Researchers have seen the positive results of a drug in an animal test.
Between 100 and 150 new cases of prion diseases are detected each year in France. Creutzfeldt-Jakob disease is one of these neurodegenerative pathologies, such as fatal familial insomnia (FFI) and Gerstmann-Straüssler-Scheinker syndrome. No treatment can currently cure these transmissible subacute spongiform encephalopathies, but scientific research is working on it. In the specialist journal Nucleic Acids ResearchAmerican researchers present the results of their work.
Different symptoms
Prion diseases are the result of an accumulation of the prion protein in the brain, in a malformed version. “It folds on itself very tightly, which makes it hydrophobic, poorly soluble and resistant to degradation”Explain Inserm. The main symptom is dementia, but several neurological disorders can appear such as vision problems, epileptic seizures or even coordination disorders. The course of the disease is inevitable and rapid.
Reduce the level of the prion protein
This new research on the treatment of prion diseases looked at reducing the level of prion proteins in animals. They have found that this can triple the chances of survival of animals suffering from this type of pathology. According to their results, even a small decrease in levels has positive effects. This type of therapy is normally effective when started before the first symptoms appear, but scientists find that a single dose of the prion protein reduction treatment can suppress markers of the disease, and this even when disease signals are visible in the brain. “There are still many steps ahead of ustempers Sonia Vallabh, lead author of the study, but these data make us optimistic.”
A researcher – patient
The scientist is directly concerned by her results, since her mother died of a prion disease. She also discovered that she carried a genetic mutation that makes her susceptible to developing prion disease in the future. His works are all devoted to this type of pathologies and his objective is to find, one day, a treatment to prevent their development.
