One million euros for research into motor neuron disease ALS
Ludo Van Den Bosch wins the Generet Award for Rare Diseases
February 16, 2023
The Generet Award for Rare Diseases, now in its fifth year, has been awarded to Ludo Van Den Bosch for his research into the underlying mechanisms of amyotrophic lateral sclerosis (ALS), a rare form of motor neuron disease. This award from the Generet Fund, managed by the King Baudouin Foundation, is accompanied by a grant of €1 million, making it the most prestigious prize in Belgium for work on rare diseases. The jury of independent experts praised Van Den Bosch's ambition to improve patients’ lives with ALS through his research.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting the nerve cells that make movement possible. The condition, also known as Lou Gehrig’s Disease, selectively affects motor neurons, the cells that communicate with the muscles. As a result, people with ALS gradually lose control of their muscles until they are completely paralyzed and unable to speak and breathe. The disease is incurable, with the age of onset typically relatively young (between 50 and 60). Patients usually die within two to five years of diagnosis. In Belgium today, about 1,000 people are suffering from this disease and 100 to 250 new diagnoses are made every year.
Ludo Van Den Bosch has been studying the underlying mechanisms of this neurodegenerative disease for many years at the Laboratory for Neurobiology at KU Leuven/VIB, Belgium's leading center for the study of ALS. The research is carried out in close collaboration with Philip Van Damme, a neurologist at Gasthuisberg University Hospital in Leuven, and with the ALS League, a Belgian association of ALS patients. They work intensively with national and international research groups and are part of the European Network to Cure ALS (ENCALS), Project MinE, and the Robert Packard Center for ALS Research in the United States.
Cause usually unknown
“In 90% of patients, the cause of the disease is unknown, while in the remaining 10% it is genetically determined - in other words, there will be multiple family members who suffer or have suffered from the disease,” explains Ludo Van Den Bosch. “We've developed various models for ALS in the lab. For example, we use fruit flies, zebrafish, and rodents to study the disease process better. For several years now, it has also been possible to make induced pluripotent stem cells (iPSCs) from patient skin cells. These cells can be converted into motor neurons and muscles having the same characteristics as those in ALS patients. This enables us to study the disease with greater precision.”
Altered neuronal transport
Ludo Van Den Bosch and his team of researchers have discovered that, in ALS patients, transport along the processes that connect the cell body in the spinal cord to the muscles is disrupted. These processes can be more than a meter long, and normal transport in these processes is critical for the normal functioning of the motor system: “We discovered that we can restore disrupted transport by selectively inhibiting a certain enzyme, HDAC6,” Van Den Bosch says. “In addition, inhibiting HDAC6 also has beneficial effects on the expansion of motor neuron processes and the formation of neuromuscular junctions, the connections between the motor neuron processes and the muscles.”
Ludo Van Den Bosch and his team will use the financial support associated with the Generet Prize to continue researching the positive effects of HDAC6 inhibition on motor neurons. In doing so, they will examine which mechanisms are responsible for these beneficial effects and will determine which proteins HDAC6 influences and how the inhibition of this enzyme induces this effect.
The therapeutic ambition is clear: “The ultimate goal of this scientific research is to translate these new insights into the action of HDAC6 inhibition into a new therapeutic strategy that can be tested in ALS patients,” says Ludo Van Den Bosch. “We hope to make an important contribution towards finding a cure for this rare and fatal neurodegenerative disease.”
About the Generet Prize
The Generet Fund, managed by the King Baudouin Foundation, aims to make Belgium an international centre for research into rare diseases. Since 2018, the Fund has awarded an annual €1 million prize to a leading researcher for his or her work on rare diseases in Belgium. The Fund does not restrict which diseases the research should focus on: all rare diseases are eligible, as are methodologies that can advance research into multiple diseases. A new Generet Fund Prize call is being launched this month. The call will target established researchers in the field of rare diseases but is also open to researchers with a solid track record in more mainstream pathologies who have a particular interest in rare diseases and who wish to pursue further research in this specific area or wish to accelerate their research.
About Prof. Dr Ludo Van Den Bosch
Since 2013, Prof. Dr Ludo Van Den Bosch has served as head of the Laboratory for Neurobiology at KU Leuven/VIB. Besides studying ALS for more than 25 years now, Dr Van Den Bosch is also a full professor at KU Leuven and group leader at the VIB-KU Leuven Center for Brain & Disease Research. He performs his research in close collaboration with Prof. Philip Van Damme, a neurologist at Gasthuisberg University Hospital in Leuven, and with Belgium’s ALS League.
The Generet Award winners to date
- 2018: Prof. Dr Mikka Vikkula (De Duve Institute - UCLouvain), for his research into the genetic causes of vascular abnormalities.
- 2019: Prof. Dr Steven Laureys (Coma Science Group - ULiège and Liège University Hospital), for his research into altered states of consciousness caused by severe brain injury.
- 2020: Prof. Dr Pierre Vanderhaegen (VIB-KU Leuven Center for Brain & Disease Research and ULB), for his research into the development of the brain and its abnormalities.
- 2021: Prof. Dr Rosa Rademakers (VIB-UAntwerp Center for Molecular Neurology) for her research into a rare form of dementia.
- 2022: Prof. Dr Ludo Van Den Bosch (VIB-KU Leuven Center for Brain & Disease Research), for his research into the underlying mechanisms of amyotrophic lateral sclerosis (ALS).