Research is how new treatments, better care, and answers about the causes of ALS/MND are found. It ranges from work in a laboratory to studies that follow people over many years. People living with ALS/MND, caregivers, and families take part at every stage.
This page explains the main types of research, how a treatment moves from the laboratory to approval, and what taking part in a clinical trial involves. It also links to Alliance pages that go into more detail and to research organizations you can follow.
On this page
Types of research │ From laboratory to approval │ Learn more │ Research organizations
Types of research
Different types of research answer different questions. Most progress depends on several of them working together.
Laboratory and pre-clinical research
Scientists study cells, tissue, and animal models to understand how ALS/MND develops and to test ideas for treatments before they are tried in people. This includes genetics and genomics, which look for genes linked to the disease, and biomarker research. Biomarkers are measurable signs, found for example in blood or spinal fluid, that may show whether a disease is present, how fast it is progressing, or whether a treatment is working.
Epidemiology
Epidemiology studies how many people are affected by ALS/MND, who they are, and why patterns differ between populations and regions.
Clinical trials
Clinical trials are research studies with people. They can test a treatment (interventional trials) or follow people over time without giving a treatment (observational studies). Interventional trials look at safety, tolerability, and whether a treatment works.
Care and clinical management research
This research looks at how to deliver better care today. Examples include how multidisciplinary clinics are organized, and how assistive technology, rehabilitation, or social support can improve quality of life.
Social science research
Social science research asks people living with ALS/MND and caregivers about their experiences and priorities, so that research and care address what matters most to them.
How a treatment moves from the laboratory to approval
Before a new treatment can be approved, it goes through several steps. Regulatory agencies review the results, and each country decides for itself whether to approve a treatment.
- Pre-clinical research. A possible treatment is tested in the laboratory to see whether it is likely to be safe and effective.
- Phase 1. A small group of people receives the treatment. The main goals are to check safety and find a suitable dose.
- Phase 2. A larger group receives the treatment. Researchers look for early signs that it works and continue to monitor safety.
- Phase 3. A larger group is studied, usually comparing the treatment with a placebo or with standard care. A placebo is an inactive treatment used for comparison. Results from this phase are the main evidence regulators review.
- Regulatory review. The regulatory agency in each country reviews the evidence and decides whether to approve the treatment.
Some newer trials are called platform trials. They test several treatments at the same time within one trial structure, which can speed up the search for effective therapies.
Learn more on this website
Recommendations for Trial Sponsors
What the Alliance expects from sponsors after a trial ends.
Open Label Extension
Continued access to a study treatment after a trial.
Clinical Trial Registry Accuracy and Maintenance in ALS/MND Research
Drugs in development: SOD1 Therapies and Trials and C9orf72 Therapies and Trials
Resources on research
These Alliance members publish research updates and news.
ALS Canada Research Program
ALS Canada · English
ALS Canada funds fundamental and clinical research in Canada and brings the national ALS research community together to share ideas and collaborate.
ALS Therapy Development Institute
ALS Therapy Development Institute · United States · English
ALS TDI is a nonprofit biotech and drug discovery laboratory focused only on ALS.
Les Turner ALS Center at Northwestern Medicine
Les Turner ALS Foundation · United States · English
The Les Turner ALS Foundation supports research at Northwestern University Feinberg School of Medicine, including studies of genetic causes of ALS and of how motor neurons degenerate.
European Network to Cure ALS (ENCALS)
ENCALS · English
ENCALS is a network of ALS centres in Europe. It holds an annual scientific meeting, gives awards to early-career researchers, and publishes a directory of ALS centres.
Project MinE
Project MinE consortium · English
Project MinE is an international research consortium that analyzes whole genome sequencing data to find genetic variants that contribute to ALS. It is building a publicly accessible repository of this data.