Different from mice
Willingham, a pathologist, says, "This kind of transfer of white cells in mice is somewhat of a different kind of strategy than it is in people, because in the mice we use in the laboratory they're very closely matched genetically, and so as a result, when you transfer any cells from mouse to mouse, it's basically like transferring cells to an identical twin. So the result we saw in the mice when we did that is that anti-cancer activity survived for a very long time, but that's not going to happen in human patients because we're all genetically different from each other in a great extent, so those cells will not survive."
But that's a good thing
"The observations in the mice suggest that this activity that kills cancer cells occurs very quickly, so these transferred cells only have to survive for a few days, and in fact they do survive for several days, in order to be effective against the cancer," Willingham says. "Consequently if they're not still there, say, a week after they're given, they can't produce the side effects that one sees when you have those kind of cells surviving for a long period of time. So as a result it's a safe strategy."
He adds that there might be other unknown differences between mice and people that could make the therapy fail. "This is an experimental study," he emphasizes.
Mouse experiments repeated
Meanwhile, the mouse studies continue in the search for an understanding of the mechanism. Cui has offered to share his cancer-proof mice with other labs so results could be independently tested.
A new study confirming the mice's cancer resistance is now soon to be published. Klaus Reineck, a clinical immunologist at the Blood Bank, Copenhagen University Hospital, in collaboration with professor Jann Hau and his group at the Department of Experimental Medicine, University of Copenhagen, Denmark, confirm they have a paper in press in the journal In Vivo, replicating the initial Wake Forest experiments.
"The findings independently lend support to Dr. Cui's discovery" that the mice "are indeed resistant" to cancer cells, Reineck wrote in an email. "The findings, however, should be further substantiated. We are very grateful to cooperate with Dr. Cui on this exciting mouse model."
Clinical trial details
The researchers plan to begin by recruiting patients who have insurance, as North Carolina is one of the states that has a law requiring insurance providers to pay for clinical trials.
Willingham says this trial will be considered a Phase II trial by the FDA and the Independent Review Board (IRB) that will oversee the trial, so it falls under the state law.
As for the donor screening part of the study, he says the funding has not been finalized.
They hope some private funders will step up to the plate, willing to support a potentially life-saving innovation.
"Usually, science doesn't like innovation at all, because it jumps over the incremental steps that science can't go ahead without," says Cui. "Often, scientists don't like our studies because we don't understand the gene or how it works.
"But, pretty much the tradition of medicine is about innovation. If it works, we don't really care about how," he says.
Wake Forest plans to launch a new website with more information about the new trial, called Leukocyte Infusion Therapy, or "LIFT".
You can also find this trial here.