Thursday, April 21, 2005

Stem Cell Therapy - An ALS Treatment?

The Associated Press has the following story about the use of Stem Cells to possibly treat ALS - Lou Gehrig's Disease:

A University of Wisconsin-Madison researcher said he would ask federal regulators Friday to approve the first clinical trial injecting special stem cells into the spinal cords of people with the degenerative nerve ailment called Lou Gehrig's disease.

The trial would test whether a technique anatomy professor Clive Svendsen has pioneered on rats afflicted with the disease is safe to use on people. If successful, Svendsen said a much larger clinical trial aimed at treating the disease could be under way in two or three years.

About 30,000 Americans currently have the disease, which gradually kills brain cells that control muscle movement. The disease, formally known as amyotrophic lateral sclerosis, or ALS, typically can lead to death in a few years and has virtually no treatment.

Svendsen and his colleagues are asking the U.S. Food and Drug Administration for approval to bypass testing the technique on primates, typically the next step after rats, and to go straight to humans. The trial would involve five ALS patients treated by neurosurgeons at the Cleveland Clinic.

The trial would build on research Svendsen published this week in the journal Human Gene Therapy, which found that injecting certain types of stem cells into the spinal cords of rats could help stave off the disease and potentially prolong their lives.

Svendsen and his colleagues said the study was the first to show that the stem cells carrying a protein that fights ALS could flourish after being injected into their bodies.

The ALS Association, which is spending millions of dollars to fund Svendsen and other researchers rushing to find a cure, called the research encouraging.

"It is so exciting to see how rapidly ideas are moving from the laboratory into potential clinical applications through strong collaborations with leading investigators," the association's science director, Lucie Bruijn, said in a statement Thursday.

While noting the promise of his research, Svendsen sought to play down expectations, saying a cure of the debilitating disease was still years away.

"We're not going to cure ALS in the first clinical trial," Svendsen said Thursday at a forum on bioethics in Madison. "We're going to tell the patients that as well."

The research does not involve human embryonic stem cells, the blank-slate cells derived from human embryos that can be molded into any type of tissue cell in the body.

Researchers are instead using neural progenitor cells in fetal brain tissue, which are in the early stages of brain development. Those cells - derived from miscarried fetuses - are obtained through the National Institutes of Health.

Svendsen's research team first created stem cells that pumped the disease-fighting protein, and then had to find the exact location in the rat's spinal cord to inject them to fight ALS. The latter step took months of trial and error but may help surgeons deliver the treatment to humans.

Svendsen acknowledged the clinical trial proposal was risky. If the research on humans fails or is deemed unsafe, it could set back the field for years.

But he said waiting to unleash a potential cure for the lethal disease was unacceptable and the research has been safe so far.

"We're hoping the FDA doesn't require a lot more animal work," he said.

Having seen this devastating disease ravage at least three men I have known, I can only pray that these trials will be successful.

Note: this treatment invovles Stem-Cells but not Embryonic Stem-Cells from Aborted fetuses.

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