Unraveling Hope for Prion Disease and Other Progressive Neurodegenerative Diseases

Neurodegenerative diseases, like prion disease, often progress rapidly, impairing the patient’s physical and mental functions. Some of these progressive diseases are difficult to diagnose, such as Creutzfeldt-Jakob disease, and by the time it is diagnosed, patients may only have a few months left to live. The root cause of the problem is the protein PrPC. The protein is meant to fold, but when it folds in a way that is not right, it becomes the pathogenic PrPSc conformation. In this configuration, the protein causes significant issues as it replicates and accumulates in the sufferer’s brain, hindering the area’s primary functionality.

There is no cure for diseases like prion disease, but a recent treatment has shown promise for Creutzfeldt-Jakob disease. The promising treatment could be used to better understand similar diseases and how to treat them.

The research team developed an antibody that could work to stabilize the precursor to the problematic protein. If this treatment is successful, it has application across other similar diseases, including Alzheimer’s. While only 1 in a million Americans is affected by Creutzfeldt-Jakob every year, the number of patients with Alzheimer’s is much higher. There are four different types of Creutzfeldt-Jakob disease, all with different causes.

The study of the treatment lasted from October 2018 to July 2019, and it focused on the progression of the disease. Six patients were observed. They were given an increasingly larger dose of the treatment over time. Though the study included only a small sample size over a fairly short period of time, the initial data was very promising, though two patients died before the final dose. Three of the patients showed decreased levels of PrPSc, indicating that the treatment was working. Unfortunately, the disease is fast acting, so there isn’t much time to test the effectiveness of treatments on patients. The promising results of the small trial provide a direction for treatment going forward.

You can read the full details of the initial hope at Unraveling Hope for Prion Disease and Other Progressive Neurodegenerative Diseases. At the same link, they have an audio version of the article, so you can listen to it at your leisure.

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