It’s easy to look at Nobel Prize winners and assume the greatness of their work. But it’s even more awe-inspiring to understand the research and see it in action.
Their separate research involved converting adult stem cells back into ones that behave like embryotic cells in their ability to be coaxed into a variety of different cell types. Yamanaka’s work in induced pluripotency stem cells is the foundation of a technology platform being used by a company called iPierian, for which Yamanaka also sits on the board of scientific advisers.
iPierian’s CEO, Nancy Stagliano, based in San Francisco, talked with MedCity News recently about how the technology is being applied to develop treatments for neurodegenerative diseases.
What is so special about these cells, and how do they fit into what iPierian does?
iPierian was founded in early 2008 based on this discovery that human adult stem cells could be made into pluripotent cells. The idea behind it was that if we could make a number of different cell types from healthy individuals and disease patients and study them, we would have a better understanding of how to make drugs. We call that disease modeling, or disease in a dish. We get cells by taking a biopsy from a patient’s arm and then make them into stem cells.
In the last year and a half, we’ve focused on making those cells into brain cells. We make them from healthy volunteers and from people with neurodegenerative diseases. In Alzheimer’s disease, it’s a real step toward making better drugs because we can’t access the brain tissue of live humans with Alzheimer’s.
So are you making drugs of your own, or partnering with pharmaceutical companies to help them develop drugs?
Right now we have two antibody therapeutics that are preclinical. Both were discovered and developed using iPierian’s models. We do have the underlying platform that we can partner with pharmaceutical companies, but we haven’t done that yet.
What conditions are those drug candidates targeting?
The first one is against a protein called Tau and the other is toward a protein in the complement pathway — both apply to Alzheimer’s, and Tau is also applied to rare brain diseases like frontotemporal dementia and progressive supranuclear palsy. But the complement program isn’t limited to brain disorders; we also see it involved in rheumatoid arthritis and many places where inflammation is going on in the body. With the planning that we’ve laid out and with continued progress through the preclinical phase, we expect to file an IND in mid-2014 for both programs, with trials starting later that year.
How big of a confidence booster is this Nobel Prize for Dr. Yamanaka?
It’s a fantastic signal from the Nobel committee and from the world that this type of science is important to medicine. We’ve been supported by some leading VCs in life sciences [Google Ventures, KPCB and the venture arm of Biogen Idec, to name a few] and we’re all certainly happy to see this kind of endorsement.
*Editor’s note: This interview has been edited for clarity and brevity.
[Photo courtesy of iPierian]
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