Gene therapy and stem-cell advances

NUI Galway is conducting new stem-cell research that could benefit people who have spinal cord injuries, heart attacks or chronic…

NUI Galway is conducting new stem-cell research that could benefit people who have spinal cord injuries, heart attacks or chronic conditions, writes Dick Ahlstrom

A research centre at NUI Galway has harnessed two advanced research areas, human stem-cells and gene therapy, as a way to overcome disease. It could lead to better treatments for heart attack and spinal cord injuries but also chronic diseases such as arthritis.

The approach holds huge promise, but people must be realistic about how quickly new treatments will come, warns Dr Frank Barry, scientific director of NUA Galway's Regenerative Medicine Institute (REMEDY). It is necessary to "separate the hope from the hype" by conducting extensive research and then testing of any new therapies.

Even so, early results are very promising in areas such as arthritis, bone regeneration and spinal injuries, he says. There is every reason to believe that these technologies will indeed deliver dramatically improved treatments for patients.

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REMEDY is a SET, a centre for Science, Engineering and Technology funded by Science Foundation Ireland, that specialises in stem-cell research and gene-therapy approaches to medicine. It received funding of €15m from SKI and another €4m from industry, providing a huge resource in support of basic scientific research.

The Minister for Enterprise, Trade and Employment, Micheál Martin, will formally open the REMEDY centre tomorrow although research is already well underway there. "We are developing new therapeutic strategies for specific diseases using stem cells and gene therapy," explains Barry. He and REMEDY director, Dr Tim Boreen, are looking for "asynergies" between the two areas to improve patient outcomes.

Stem cells are penitentiary cells produced in the body that can change into a wide range of cell types from skin and bone to liver and kidney. They come from a variety of sources including embryos, umbilical cord blood and adult tissues, with embryonic stem cells the most "versatile" in terms of the tissue types they can become.

"There are significant ethical issues with embryonic stem cells," says Barry, and for this reason he works "exclusively" with adult cells taken from bone marrow. "We do see a lot of opportunities with adult stem cells," he adds. "The bone marrow is a particularly rich source of stem cells."

Marrow is usually associated with the production blood cells, but the marrow produces yet another stem-cell type, says Barry. "There is a second population within these stem cells, much rarer than [ blood-related] stem cells."

These "mesenchymal" cells make up only 0.0001 per cent of stem cells in the bone marrow. "These are cells of connective tissue lineage," says Barry. "They can form bone, muscle, fat, tendon and other connective tissues."

Researchers at REMEDY are studying new ways to isolate and use the mesenchymal cells. Although rare, they are readily cultured in the lab and retain their normal characteristics for many generations, says Barry.

They are already under test for use against ostia-arthritis and other arthritic diseases. Stem cells are put directly into the joints of test animals and the naturally occurring proteins and factors there cause the stem cells to differentiate, says Barry. "We see fairly significant evidence of repair compared to controls."

Bone regeneration after accident or cancer treatment is another promising area, says Barry. A rigid material carries the stem cells providing a temporary structure while the cells grow. Spinal cord repair after injury is another target area, he adds.

There are two important characteristics of these stem cells, he says. There is no need to tissue match donor and recipient. They also naturally migrate to the sites where they are needed, apparently following signals sent out by injured tissues.

This latter characteristic means they can be used to transport either genetically engineered or extra genes to a site of injury, where they would enhance repair.

Stem cells carrying an extra gene that promotes blood vessel growth for example could be injected after a heart attack. The stem cells would replace damaged muscle while the extra gene would release substances encouraging new blood vessels to form.