New experimental vaccine for Lou Gehrig’s disease gets EU funding

Intravacc, a contract development and manufacturing organization (CDMO) and the German Center for Neurogenerative Diseases (DZNE), have received US$2.5 mn to develop a vaccine against a form of motor neuron disease.

The funding has been awarded by the European Union’s EIC Transition Grant and will be used to research a vaccine for amyotrophic lateral sclerosis (ALS), a neurodegenerative disease also known as Lou Gehrig’s disease.

The project aims to develop a prototype ALS vaccine targeting the most common genetic ALS variant known as the C9orf72 gene, which causes between five and 10 percent of all ALS cases.

The investment will fund process development, scale-up and a toxicology study. Once the vaccine candidate is identified at DZNE, it will be clinically tested in humans.

Targeting gene mutations

ALS is triggered by protein aggregation in the brain and the spinal cord motor neurons that leads to paralysis and ultimately death, usually within two to five years.

Gene mutations have been identified as triggers for some forms of the disease, with a mutation in the C9orf72 gene being the most common. These mutations create toxic proteins called poly-Glycine-Alanine (poly-GA) that cause neurons to die.

DZNE has developed a vaccine that triggers an immune response to poly-GA, preventing them from replicating. Researchers believe that with regular vaccination, sufficient antibodies can be produced and maintained to reduce poly-GA levels. It is also hoped the vaccine could benefit those at risk of the disease as well as patients that develop related illnesses such as dementia.

The research is significant as there is currently no cure for motor neuron diseases. In the US alone it is estimated that there are 30,000 C9orf72 ALS cases, with 5,000 new cases diagnosed each year. An additional 9,000 mutation carriers who currently show no symptoms may be at risk of developing the illness.

The search for a cure

Despite its categorization as a rare illness, ALS is the most common type of motor neuron disease. It typically occurs in people between 40–70 years old, with men affected slightly more often than women.

Despite ongoing research, existing therapies can only alleviate symptoms rather than stop progression of the disease. There are two approved medications that can reduce symptoms: riluzole, currently marketed by Sanofi, and edaravone, sold by Mitsubishi Tanabe under the brand name Radicava.

A number of other studies are underway, among them research into a stem cell treatment called NurOwn, investigated by Brainstorm-Cell Therapeutics. While promising, at the end of its Phase III trial there was no significant difference between responses to NurOwn and responses to a placebo.

Commenting on Intravacc’s research into a new vaccine, its CEO Dr Jan Groen said: “There 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 are expected to commence in 2025.”

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