more regenerative medicine treatments get to market faster. “We have a lot of cell types that have a lot of therapeutic benefit. There’s no company that could advance all of them,” he says. “But if we partner with a lot of companies, a lot of researchers, lots of therapies can get advanced simultaneously.”
All of this is a moot point, of course, if CDI and its partners can’t make cell therapies that work.
Palay is confident the cells will be safe for humans. He says CDI’s method for reprogramming the donor cells into a pluripotent state doesn’t introduce foreign genetic material into the genome, which could potentially cause abnormalities. Also, the therapeutic cells implanted into patients would have been derived from the patient’s own blood or skin tissue, which CDI says decreases the likelihood of the body rejecting the new cells.
Both of those points “are widely considered important hurdles to clear when envisioning clinical applications involving human iPS cells,” so CDI is making good moves to try to alleviate those concerns, says Harvard stem cell expert Lensch. Nevertheless, there are other potential safety complications that CDI will need to avoid, he adds.
For one, after the stem cells have been coaxed into retinal cells, CDI must make sure there are no cells left over from the previous states, Lensch says.
Such “hitchhiker” cells “could be problematic, as they may lead to tumor formation,” Lensch says. “Such tumors may well be benign, but would present a situation that everyone involved would rather avoid altogether.”
Cell purity, in other words, is crucial. CDI has quality assurance checks built into its manufacturing process to ensure cell purity, Palay says. The company intends to work with NEI to hone the manufacturing procedures for the retinal cells to meet FDA good manufacturing practice (GMP) standards.
That’s important, Lensch says, because GMP lessens the chance of cells picking up impurities from the medium—that is, the soup of nutrients—in which they’re grown. And impurities could trigger immune reactions in patients receiving the cell transplants.
Proving safety is one thing. It’ll also be crucial to find the best method for delivering the therapeutic cells to the injury site and ensuring they integrate seamlessly with the body, Palay says.
“The issue is, is it going to work?” Palay says. “Does it integrate, does it function like your cell?”
Like any emerging field, there are a host of “interesting challenges,” Hood says. “But I think CDI has the tools to attack these problems as well as any of the stem cell companies and people out there. They understand this process maybe better than anybody else does.”
