THE ODDS (Debbie Does ALS)


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Sepsis treatment 'may slow Lou Gehrig's disease'

WASHINGTON — An enzyme similar to that used to treat serious sepsis could be used to slow the progress of the motor neuron disorder known as Lou Gehrig's disease, according to new medical research.

A study, released late Monday, found that mice suffering from the disease experienced slower cell death when treated with the enzyme, called activated Protein C or APC.

The protein also extended by about 25 percent the life of mice suffering from a particularly aggressive form of the disease, which is formally known as amyotrophic lateral sclerosis, according to the study.

The report, which was published in the online edition of the Journal of Clinical Investigation, was produced by researchers from a group of US universities and a biotech startup company in New York state.

Among other promising developments, the study found the compound extended the amount of time that mice suffering from the disease were able to function normally, despite showing some symptoms of the disorder, which is incurable and almost always fatal.

The protein also slowed muscle wasting associated with the illness, which is named after the star US baseball player Lou Gehrig, who died in 1941 after suffering from the disease.

Though additional research will be needed before the treatment can be used in humans, the researchers said they were encouraged that the breakthrough involves a compound that is already used to treat patients with sepsis and has been proven safe.

They hope clinical trials on patients could begin within five years.

"The success of this research project has been very gratifying, and we are hopeful that a form of APC will ultimately be useful as a treatment for this disease," said Berislav Zlokovic, a professor of Neurosurgery and Neurology at the University of Rochester.

The researchers found that APC may also be useful in mitigating some of the secondary effects of the illness, including a mutation that weakens barriers between blood and the spinal cord, allowing toxic substances to enter the spinal cord.

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