Scientific research into ALS
ALS Centre Netherlands conducts cutting-edge scientific research into the causes of Amyotrophic Lateral Sclerosis (ALS), diagnosis, drug treatments, symptom management and care for people with ALS, Progressive Muscular Atrophy (PMA) and Primary Lateral Sclerosis (PLS). Below is an overview of the different areas of research carried out at ALS Centre Netherlands.
Types of research
Genetics:
When looking at the hereditary aspects of ALS, there are broadly two forms: familial and sporadic (non-familial) ALS. Familial ALS is usually hereditary, whereas sporadic ALS is not hereditary in most cases (approximately 90%).
Familial ALS is caused by a mutation in one or more ALS-related genes. At present, a mutation in one of the known ALS-related genes can be identified in approximately 60% of people with familial ALS. For the other 40%, it is still unknown which DNA change in which gene causes ALS. Scientific research continues to identify new ALS-related genes. The research group is led by Prof. Jan Veldink.
Project Mine
Project MinE is a groundbreaking genetic research project on ALS conducted on a global scale. The project involves collecting genetic data from people with ALS and control participants. Researchers, including those at the ALS Centre, then ‘mine’ the collected data and compare the genetic information to identify the genetic causes of ALS. The ultimate goal is to find specific leads for developing new treatments for ALS.
More information on Project Mine
Quality of life:
A great deal of research focuses on quality of life, symptom management and the use and application of assistive devices to provide people with ALS, PMA and PLS and those around them with the best possible care and support. Researchers also study the development of new assistive devices. The research group is led by Prof. Anne Visser.
Causes and disease progression:
To better understand ALS, PMA and PLS, researchers study the causes and progression of these diseases. We also use techniques such as EEG and MRI to study changes in the brain and nervous system. Understanding how these diseases develop and progress is important for finding effective treatments. The research group is led by Prof. Leonard H. van den Berg.
Patient models:
Our bodies contain a complex network of nerve fibres that allows us to move. In ALS, these networks become damaged and no longer function properly. In the laboratory, researchers at the ALS Centre can use stem cells to recreate parts of these nerve structures. These models are also known as organoids.
By recreating body structures in this way, researchers can investigate which systems are affected by ALS and subsequently study how these systems might be restored. The research group is led by Prof. Jeroen Pasterkamp.
Clinical drug trials:
Several national and international clinical drug trials are taking place at the ALS Centre to find treatments for ALS, PMA and PLS.
Clinical drug development consists of three phases. Phase 1 research examines how a drug behaves in the body. Phase 2 research investigates the possible side effects of the drug. Phase 3 is the largest phase, involving the most participants, and examines how effective the drug is.
Most clinical drug trials are developed by the pharmaceutical industry. Pharmaceutical companies write the study protocol, determine the eligibility criteria and decide how the study will be conducted. The study is then carried out at several hospitals internationally, including the ALS Centre at UMC Utrecht.
TRICALS
The clinical drug research often takes place through TRICALS, the largest international research initiative bringing together people with ALS, PMA and PLS, researchers and ALS organisations to find treatments for these diseases. To make participation as accessible as possible, TRICALS has developed a dedicated registry. People with ALS, PMA and PLS can register to receive updates when a new clinical trial starts in their country. People can register online using the registration form on the TRICALS website.
Neuromuscular Diseases Biobank
The Neuromuscular Diseases Biobank (‘Biobank NMZ’, in Dutch) is a large-scale biobank that collects data from patients and control participants for research into ALS, PMA, PLS and other neuromuscular diseases. The Neuromuscular Diseases Biobank collects data that researchers use to gain a better understanding of different neuromuscular diseases, investigate their causes and identify new possibilities for treatment.
GoALS research programme
To accelerate the search for a breakthrough treatment for ALS, the progressive neurological and muscular disease, the Duth ALS Patients’ Association, ALS Netherlands Foundation and ALS Centre (UMC Utrecht) have joined forces. Together, they established GoALS.
GoALS is a multi-year research programme with a budget of €50 million and more than 120 researchers. It brings together different research projects across three main areas: genetic research, laboratory-based (preclinical) research and research involving patients (clinical research). By conducting these different types of research together within one programme, the process of finding a treatment becomes faster and more efficient. All research follows one integrated plan and takes place within one organisation.
Funding is distributed in a way that enables researchers to work together more effectively and build more easily on each other’s results. This helps researchers work faster and more efficiently towards a breakthrough in ALS treatment. The programme also involves collaboration with companies in the pharmaceutical industry to make promising treatments available to patients more quickly.