German researchers are working on a therapeutic vaccination against amyotrophic lateral sclerosis (ALS), a nervous disease. Thanks to EU funding, the prototype can now be further developed and, at best, will arrive at the clinic in a few years.
Amyotrophic lateral sclerosis, abbreviated to ALS, is a diagnosis of shock. Neurodegenerative disease leads to the most severe disabilities and is always fatal. The reason is that harmful proteins are deposited in the motor neurons of the brain and spinal cord and cause their death. About 5-10% of all ALS diseases are caused by a mutation in the so-called C9orf72 gene and are therefore the most common genetic variant of ALS.
The vaccine targets the most common genetic mutation
Scientists from the German Center for Neurodegenerative Diseases (DZNE) now want to use this genetic mutation as a target for a therapeutic vaccine. There is already a prototype. Thanks to EU funding of € 2.5 million, the vaccine can now be further developed. The goal of the project is to bring the vaccine candidate into clinical research. To this end, DZNE is partnering with Intravacc BV, a global contractor for the development and production of preventive and therapeutic vaccines.
Much research is still needed
“Before we can test this approach in people with ALS, we need to establish the production of our vaccine in clinical quality and demonstrate the safety of our approach in further studies,” explains Prof. Dieter Edbauer, leader of the research group at the DZNE site. of Monaco. “All in all, we hope that the results of this joint project, with the help of Intravacc, will foster the widespread use of vaccines in debilitating neurodegenerative diseases.”
Vaccination is supposed to strengthen the immune system
Edbauer’s research team found that mutations in the C9orf72 gene lead to the formation of toxic proteins, particularly large chain molecules called “poly-glycine-alanine” (poly-GA). In transgenic mice, they trigger a clinical picture similar to ALS, which ultimately leads to the death of nerve cells. The vaccine candidate aims to stimulate the immune system to produce antibodies against these harmful poly-GA molecules. Experiments in mice showed that administration of the vaccine reduced poly-GA aggregates and largely prevented motor deficits. To maintain adequate antibody levels, the vaccine must be administered regularly.
The first clinical trial could start in 2025
“The goal of our current project is to develop the vaccine to the point where it can be tested in humans,” says Dr. Jan Groen, CEO of Intravacc. Clinical trials are therefore likely to begin in 2025. “Our experience in developing similar conjugate vaccines for infectious diseases will significantly accelerate preclinical development and support the start of the first clinical trial of ALS vaccines in humans,” he says. Groen with confidence.
More than 2,500 cases of the C9orf72 variant of ALS have been recorded in the United States and Europe. It is estimated that around 9,000 mutation carriers who are currently asymptomatic but at risk of developing the disease within 10 years could also benefit from the new approach.