Potential drug candidate for slowing Lou Gehrig's disease progression identified
20 Oct 2009, 1541 Hrs

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Washington, Oct 20 A chemical cousin of a drug currently used to treat sepsis has been found to drastically reduce the spread of amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease.

The researchers focussed their study on the use of a form of an enzyme known as activated protein C, or APC, to slow the cell death that occurs in ALS.

They could successfully extend the lifespan of mice with an aggressive form of the disease significantly, by about 25 percent.

The compound also extended the length of time that the mice were able to function well despite showing some symptoms of the disease, and it reduced the pace of muscle wasting that is a hallmark of ALS.

The research involved mice with a mutation in a gene known as superoxide dismutase 1 (SOD1), which plays an important role keeping cells safe from damaging molecules known as free radicals.

While the cause of most cases of ALS is unknown, scientists do know that SOD1 plays a role in approximately 3 or 4 percent of cases - providing an opportunity to study the disease's initial steps, which occur long before key nerve cells appear sick or die.

In addition, recent studies have suggested that the accumulation of mutant forms of SOD1 is linked to most sporadic cases of ALS.

The team has shown that APC dramatically lessens the activity of the SOD1 mutation.

This protects neurons that are under assault by blocking the synthesis of aberrant forms of the molecule in motor neurons and other cells in the spinal cord.

In addition to reduced SOD1 activity, the flow of dangerous byproducts of hemoglobin into the spinal cord was eliminated by APC, saving neurons.

The study appears in Journal of Clinical Investigation. (ANI)

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