Scientists at the University of California, San Francisco, have found a possible cure for amyotrophic lateral sclerosis (ALS), a debilitating degenerative disease that afflicts nearly a million people worldwide.
The team, led by neuroscientist Michael Siegel, reports that they have discovered a compound that blocks the activity of a protein in the central nervous system called PI3K, a key molecule involved in the normal functioning of neurons and other body systems.
The researchers say the compound may have therapeutic value for treating amyotrophic lateral sclerosis, also known as ALS, which affects people of all ages and stages of life, and affects millions worldwide.
Amyotrophic laryngotrophic disease (ALS) is caused by a deficiency of the protein p38MAPK, which is required for nerve cells to communicate.
“Our findings show that our compound could prevent the protein from triggering the activation of the brain’s immune system that is responsible for controlling the activity and activity of cells in the brain,” Professor Siegel said.
“We also found that it can be used to treat other forms of ALS.”
He said the compound had no direct effect on the function of the central brainstem, but could be administered as a nasal spray or a gel to control the symptoms of the disease.
“What we are really saying is that our study points to a possible treatment of ALS,” he said.
The findings were published in the journal Science Advances.
Professor Siegel is a member of the UC San Francisco College of Medicine.
“When we are looking at a protein, we can often use a model to see what happens to it, and then we can design a drug based on that model,” he told ABC News.
“For example, a protein that is normally associated with inflammation and inflammation-related diseases, like C-reactive protein, is seen as being the one that might be helpful for people with these diseases.”
But the drug could potentially work even when there is inflammation-associated disease.
“If there is no inflammation, there is really no reason for a protein to be the one you want to target.”
The team’s findings were based on mice, which had been genetically altered so they were unable to develop amyotropic lateral syndrome.
The compound, called MDA-MB-231, was administered to mice by a nasal cannula, which allowed them to inhale the compound as well as exhale it.
The mice were then tested for signs of disease.
The results showed that mice with amyotrophy-like symptoms did not respond to the drug.
“The animals who were genetically engineered to have a deficiency in p38 MAPK did not show any disease at all, and we were able to find that it could be given to mice,” Professor David Schulte, professor of biomedical engineering and director of the California Institute of Technology, said.
The team also found the drug reduced inflammation in the spinal cord of the mice, and also slowed their breathing.
“The effect was very remarkable.
It really seems to be a very strong immune-modulating agent, and it is a very effective agent,” he explained.”
This compound may also have a role in treating amycotrophic lateral syndrome and other forms or neurodegenerative diseases, but we do not know whether it would work in humans.””
It is not clear whether it will be able to treat ALS, but there is some evidence that it may have some effect on amyotaxis, which we suspect is a mechanism for controlling inflammation.”
The compound was tested in two strains of mice, but Professor Sacher said the results showed the compound worked in both strains of the animals.
“It appears that this compound may be effective against amyotopias in other diseases as well,” he added.
“So, it could potentially have some role in some of these other conditions that affect the nervous system.”
In our experiments, the compound inhibited the inflammation-induced responses, and at the same time, the mice were not affected by the inflammatory responses.
“The study was funded by the National Institutes of Health, and the research was conducted in collaboration with Drs.
Daniel S. Fiszman, Miki N. Nakano and Shai Siegel at the UC Berkeley Department of Neuroscience.
It is published in Science Adv.