New drug saves mice from dementia symptoms Bruce Willis Neuron: gene therapy reverses frontotemporal dementia symptoms in mice 18:18

A gene therapy drug developed by Australia that requires a single dose stopped the development of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) in mice. What’s more, it also reversed some symptoms of dementia, according to a study in the journal. Neuron.

Normally, nerve cells produce the protein TDP-43, which is important for their function. But its buildup can damage cells, which occurs in some types of dementia. The new drug CTx1000 targets clusters of the TDP-43 protein. At the same time, it affects only the pathological form of the molecule, while the healthy one continues to perform its functions.

Experiments in mice showed that CTx1000 halted the progression of amyotrophic lateral sclerosis and frontotemporal dementia, even in very advanced stages, and eliminated behavioral symptoms associated with dementia.

Deposition of the TDP-43 protein is also seen in approximately 50% of cases of Alzheimer’s disease (the most common form of dementia). Therefore, the new drug may also be beneficial for people with this disorder.

Amyotrophic lateral sclerosis causes the progressive loss of neurons necessary for brain-muscle communication. In the early stages, patients experience muscle weakness, but as the disease progresses, they gradually lose the ability to walk, talk, swallow and breathe without assistance. Frontotemporal dementia is one of the rarest types of dementia, but the second most common type in people under 65. Actor Bruce Willis was diagnosed with the disease in 2023. It causes a decrease in cognitive functions, anxiety and character changes. There is no cure for either disease.

Scientists hope to begin testing CTx1000 in humans within two years and have already received a $1.2 million preclinical grant. The total cost of the tests is estimated to be 22 million dollars.

used to be a doctor in the name Early symptoms of Alzheimer’s disease.