. Jeremy M. Shefner (University of Rochester)
• Co-Principal Investigators: Dr. Matthew B. Harms, Dr. Michael Benatar
• Scientific Advisory Board: Leading ALS researchers internationally
• Patient Advisory Board: People living with ALS and caregivers

Clinical Trial Sites

NEALS encompasses over 100 clinical sites across North America, Europe, and Asia-Pacific, including:

• Major academic medical centers
• University-affiliated research hospitals
• Specialized ALS clinics
• International partner sites

Key Functions

Clinical Trial Operations

NEALS provides comprehensive infrastructure for ALS clinical trials:

| Function | Description |
|----------|-------------|
| Site Certification | Standardized training and certification for trial sites |
| Protocol Development | Expert input on trial design and endpoints |
| Patient Recruitment | Centralized databases and outreach programs |
| Data Management | Standardized data collection and quality control |
| Regulatory Support | IND filing and FDA engagement |

Research Programs

Therapeutic Focus Areas

Biomarker Initiatives

• Biofluid Biomarkers: [Neurofilament light](/biomarkers/neurofilament-light-chain-nfl) chain (NfL), neurofilament heavy chain (NfH)
• Neuroimaging: MRI and PET biomarkers
• Electrophysiology: Motor unit number estimation (MUNE)
• Digital Biomarkers: Wearable devices and remote monitoring

Major Clinical Trials

Completed Landmark Trials

Ongoing and Recent Trials

| Trial | Therapy Type | Status |
|-------|--------------|--------|
| VALOR | Gene therapy (SOD1) | Completed |
| ATLAS | Gene therapy (SOD1) | Phase 3 |
| HEALEY | Platform trial | Ongoing |
| C9orf72 Trials | Antisense oligonucleotide | Phase 1/2 |
| FUS Trials | Gene therapy | Phase 1/2 |

Impact on ALS Research

Contributions to the Field

NEALS has made significant contributions to ALS research:

• Accelerated Timelines: Reduced time from protocol approval to first patient enrolled
• Standardization: Established gold-standard protocols adopted globally
• Patient Access: Expanded access to experimental therapies through multicenter trials
• Data Sharing: Created open-access datasets advancing understanding of ALS
• Regulatory Engagement: Pioneered work with FDA on adaptive trial designs

Publications and Resources

NEALS researchers have published hundreds of peer-reviewed articles on ALS clinical trials, biomarkers, and disease mechanisms. Key resources include:

• NEALS Clinical Trial Manual
• ALS Clinical Outcome Measures Guidelines
• Motor Function Assessment Standards

Collaborations and Partnerships

Federal Agencies

• National Institute of Neurological Disorders and Stroke (NINDS): Primary federal funder
• National Center for Advancing Translational Sciences (NCATS)
• Food and Drug Administration (FDA): Regulatory guidance and accelerated approval pathways

Patient Advocacy Organizations

• ALS Association: Research funding and patient support
• Muscular Dystrophy Association (MDA): Research support
• I AM ALS: Patient advocacy and awareness

Industry Partners

NEALS collaborates with pharmaceutical and biotech companies to conduct clinical trials, including:

• Biogen (tofersen, BIIB105)
• Amylyx Pharmaceuticals (AMX0035)
• Cytokinetics (tirasemtiv)
• BrainStorm Cell Therapeutics (NurOwn)

International Networks

• European ALS Consortium (ENCALS)
• Japanese ALS Association
• Australian Motor Neurone Disease Research Institute

Related Pages

• [[Amyotrophic Lateral Sclerosis](/diseases/als)](/diseases)
• [<a href="/diseases/als](/diseases)
• [[SOD1](/genes/sod1)](/mechanisms/dopaminergic-neuron-vulnerability)
• [[C9orf72](/genes/c9orf72)](/mechanisms/dopaminergic-neuron-vulnerability)
• [[FUS](/genes/fus)](/mechanisms/dopaminergic-neuron-vulnerability)
• [<a href="/mechanisms/als](/mechanisms)
• [<a href="/clinical](/clinical-trials)
• [<a href="/institutions/nih">NIH](/institutions)
• [<a href="/institutions/ninds">NINDS](/institutions)
• [<a href="/institutions/massachusetts](/institutions)

See Also

• [Alzheimer's Disease](/diseases/alzheimers-disease)
• [Parkinson's Disease](/diseases/parkinsons-disease)

External Links

• [PubMed](https://pubmed.ncbi.nlm.nih.gov/)
• [KEGG Pathways](https://www.genome.jp/kegg/pathway.html)

References