| ▲ A new gene silencing treatment that lowers the level of tau protein in the body may help treat Alzheimer’s disease, a study has found. (Photo = DB) |
[메디컬투데이=최재백 기자] A new treatment that lowers the level of tau protein in the body may help treat Alzheimer’s disease, a new study has found.
A new gene silencing treatment that lowers the level of tau protein in the body can help treat Alzheimer’s disease, the results of a study were published in the journal Nature Medicine.
Tau protein is commonly found in nerve cells that make up the nervous system, and in a healthy brain, tau protein plays a role in stabilizing the internal structure of nerve cells.
However, in the brains of patients with Alzheimer’s disease, tau protein aggregates and accumulates abnormally in brain cells, rather the structure of nerve cells collapses, and signal transmission between nerve cells does not occur.
The research team said they were able to lower the level of tau protein in the body by developing a gene silencing technology that suppresses the expression of the ‘microtubule-associated protein tau (MAPT)’ gene that induces tau protein formation.
BIIB080, an experimental drug under development by the research team, is an antisense oligonucleotide that utilizes small RNA or DNA fragments to block the function of a specific RNA to produce tau protein.
In the first phase of the clinical trial, the research team recruited 46 mild Alzheimer’s patients with an average age of 66 years old and observed the effect of different doses when BIIB080 was injected into the nervous system through the spinal canal. The treatment lasted 13 weeks, and the patients were injected with the drug every 4 weeks or every 12 weeks.
As a result of the study, the treatment group that received the highest dose showed more than 50% reduction in total tau and phosphorylated tau concentrations after 24 weeks of treatment.
On the other hand, mild to moderate side effects were reported in 94% of treated patients, the most common side effect being headache.
Experts explained that research should be conducted to develop treatments that can improve the quality of life of people with or predisposed to Alzheimer’s disease.
They noted the need to test the safety and tolerability of BIIB080 in a phase 2 clinical trial, while cautioning that lowering the level of tau protein does not prevent cognitive decline.
He added that further phase 3 clinical trials should properly evaluate the therapeutic efficacy of BIIB080 to determine whether memory can be improved or whether memory decline can be delayed.
In addition, since tau protein is involved in not only Alzheimer’s disease but also other dementia diseases, it was evaluated that the results of this study could be widely applied to psychiatric diseases.
Medical Today Reporter Jaebaek Choi ([email protected])
[ⓒ 메디컬투데이. 무단전재-재배포 금지]
2023-05-07 22:37:34
#gene #therapy #prevent #Alzheimers #disease
