"The past two years have been nothing short of a revolution," says John Dimos, a senior scientist at iZumi Bio. "These cells didn't really exist two years ago. This is all brand-new technology, and it's opening up the potential for brand-new science." The company John is with, Izumi Bio, plans to take advantage of that potential by developing a bank of iPS cells from patients with various diseases and using the cells to screen candidates for drug development.
As a postdoc at Harvard, John Dimos built a cellular model of ALS (amyotrophic lateral sclerosis - Lou Gherig's Disease), making it possible to study a neurodegenerative disease outside an animal for the first time. He and his colleagues collected skin cells from an 82-year-old woman with ALS, reprogrammed them into iPS cells, and directed the cells to differentiate into motor neurons that were genetically identical to the donor's defective cells. "It was the first paper to show that you can use a stem cell to see disease pathology in a petri dish," says Douglas Melton, codirector of the Harvard Stem Cell Institute. "That means you can now study diseases in petri dishes and not in people. That's huge."
Several of our Sector Companies are attempting to take advantage of the growing market for supplying stem cells for use in research. So far none of them are using Induced Pluripotent Stem Cells (iPS). MIT's Technology Review cites three companies (Fate Therapeutics, James Thompson's company in Wisconsin: Cellular Dynamics, and GlaxoSmithKline) besides Izumi Bio concentrating in finding potential commercial applications for iPS cell technology. Of these, only Glaxo is publicly traded.
As a physician and venture capitalist closely following stem-cell research, Beth Seidenberg saw the potential for iPS cells almost immediately. Seidenberg, a partner at Kleiner Perkins Caufield and Byers, teamed up with another venture capital firm, Highland Capital Partners, to found iZumi in 2007, funding the company with $20 million. After 20 years in pharmaceutical research, Seidenberg has had a lot of time to think about what the industry is doing right and where it's going wrong. She says, "I became really intrigued by the idea of starting with a patient who had a disease and working backwards, which is exactly the opposite of how we pursue new therapies for treatment of disease today."
To illustrate the role iPS cells could play in drug discovery, John Dimos points to amyotrophic lateral sclerosis (ALS), a neurodegenerative disease he has studied for years. About 2 percent of all cases have a known genetic cause--a mutation in a gene called SOD1. Nearly all the work in animal models has focused on this rare form of the disease, because researchers know how to use the gene to trigger it in mice. With the new technology, however, scientists can use a skin biopsy to generate pluripotent stem cells from any patient with ALS. The genetics and other possible factors underlying the disease are captured in the cells, even if no one knows explicitly what those factors are. The same holds true for Alzheimer's, diabetes, autism, heart disease, and myriad other conditions whose complex origins have proved difficult to identify.
Because they are derived from human patients with documented medical histories, iPS cells are accompanied by reams of previously inaccessible information. "You can see from their medical history the progression of the disease, how they responded to different drugs, exactly what symptoms they experienced, and when," says Dimos. Certain drugs may be more or less effective depending on a patient's genetic makeup; some people, for instance, respond well to the breast-cancer medication taxol, while others may have no response at all. If scientists knew that specific medications worked for certain people or, conversely, caused them to suffer severe side effects, they could use their cells to try to figure out why--and use that information to develop better therapies.
The information in this post is primarily from an article by Lauren Gravitz in MIT's Technology Review. It is well worth reading in its entirety.