All too often we hear heartbreaking stories of a child needing a kidney or liver transplant, but neither parents match.
Transplanting an organ that is not closely matched to a recipient's blood type, antibodies and other key markers generally means that the receiver of the donated organ will immediately begin to reject it. The human immune system is a master at doing its primary job — rejecting anything in the body that it does not recognize.
However, science uncovered that a small percentage of humans have a type of human leukocyte antigen (HLA) that allows their tissues to be transplanted more easily. Researchers have found that these "partial matches" are accepted by recipient bodies with less chance of rejection.
Now there is talk about creating a biobank of these cells from HLA "matching superdonors." And one company is poised to push forward with the production of these "superdonor" cells.
This is possible thanks to induced pluripotent stem cells (iPSCs), which were first discovered in 2007. The United States National Institutes of Health (NIH) explains that iPSCs are "adult cells that have been genetically reprogrammed to an embryonic stem cell-like state by being forced to express genes and factors important for maintaining the defining properties of embryonic stem cells."
According to the NIH, using iPSCs is not easy, as "viruses are currently used to introduce the reprogramming factors into adult cells, and this process must be carefully controlled and tested before the technique can lead to a useful treatment for humans."
But Cellular Dynamics International (CDI) may have the solution. According to Forbes Magazine, CDI announced Feb. 9 that it "has obtained and begun manufacturing" cells from two superdonor lines that are thought to be "genetically compatible" with as much as 19 percent of the population in the United States.
CDI plans to make the two lines into uncomplicated, efficient replacement cell therapies. Eventually, CDI believes that the two line of cells will lead to cells harvested from 184 lines of superdonors, At that point, 95 percent of the U.S. population will be protected from immune rejection.
"Manufacturing human cells is hard," CDI President Robert Palay said. "It's very hard to do in the quality and quantity we do. You have to understand the conditions and keep them under control. The heat, light, moisture, media, reagents all have to be right. What we do that no one else does is manufacture cells in quality and volume that's really unprecedented."
CDI's main focus is on making cells for pharmaceutical companies and other research organizations. They use these cells to test for toxicity and other compounds in new drugs. The company also has grants for the manufacture of stem cells "derived from donor tissue samples and amassed in biobanks for researchers worldwide to use."
"Our model is to make great cells and put them in the hands of great researchers to do great science," Palay said in a November 2014 interview with Xconomy.
Up until now, CDI has only been the provider of cells for use in research. Although the company is conservative and prefers to support its research partners, it has taken a small step toward creating a therapy with its first development deal that led to the grants. The question now is whether it will become a major innovator in "regenerative medicine."
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"On this frontier, it's not surprising CDI is moving deliberately," writes Xconomy's Jeff Engel. But he notes that "there are plenty of regulatory hurdles."
Nevertheless, experts such as Chuck Murry, a stem cell biologist at the University of Washington and Sir Ian Wilmut, believe that superdonors are one of the most exciting aspects of stem cell biology and that CDI is poised to become great in the field.
Starting in 2014, Palay had begun saying that CDI would soon have its first deal with a partner for the co-development of a therapy. In late October, it was announced that CDI and the National Eye Institute, part of the National Institutes of Health signed a $1.2 million contract for the development of a therapy for early-stage treatment for dry age-related macular degeneration.
