Cure For ALS On The Horizon After Promising Animal Study

HELSINKI, Finland — A cure for amyotrophic lateral sclerosis (ALS) could be just a few years away, thanks to a promising animal study. Researchers in Finland have found that treating cells using a protein called cerebral dopamine neurotrophic factor (CDNF) could slow one of the biggest triggers for ALS.

ALS is a fatal neurodegenerative disease that weakens the muscles over time. The condition, also known as Lou Gehrig’s disease in the United States, targets the nerve cells in the brain and spinal cord, especially motor neurons in the spinal cord. The death of these cells means the brain is unable to relay messages to muscles. This can translate to difficulty controlling muscle movements, and in the disease’s later stages, weakening of the muscles and paralysis.

ALS can affect all muscles — including the ones that help you breathe and swallow. People with ALS often die from respiratory failure within one to three years from the onset of symptoms.

Currently, there is no cure for ALS. One of the few treatments available, riluzole, extends the survival rate of patients by only a few months. An issue researchers run into when developing ALS treatments is pinpointing what drives the disease to progressively worsen.

Previous research has pointed to endoplasmic reticulum (ER) stress as one of the main culprits behind a person’s deteriorating state. The endoplasmic reticulum inside cells is involved in creating one-third of all the cell’s protein. ER stress is a cellular response to correct misfolding of proteins. However, this protective mechanism is meant to be temporary. Chronic ER stress would not protect cells but rather induce cell death.

In 2007, scientists discovered a CDNF protein that could potentially avoid ER stress. It has been previously shown to help people with Parkinson’s disease, possibly by keeping motor neurons alive.

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The study used three genetically modified animal models that mimicked human mutations people with ALS have in their bodies. When researchers introduced CDNF to rats and mice, it significantly improved their motor behavior and slowed down paralysis symptoms. This reflected the higher number of surviving motor neurons in the spinal cord in animals who received the treatment versus those who did not.

“Our experiments suggest that CDNF may rescue motoneurons by reducing the ER stress response and, therefore, cell death. Importantly, ER stress was present in all our animal models, independently of the specific genetic mutations,” says Dr. Francesca De Lorenzo, a PhD graduate at the University of Helsinki in Finland and lead author of the study, in a media release.

“CDNF holds great promise for the design of new rational treatments for ALS,” adds Dr. Merja Voutilainen, an assistant professor at the University of Helsinki and the senior author of the study.

The study is published in the journal Brain.

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About the Author

Jocelyn Solis-Moreira

Jocelyn is a New York-based science journalist whose work has appeared in Discover Magazine, Health, and Live Science, among other publications. She holds a Master’s of Science in Psychology with a concentration in behavioral neuroscience and a Bachelor’s of Science in integrative neuroscience from Binghamton University. Jocelyn has reported on several medical and science topics ranging from coronavirus news to the latest findings in women’s health.

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