By David Baugher, special to the Beacon
7:01 am on Fri, 10.19.12
When it comes to stem-cell therapies, Dr. Marie Csete is looking to the future.
“What are the best things? Where are the leading edges?” she said. “I’m going to try to make a little bit of a prediction about where the field is going.”
In a period of highly charged debate over the controversial topic, those leading edges can sometimes be hard to see. But Missouri Cures Education Foundation believes Csete, who will speak later this month at the Donald Danforth Plant Science Center, is just the person to point them out.
“She’s really the whole package and can talk from every angle,” said Dena Ladd, executive director of the Missouri Cures Education Foundation, which is sponsoring the talk as part of its speakers’ series. “I was also thrilled to finally bring in a female researcher because all of the researchers at our other events have been men.”
The noted researcher directs cellular therapy and regenerative medicine activities at AABB, a Bethesda, Md., nonprofit formerly known as the American Association of Blood Banks, which represents individuals and institutions involved in transfusion medicine and cellular therapies. A member of the adjunct faculty on the liver transplant team at the University of California-San Diego, she is a former chief scientific officer at the California Institute for Regenerative Medicine and has conducted extensive stem-cell research as founding director of the Emory University/Georgia Institute of Technology Human Embryonic Stem Cell Core facility.
An active member of the International Society for Stem Cell Research, Csete said that future challenges in the maturing area of research will center on issues of standardization, which, while they might sound mundane, are vital to keep front and center if meaningful progress is to be made.
“It sounds really dry, but it’s incredibly important,” she said. “When you give some stories about cell therapies that were slowed down by inadequate standardization in the way people were doing things and talking to one another about how they do things, people will understand the importance of this. I’m particularly interested in trying to make the point that without standardization at this stage in this growing field, the kinds of data we’ll get from clinical trials will be really uninterpretable.”
That’s a big fear for scientists who need to build on one another’s work but may find that impossible without uniform methods of doing their work. Even fairly minor factors can make a huge difference. Csete recalled the story of researchers who received FDA approval to move forward with promising results only to find problems crop up unexpectedly.
“As soon as they did their clinical trial they found out that they were having a very safe transplant procedure but they weren’t seeing the efficacy that happened in animals,” she said. “The look back on how the cells were handled by the scientists who did the animal work versus the scientists who were preparing the cells for clinical trials made them realize that there was a stage at which the temperature was different in the way in which the cells were handled.”
Those differences can come up a lot in research as well as in commercialization and manufacturing where they can present an equally daunting issue.
“I’m also going to be talking about the need for comparative efficacy studies which can be difficult to sell to the community,” she said. “Once there is academic status and company money involved in something, there is very little incentive for people to compare their products to somebody else’s.”
Csete will address some advancements in the field including a corneal resurfacing procedure that has shown great promise in helping those with chemical burns to the eye, a source of both blindness and constant discomfort.
“There’s really quite a remarkable success rate in these recently published articles from Italy which showed that about 75 percent of the patients had permanent restoration of the corneal surface with vision restoration and relief of their pain,” she said. “If you had a result like that in cancer biology you would be completely going nuts.”
There are also other success stories including a new procedure from one researcher that can engineer a trachea from scratch using a scaffold, a surface on which cells can grow.
“He created a scaffold and seeded the patient’s own airway cells on it and then transplanted it,” she said. “Over time, with the experience they’d gotten clinically, they realized that they didn’t need to seed it or do elaborate machinations to get the construct to be vascularized. They were able to just put in a completely engineered scaffold and the patient’s own cells would eventually repopulate.”
She feels the controversy surrounding stem cells is not what it once was, particularly as states begin to back research on a wider basis.
However, that doesn’t mean all challenges are at an end. Money remains a concern even where legal restrictions aren’t a problem.
“I think people have figured out a way around (the politics) but the funding issue is fundamental and not only for stem cell biology,” she said. “It’s for all medical research. I think we’ve reached a point where traditional drug discovery is not yielding as many therapeutic products as it once did. When you are completely starting from scratch in new fields like regenerative medicine, it’s an incredibly expensive process.”
Although all of the therapies she will discuss are derived from adult stem cells, Csete still believes all avenues of stem-cell work need to be explored, even with the development of the adult and induced pleuripotent varieties that do not require the use of a fertilized egg.
“Even if human embryonic stem cells fall by the wayside as a source of cells for therapy and are replaced by other things,” she said, “what scientists really understand that the lay public doesn’t, is that in order to understand one stem-cell type you have to be doing comparable research in all the other types. All the information feeds in.”