Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a fatal neurodegenerative disease that affects muscles, including those essential for breathing.
This condition mainly affects motor neurons in the brain and spinal cord, leading to difficulty controlling muscle movements and, in advanced stages, paralysis. Finnish researchers recently identified a drug candidate called brain-derived neurotrophic dopamine factor (CDNF) that shows potential for slowing the progression of ALS, according to preclinical studies.
ALS can affect all the muscles in the body, including those responsible for breathing and swallowing. ALS patients have a poor survival rate and may die within one to three years of the onset of symptoms. To date, there is no effective treatment for this disease. The most widely used therapeutic option, riluzole, extends patients’ lives by only a few months.
One of the major obstacles in developing treatments for ALS has been identifying the factors that contribute to the progression of the disease. Previous research has indicated that endoplasmic reticulum (ER) stress may play a crucial role in nerve cell damage in ALS. ER is a component of cells involved in protein production, and its chronic stress can lead to cell death.
The protein CDNF, which has been studied in the past for the treatment of Parkinson’s disease, has also been shown to be able to help people with ALS by protecting motor neurons and reducing stress in the ER.
The Finnish study used genetically engineered animal models to mimic specific ALS mutations in humans. When the researchers administered CDNF to these animals, it significantly improved motor functions and slowed the symptoms of paralysis. It was also observed that a greater number of motor neurons survived in the spinal cord of animals that received the CDNF treatment compared to those that did not.
The results of this study, published in the journal Brain, suggest that CDNF could be a promising way to treat ALS. However, it is important to note that these are results from preclinical studies, and further research is needed to validate the efficacy and safety of this drug candidate in human patients.
Source: 360medical.ro
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2023-10-08 09:43:39
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