Laboratory cultivation of "mini brains": potential treatments for motor neuron disease and frontotemporal dementia

Researchers at the University of Cambridge have developed a "mini brain" that allows them to study a deadly, incurable neurological disease that can lead to paralysis and dementia, and this is the first time they have been able to cultivate this in nearly a year.

Amyotrophic lateral sclerosis is a common motor neuron disease that often overlaps with frontotemporal dementia (ALS/FTD) and can affect young people, mainly after 40-45 years of age

In general, organoids commonly referred to as "mini-organs" are increasingly being used to simulate human biology and disease

Normally, researchers extract cells from the skin of patients and reprogram these cells to return to the stem cell stage

Scientists at the John Van Gist Brain Repair Center at the University of Cambridge used stem cells from ALS/FTD patients to grow brain organs

Although this is not the first time scientists have cultivated the mini-brains of patients with neurodegenerative diseases, most efforts can only cultivate them in a relatively short period of time, which represents a limited range of diseases related to dementia

Dr.

"In order to get close to capturing this complexity, we need longer-lived models and replicate the composition of human brain cell populations that normally interfere.

Although organoids usually grow in the form of cell balls, the first author, Dr.

Dr.

In this way, Dr.

Dr.

In addition to helping to understand the development of the disease, organoids can also be a powerful tool for screening potential drugs to determine which drugs can prevent or slow the progression of the disease

The research team found that a drug called GSK2606414 can effectively alleviate common cellular problems in ALS/FTD patients, including the accumulation of toxic proteins, cellular stress, and loss of nerve cells, thereby blocking a pathway that leads to the disease

Dr.

Dr.
Lakatos added: "At present, we do not have a very effective method to treat ALS/FTD.
Although there is still a lot of work to be done after our discovery, it at least gives us hope and may be able to prevent it in time.
Or slow down the disease process
.
"

"In the future, it is also possible to extract skin cells from patients, reprogram them to grow'mini brains', and test which unique drug combination is best for their disease
.

Article title

Human ALS/FTD Brain Organoid Slice Cultures Display Distinct Early Astrocyte and Targetable Neuronal Pathology