Stem Cells Likely to Help Genetic Disorders First

Jan. 6 -- TUESDAY, Jan. 5 (HealthDay News) -- With new rules in place that lifted restrictions on federal funding of human embryonic stem cell research, health-care advocates are looking down the line and wondering when the first medical advances based on stem cells might occur.

That will take some years yet, according to experts in the field. And when breakthroughs do come, they might not be what most people think of when they envision potential stem cell therapies.

The best-known potential use of human embryonic stem cells involves cell-based therapy, where stem cells are used to repair damaged heart muscle or severed spinal cords.

But the first advances are more likely to come from stem cells used to research genetic diseases and develop therapies and cures for those diseases, said Story Landis, director of the U.S. National Institute of Neurological Disorders and Stroke and chairman of a stem cell task force at the National Institutes of Health.

"I am sure we will see advances from those kind of studies before we will see advances in cell replacement therapy," Landis said. "They (stem cells) aren't just for cell replacement. They are also for understanding disease processes and developing better therapies."

Stem cell research under President Bush was limited to 21 lines of human embryonic stem cells. However, Landis said that none of those cell lines contained mutations that could lead to genetic diseases or disorders.

By opening up research to more stem cell lines, scientists now can review lines that contain those mutations and develop better therapies for genetic diseases, she said.

"Instead of having 21 stem cell lines, none of which had disease-causing mutations, we could have hundreds of lines, and any of those will contain mutations that cause disease," Landis said. "It's like having vanilla ice cream versus having 27 flavors."

As an example, Landis cited human embryonic stem cells with a mutated tendency to develop Huntington's disease, a neurodegenerative disorder. Researchers could develop neuron cells from the stem cells in a laboratory and then test different medications to see which drugs might prevent the disease from killing the neurons, she explained.