to use in-house. If iPierian plays its cards right, the Big Pharma cash, some more venture capital, and support from the California Institute for Regenerative Medicine and private disease foundation, will provide enough cash for this 50-person operation to discover its own drugs that it can take all the way through to FDA approval and the marketplace, Walker says.
Before settling in on what iPierian wanted to do, it was useful to find out what it didn’t want to do. The company chose not to try to make stem cell-based therapies, at least not in the beginning. One problem is that viruses are needed to induce the adult cells into a pluripotent state, which isn’t really something you want to inject in people as a therapy. Then there’s bound to be battles over who really owns the intellectual property. And blazing the first regulatory trail through the FDA is going to be tough.
One idea was that iPierian could create induced stem cells, and differentiate them into banks of heart cells, or liver cells. Those cells could be particularly useful to big drugmakers, so that they could test experimental compounds to see if they would likely be too toxic for the heart or liver—a couple places that often trip up drug candidates after years of work are wasted. Madison, WI-based Cellular Dynamics, co-founded by stem cell researcher James Thomson, has chosen to go this route.
San Diego-based Fate Therapeutics, probably the best known stem cell startup in the country, has a lead drug candidate in clinical trials that essentially is a conventional molecule that seeks to induce adult stem cells in the body. One of its scientific co-founders said he has found a way to use cheap, simple small molecules to induce cells into a pluripotent state, which is thought to be a key step in the industrialization of stem cells for drug discovery, Walker says.
iPierian says it doesn’t see Fate as an obvious rival, although it certainly competes for venture money and attention. Although Walker didn’t say it in quite so many words, he clearly doubts that Fate can do as much as people think.
“When you read papers about new methods development, there are issues like how long will it take to actually see the re-programming? What types of throughput did they get? How resilient were these lines for future manipulation and differentiation?” Walker says. “That becomes a very important consideration. How robust are the lines actually created.”
Those, anyway, are some of the questions Big Pharma companies ask when they mull over whether to jump on the stem cell wagon.
iPierian hopes that by gathering all these cells, with the genetics that are unique to each individual, they will provide a more reliable guide in the lab for drug testing. Neurodegenerative diseases seemed like a perfect place to start, because it’s sort of hard to get
