New therapy that destroys tau tangles shows promise for Alzheimer’s. It selectively remove aggregated tau proteins and improve symptoms of neurodegeneration in mice. The progression of their neurodegeneration symptoms slowed and they showed significantly better motor function.

I’ve linked to the press release in the post above. In this comment, for those interested, here’s the link to the peer reviewed journal article:

https://www.cell.com/cell/fulltext/S0092-8674(24)00912-7

From the linked article:

New therapy that destroys tau tangles shows promise for Alzheimer’s

Scientists have developed potential therapies that selectively remove aggregated tau proteins and improve symptoms of neurodegeneration in mice.

The team of scientists are from the Medical Research Council Laboratory of Molecular Biology (MRC LMB), and the UK Dementia Research Institute (UK DRI) at the University of Cambridge. They say this promising approach for Alzheimer’s disease could also be applied in future to other brain disorders driven by protein aggregation inside cells, such as:

motor neuron disease
Huntington’s disease
Parkinson’s disease
Alzheimer’s disease

In two papers, published in Cell and Science, they demonstrated how utilising the unique capabilities of a protein called TRIM21 gives the potential therapies two key advantages.

Firstly, they only destroyed the disease-linked tau aggregates, leaving healthy tau proteins intact. And secondly, the therapies removed already established tau aggregates in mice, not just preventing the formation of new aggregates.

Mice walk better after therapy

For the treatment to work in an animal, it needs to not only get into the brain, but also get inside the cells within the brain. To do this, the researchers used a harmless virus that has previously been developed to deliver therapies like this, called an adeno-associated virus (AAV). It delivers DNA instructions to make the custom proteins inside brain cells.

Elderly mice with tau protein aggregates were injected with a single dose of the gene therapy vector containing either the treatment, or a placebo.

Within a few weeks, there was a significant reduction in the amount of aggregated tau in the brain cells of the treated animals.

Importantly, in the mice given the RING-Bait treatment, the progression of their neurodegeneration symptoms slowed and they showed significantly better motor function, as assessed by an artificial intelligence programme that scored how well they ran.