Researchers from across the globe gathered at the World Stem Cell Summit Monday in Madison, stressing the need for further clinical trials funded by the federal government.
Mice and fruit flies have been used extensively in preliminary studies to make sure the new form of regenitive medicine is safe for human trials.
Larry Goldstein, professor of cellular medicine at the University of California-San Diego, reminded the diverse group of attendees that Alzheimer’s or Lou Gehrig’s disease in mice is not identical to the disease in humans.
“We have studied for many years animal versions of Alzheimer’s disease, but the problem at the end of the day is that humans are not just big mice,” Goldstein said.
There are serious concerns about the safety of the therapy in humans, including a possible risk of cancer or immune system response.
“The first clinical trials are not meant to cure the patient,” said John Wagner, University of Minnesota pediatrics professor. “The first clinical trials are directed toward verifying the safety of the therapy we’re employing.”
In clinical trials, researchers will have to determine the maximum dose of cells that can be tolerated, where and how they ought to be delivered and any adverse side affects.
Even before that, some more trials with animals will need to be done, Wagner said.
“We don’t have the best animal models,” Wagner said. “[We need] animal models that will reliably provide what kind of risk there will be.”
Presenters also discussed the importance of using both embryonic stem cells and induced pluripotent stem cells, or iPS cells.
But University of Wisconsin anatomy professor James Thomson, who was the first researcher to isolate embryonic stem cells in 1998, said though cell replacement therapy may be the “sexy” result of research, it may not be the primary result of it.
Thomson said he believes if blood cells can be made cheap enough, they will one day replace transfusion as the primary source of blood replacement.
He also explained that clinical research could lead to the discovery of what causes many diseases.
“I wouldn’t be extremely shocked if over my scientific career, scientists find out what causes Parkinson’s to work at preventing it,” Thomson said.
Participants in the panel also discussed possible roadblocks to clinical research. Goldstein said the answer to that question is always money.
There has been a decline in federal research funding across the board. Goldstein called the lack of funding “absolutely disastrous” and said despite tough economic times the funding needs to be continued.
“The temptation is to say ‘Well, we should just cut spending on everything,'” Goldstein said. “But the fact is that as a proportion of what we invest our national capital in, scientific research is a tiny fraction, yet it reaps enormous benefits and is in fact an investment.”
Thomson said, “Scientists are notoriously bad for producing timelines.”
“We just need to roll up our sleeves and do a great deal of work here,” Thomson said. “It’s not going to happen overnight.”
The summit continues today with a panel featuring former Gov. Tommy Thompson.