The decades-long dream of treating debilitating injury and illness with stem cells is quickly becoming reality.
The world's first test on patients of a treatment using human embryonic stem cells has cleared a year of safety trials with no adverse effects, according to Geron Corp.
Meanwhile, a second clinical trial of another stem-cell treatment has begun in Switzerland.
Both treatments target spinal cord injuries, and both were developed by scientists at UC Irvine.
Stem cells have the potential to develop into a variety of cell types within the body. Some are derived from embryos, stirring controversy, but their promise is undeniable: allowing the paralyzed to walk, or reversing degenerative diseases such as Alzheimer's or multiple sclerosis.
Hans Keirstead, whose treatment for acute spinal cord injuries became the world's first clinical trial using human embryonic stem cells, is pushing ahead to develop new treatments, and to increase the number of people who could be helped by those already being tested.
Q. Are you doing further work on the stem-cell treatment for acute spinal-cord injury?
A. I'm doing a lot of work in order to expand the patient population that might benefit from the treatment. I recently published a paper (about using) the treatment around the neck. Before we showed only that it worked down near the stomach, the thoracic region. We have to show that it works in rats before the FDA will allow us to go into humans. I published a paper showing it works just as well in the cervical (neck) region, as in the thoracic. I'm pretty happy about that.
Q. And you have new research efforts underway?
A. I'm working on two new projects, and I'm really excited about them. Both are on chronic (long term) spinal-cord injuries. One involves getting rid of the scar that forms after an injury. If you get rid of the scar, that would make an old injury a young injury again. Therefore, you could apply all these new technologies that are emerging to treat acute (short term) spinal cord injury.
It looks like we've been able to do it in a dish. We've grown spinal cord cells in a dish, we've made them become scarred, and we're able to reverse that scar in a dish. Now we move on to rats.
One thing that is very interesting: if it's successful, this treatment also could be amenable to treatment of multiple sclerosis, which is a brain auto-immune disease, where one's own immune system attacks the brain, and causes scarring. We would use the same approach to reverse the scarring, remove the scarring. This is one of the greatest challenges in central nervous system regeneration research. No one has been able to come close to doing it. It remains untouched.
Q. Where does the scarring occurr in M.S.?
A. Multiple places, various areas. Random areas. It would also be advantageous in the treatment of brain injuries and stroke, which also accumulate scars. The spinal cord tissue itself scars.
Q. You've also worked on a treatment for spinal muscular atrophy, which typically kills children in their first years of life.
A. It's beyond the university now (an Irvine company called California Stem Cell is now working to prepare the treatment for its first clinical trial). The university is a place for innovation and invention; companies are a place for commercial development.
Q. What is the second research initiative?
A. The second one is a combination of what I think are two of the greatest developments in regeneration research. Oswald Steward at the UCI Reeve-Irvine Research Center recently took a technique, invented at Harvard, and moved it into spinal cord injury. He showed that he can get the spinal cord to grow better than it's ever been able to grow before. How he did it is how everyone in the world is doing it using transgenic animals. They alter the genome of the entire animal in order to get it done.
Clearly, you can't do that in a human. What I'm doing is combining that technique into stem cells. You can, in effect, supercharge stem cell growth, then put them into an animal -- soon, hopefully, with a human.
We take this trick of supercharging the growth properties of the cell. Instead of applying it to the entire animal, we're applying it only to the stem cells. I describe it to students as 'stem cells on steroids.' Genetic manipulation causes the stem cells to grow extraordinarily well.
It would mean that transplanted cells would be able to grow quicker, and therefore also avoid scarring.
Spinal cord injury is followed by a degenerative cascade, which involves disruption of blood and the death of many spinal cord cells, which releases toxins.
Transplanted cells are prone to those same killers. By supercharging them, we may be able to allow them to integrate into the spinal cord, and survive that degenerative cascade. They may be able to knit the spinal cord together over greater distances.
This would be a fundamental leap for the field, and could be applied to every transplant technique within the nervous system.