Intravacc and DZNE Secure EU Funding to Develop ALS Gene Variant Vaccine
- Total amount of 2.5 million euros for the development of a vaccine against amyotrophic lateral sclerosis (ALS)
- ALS vaccine targets the most common genetic variant of ALS
- 5-10% of all ALS cases are caused by a mutation in the C9orf72 gene
BILTHOVEN, Netherlands and MUNICH, July 6, 2022 /PRNewswire/ — Intravac, a global contract development and manufacturing organization (CDMO) for preventive and therapeutic vaccines and the German Center for Neurogenerative Diseases (DZNE), have received funding of €2.5 million from the European Union (EIC Transition Grant) to continue the development of a prototype ALS vaccine, including process development, the scale and the toxicological study. The project aims to develop the vaccine candidate identified at DZNE until it can be clinically tested in humans.
ALS is a fatal neurodegenerative disease that is triggered by the aggregation of proteins in the motor neurons of the brain and spinal cord that leads to paralysis and ultimately death. Gene mutations have been identified as triggers for some forms of the disease. About 5-10% of all ALS cases are caused by a mutation in the C9orf72 gene, making it the most common genetic variant of ALS. Unlike most individuals, these patients carry a massively extended repeat region in an otherwise silent part of this gene. Nevertheless, the research group of Prof. dr. Dieter Edbauer at DZNE, discovered that these additional sequences are translated into toxic proteins, most abundantly, large chain-like molecules called poly-Glycine-Alanine (poly-GA). These molecules trigger downstream pathology in mouse models, ultimately causing neurons to die.
An experimental vaccine
DZNE has developed an experimental vaccine that instructs the immune system to produce antibodies against these harmful poly-GA molecules. In a mouse model, this reduces poly-GA aggregates and largely prevents motor deficits. Regular vaccination is necessary to maintain sufficient antibody levels. In humans, more than 2,500 prevalent cases of C9orf72 ALS have been reported in the United States, and Europe only. An estimated 9,000 mutation carriers who currently have no symptoms but are at risk of developing disease within 10 years could also benefit from this approach. Similar vaccine concepts might even help patients who develop a related condition called frontotemporal dementia.
Teacher. dr. Dieter Edbauer, group leader at DZNE, said:
“Before we can test this approach in ALS patients, we need to establish clinical-grade production of our vaccine and conduct further safety studies. We are grateful that the EU is supporting this development with the EIC Bridge Grant. . Overall, we hope that with the help of Intravacc, the results of this joint project will advance the large-scale application of vaccines in debilitating neurodegenerative diseases.”
dr. John GreenCEO of Intravacc, said:
“There is an unmet need for effective, disease-modifying therapies to treat patients with ALS. The goal of our current project is to develop the vaccine to the point where it can be tested in humans. Clinical trials for ALS C9orf72, which is the most common genetic variant of ALS, are expected 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 ever clinical vaccine trial against ALS in humans.“
Amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, is a progressive neurodegenerative disease of the motor neurons of the brain and spinal cord, resulting in progressive paralysis, with death usually within 2 to 5 years of diagnosis. ALS usually occurs in people between the ages of 40 and 70, with men being affected slightly more often than women. To date, there is no cure for ALS. Current therapies can only relieve symptoms, but cannot stop neuron loss and disease progression. ALS is a rare disease with a multifactorial etiology, and the precise pathogenetic mechanism is still unknown. In most patients (85-90%), the cause of ALS is unknown. This situation is called “sporadic ALS”. 10 to 15% of ALS cases have genetic causes. Genetic variants of ALS triggered by known mutations, such as C9orf72 repeated expansion offers a unique chance for targeted therapy.
About Intravacc BV
Intravacc, located at Utrecht Science Park Bilthoven at the Netherlands, is one of the world’s leading contract development and manufacturing organizations (CDMOs) of innovative infectious disease vaccines. As an established independent CDMO with outstanding experience in vaccine development and vaccine technologies, Intravacc has transferred its technology related to poliomyelitis vaccines, measles vaccines, DTP vaccines, Hib vaccines and flu around the world. Approximately 40% of childhood disease vaccines are based on Intravacc’s proprietary technology. Intravacc offers a wide range of expertise to develop vaccines from concept to phase I/II clinical studies for partners around the world, including universities, public health organizations (WHO, Bill & Melinda Gates Foundation), corporations biotechnology and pharmaceuticals. For more information, please visit www.intravacc.nl.
About the Deutsches Zentrum für Neurodegenerative Erkrankungen, DZNE
(German Center for Neurodegenerative Diseases)
The DZNE is a research institute funded by the German federal and state governments, comprising ten sites across Germany. It is dedicated to diseases of the brain and nervous system, such as Alzheimer’s disease, Parkinson’s disease and ALS, which are associated with dementia, movement disorders and other serious health conditions. To date, there is no cure for these diseases, which place a huge burden on countless affected individuals, their families and the healthcare system. The goal of DZNE is to develop new strategies for prevention, diagnosis, care, as well as treatment, and put them into practice. To this end, DZNE cooperates with universities, teaching hospitals, research centers and other institutions in Germany and abroad. The institute is a member of the Helmholtz Association and belongs to the German Centers for Health Research. www.dzne.de/en
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