Experimental stroke drug also shows promise for people with Lou Gehrig's disease

March 3, 2014
A fluorescent image shows cells of the neurovascular unit in the mouse spinal cord, which consists of motor neurons (green) and blood vessels containing pericytes (red) and endothelial cells (blue). Winkler et al. (2014) show that disruption of blood vessels accelerates injury of motor neurons in amyotrophic lateral sclerosis. Credit: Ethan A. Winkler and Berislav V. Zlokovic/University of Southern California

Keck School of Medicine of USC neuroscientists have unlocked a piece of the puzzle in the fight against Lou Gehrig's disease, a debilitating neurological disorder that robs people of their motor skills. Their findings appear in the March 3, 2014, online edition of the Proceedings of the National Academy of Sciences of the United States of America, the official scientific journal of the U.S. National Academy of Sciences.

"We know that both people and transgenic rodents afflicted with this disease develop spontaneous breakdown of the blood-spinal cord barrier, but how these microscopic lesions affect the development of the disease has been unclear," said Berislav V. Zlokovic, M.D., Ph.D., the study's principal investigator and director of the Zilkha Neurogenetic Institute at USC. "In this study, we show that early motor neuron dysfunction related to the disease in mice is proportional to the degree of damage to the blood-spinal cord barrier and that restoring the integrity of the barrier delays motor neuron degeneration. We are hopeful that we can apply these findings to the corresponding disease mechanism in people. "

In this study, Zlokovic and colleagues found that an experimental drug now being studied in human stroke patients appears to protect the blood-spinal cord barrier's integrity in mice and delay motor neuron impairment and degeneration. The drug, an activated protein C analog called 3K3A-APC, was developed by Zlokovic's start-up biotechnology company, ZZ Biotech.

Lou Gehrig's disease, also called amyotrophic lateral sclerosis, or ALS, attacks , which are cells that control the muscles. The progressive degeneration of the motor neurons in ALS eventually leads to paralysis and difficulty breathing, eating and swallowing.

According to The ALS Association, approximately 15 people in the United States are diagnosed with ALS every day. It is estimated that as many as 30,000 Americans live with the disease. Most people who develop ALS are between the ages of 40 and 70, with an average age of 55 upon diagnosis. Life expectancy of an ALS patient averages about two to five years from the onset of symptoms.

ALS's causes are not completely understood, and no cure has yet been found. Only one Food and Drug Administration-approved drug called riluzole has been shown to prolong life by two to three months. There are, however, devices and therapies that can manage the symptoms of the disease to help people maintain as much independence as possible and prolong survival.

Explore further: Toxin from brain cells triggers neuron loss in human ALS model

More information: Blood–spinal cord barrier disruption contributes to early motor-neuron degeneration in ALS-model mice, Winkler, E. A., Sengillo, J. D., Sagare, A. P., Zhao, Z., Ma, Q., Zuniga, E., & Zlokovic, B. V. (2014). PNAS Early Edition, 1-8. Published online March 3, 2014; DOI: 10.1073/pnas.1401595111

Related Stories

Toxin from brain cells triggers neuron loss in human ALS model

February 6, 2014
In most cases of amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, a toxin released by cells that normally nurture neurons in the brain and spinal cord can trigger loss of the nerve cells affected in the disease, ...

Disease progression halted in rat model of Lou Gehrig's disease

December 12, 2011
Amyotrophic lateral sclerosis (ALS; also known as Lou Gehrig's disease) is an incurable adult neurodegenerative disorder that progresses to paralysis and death. Genetic mutations are the cause of disease in 5% of patients ...

Researchers discover how ALS spreads

February 18, 2014
A study led by University of British Columbia and Vancouver Coastal Health Research Institute researchers has revealed how the fatal neurodegenerative disease amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's ...

Potential new drug target in Lou Gehrig's disease

November 14, 2011
Two proteins conspire to promote a lethal neurological disease, according to a study published online this week in the Journal of Experimental Medicine.

A new development in the relief of spasms related to amyotrophic lateral sclerosis

November 6, 2012
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with an occurrence rate in France similar to multiple sclerosis (two to three new cases per year for every 100,000 residents). It has a specific affect on ...

Experimental drug reduces brain damage, eliminates brain hemorrhaging in rodents afflicted by stroke

October 25, 2013
An experimental drug called 3K3A-APC appears to reduce brain damage, eliminate brain hemorrhaging and improve motor skills in older stroke-afflicted mice and stroke-afflicted rats with comorbid conditions such as hypertension, ...

Recommended for you

What if consciousness is not what drives the human mind?

November 22, 2017
Everyone knows what it feels like to have consciousness: it's that self-evident sense of personal awareness, which gives us a feeling of ownership and control over the thoughts, emotions and experiences that we have every ...

Team constructs whole-brain map of electrical connections key to forming memories

November 22, 2017
A team of neuroscientists at the University of Pennsylvania has constructed the first whole-brain map of electrical connectivity in the brain based on data from nearly 300 neurosurgical patients with electrodes implanted ...

To forget or to remember? Memory depends on subtle brain signals, scientists find

November 22, 2017
The fragrance of hot pumpkin pie can bring back pleasant memories of holidays past, while the scent of an antiseptic hospital room may cause a shudder. The power of odors to activate memories both pleasing and aversive exists ...

Pitch imperfect? How the brain decodes pitch may improve cochlear implants

November 22, 2017
Picture yourself with a friend in a crowded restaurant. The din of other diners, the clattering of dishes, the muffled notes of background music, the voice of your friend, not to mention your own – all compete for your ...

Now you like it, now you don't: Brain stimulation can change how much we enjoy and value music

November 20, 2017
Enjoyment of music is considered a subjective experience; what one person finds gratifying, another may find irritating. Music theorists have long emphasized that although musical taste is relative, our enjoyment of music, ...

MRI uncovers brain abnormalities in people with depression and anxiety

November 20, 2017
Researchers using MRI have discovered a common pattern of structural abnormalities in the brains of people with depression and social anxiety, according to a study presented being next week at the annual meeting of the Radiological ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.