Stem cells captivated the broad public imagination more than a decade ago, and despite all the hyperbole about creating Lazarus-like regenerative medicines, nobody has built a powerhouse business on this technology. Now a little company in South San Francisco, iPierian has a vision for using stem cells in a way that may not generate magazine covers, but which could enable it to do a better job of discovering more conventional drugs.
That’s the idea anyway, and it’s being pushed by some big-name scientists and venture capitalists who are gathering today in San Francisco for an international stem cell meeting. The story actually began two companies. One, in Boston, was called Pierian, co-founded by George Daley, Doug Melton and Lee Rubin, a trio of stars from the Harvard Stem Cell Institute. The other, iZumi Bio, in Mountain View, CA, had the entrepreneurial leadership of Corey Goodman as chairman, and John Walker, a longtime director of stem cell pioneer Geron, as CEO. The merged operation boasts a blue chip crew of backers—Kleiner Perkins Caufield & Byers, Highland Capital, MPM Capital, and FinTech Global Capital, who have pumped in a combined $31.5 million.
Impressive as the names are, their ambition isn’t what most people think, to create some Lazarus-like regenerative medicine. It’s more about using the power of stem cells to discover new drugs, so that iPierian can find treatments that no one ever could before because they were constrained by limited tools. iPierian has set its sights from the start on neurodegenerative diseases that really have no good treatments—like Lou Gehrig’s disease, Huntington’s disease, and eventually Parkinson’s and Alzheimer’s. The cells, with all their talent to morph into any kind of adult cell scientists want, won’t get injected into the patient to replace missing or damaged tissue. Instead, they could help the scientists gather new insights into these intractable diseases, speed up the development process, and save time. Then iPierian will use all of this information to make conventional small molecule pills, or injectable protein drugs. If it is successful, the patients wouldn’t even know it was because of stem cells.
“The long term perspective here is to be a drug company,” says Walker, the CEO, who previously ran a publicly traded drug company called Novacea. “We’ll be discovering therapeutics, based on cell reprogramming and differentiated cells.”
A lot, and I really mean a lot, needs to happen before iPierian gets to that point. But the company has made some intriguing progress to date. It has set a goal of creating an “industrialized” process of taking adult skin cells from a human, and re-programming them into what are called “induced pluripotent stem cells.” These cells, which are thought to have potential of embryonic cells to differentiate into all 200 cell types of the body, are then coaxed to differentiate into a certain adult cell of interest in the lab.
It’s no small thing to do this work on an industrial scale. iPierian estimates that more than a year ago, it cost about $30,000 to generate a single line of induced pluripotent stem cells (IPSCs). Scientists have not even shown they can make these cells differentiate into every kind of cell type, much less done it on a consistent, large scale. But iPierian has brought the cost down 15-fold in the past year alone, Walker says. It has 200 different “lines” of induced pluripotent stem cells from different individuals socked away in the freezer, from patients with neurodegenerative diseases like Spinal Muscular Atrophy, Huntington’s, Parkinson’s, Alzheimer’s, from prople with Type 2 diabetes, and cells from healthy relatives to use for comparison to better home in on what might be going awry.
The cells, iPierian hopes, will provide the basis for a new way of discovering drugs, which Big Pharma companies will pay big money for the right
