Intravacc, a world leading
contract development and manufacturing organization (CDMO) of preventive and
therapeutic vaccines and the German Center for Neurogenerative Diseases (DZNE),
have been awarded a funding of € 2.5 million from the European Union (EIC
Transition Grant) to further develop a prototype ALS vaccine, including process
development, scale-up and toxicology study. The project aims to develop the
vaccine candidate identified at DZNE to the point where it can be clinically
tested in humans.
a fatal neurodegenerative disease that is triggered by protein aggregation in
the brain and spinal cord motor neurons that leads to paralysis and ultimately
to 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 ALS variant. In contrast to most
individuals, these patients carry a massively expanded repeat region in an otherwise
silent part of this gene. Nevertheless, the research group of Prof. Dr. Dieter
Edbauer at DZNE, discovered that these extra 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
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 required to maintain sufficient antibody levels. In humans, over
2,500 prevalent C9orf72 ALS cases have been reported in the US, and Europe alone.
An estimated 9,000 mutation carriers who currently show no symptoms but are at
risk to develop disease within 10 years could also benefit from this approach.
Similar vaccine concepts could perhaps even help patients that develop a
related disease, called frontotemporal dementia.
Prof. Dr. Dieter Edbauer, group leader at DZNE, said:
we can test this approach in ALS patients, we need to establish clinical grade
production of our vaccine and do further safety studies. We are grateful that
the EU supports this development with the EIC Transition Grant. All in
all, we hope that with the help of Intravacc, results from this joint project
will advance the broad application of vaccines in debilitating
Dr. Jan Groen, Intravacc’s CEO, said:
is an unmet need for effective, disease-modifying therapies to treat ALS
patients. The goal of our current project is to develop the vaccine to the
point where it can be tested in humans. Clinical trials for C9orf72 ALS, which
is the most common genetic variant of ALS, are expected to commence in 2025.
Our experience in developing similar conjugate vaccines for infectious diseases
will greatly accelerate the preclinical development and support the start of
the first ever in human ALS vaccine clinical trial.”