The past decade has been rich in biological and biomedical advances. The decade opened with the reports of the large number of new genes within the human genome that encode small non-coding RNAs, or microRNAs. We have learned that these RNAs a) control at least half of all genes, b) are dysfunctional in many cancers and c) are critical for many normal processes. In a recent study, microRNAs were used to control Hepatitis C Virus infection. In fact, the development of small RNA therapy based on RNA interference has advanced over the decade with clinical trials in many diseases.

However, the most important discovery of the past decade is that of “induced pluripotent stem cells” or “iPS cells,” which are adult cells that have been coaxed back into a embryonic-stem-cell-like state. The discovery of how to do that coaxing, made by Kyoto University’s Shinya Yamanaka in 2006, opened new avenues to consider for future treatment of diseases such as Parkinson Disease and type 1 diabetes—and has also radically changed our understanding of the plasticity of mature cell traits. For example, if any cell in the body can convert into any other cell type, i.e. from a neuronal cell to an immune cell, then classifications and treatment of cancers by cell type may be misleading. We are entering a new frontier in determining the nature of systems of genes and their proteins that control cell state and cell growth properties.

[Editor's Note: As the decade comes to an end, we've asked Xconomists around the country to weigh in with the top innovations they've seen in their respective fields the past 10 years, or the top disruptive technologies that will impact the next decade.]

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One of the scientific co-founders of San Diego-based Fate Therapeutics, along with his team at The Scripps Research Institute, is reporting a major advance that will make it faster, cheaper, and potentially practical on an industrial scale to turn adult cells into stem cells that can morph into any type of cell in the human body.

Sheng Ding and his colleagues at Scripps have found a combination of three conventional small-molecule chemical compounds that can coax adult human cells into an embryonic-like state. The new technique is about twice as fast as existing methods, and produces 200 times more cells per batch. The research in how to efficiently make these so-called “induced pluripotent stem cells” was sponsored by Fate, and is being published online today in the journal Nature Methods.

The technology, which is exclusively licensed to Fate through its sponsored research agreement with Scripps, is a big feather in the cap for the startup company as it seeks to strike deals with pharmaceutical and biotech companies that are looking get into the stem cell game. Fate has been a leader in the field since its founding two years ago by a group of top stem cell scientists from Harvard University, the University of Washington, Stanford University, and Scripps. One of those co-founders was Ding, a young scientist who got his first faculty post in 2003 at Scripps.

“This is the first example in human cells of how reprogramming speed can be accelerated. I believe that the field will quickly adopt this method, accelerating [induced pluripotent stem cell] research significantly,” Ding said in a statement from Scripps.

The latest advance builds on the discoveries of Shinya Yamanaka of Kyoto University and James Thomson of the University of Wisconsin, who showed for the first time two years ago that scientists could transform adult human cells into a pluripotent state, like that of cells in an early embryo. That was important because it was a way to circumvent the political and ethical controversy over destroying embryos in order to harvest their stem cells for research.

Pioneering as that work was, it was nowhere near ready for prime-time use in the biotech and pharmaceutical industries. Yamanaka and Thomson used viruses to insert multiple copies of four genes into adult cells. Two of the genes are known to cause cancer. Given that risk, it’s almost impossible to imagine regulators ever allowing cells with that kind of genetic modification to be injected into people who want to, say, regenerate new pancreas cells to treat their diabetes. The other big problem with the original method was that it took four weeks from start to finish, and only worked in about one out of every 10,000 cells.

Today’s announcement is the second big stem cell paper this year from the Ding lab. In May, the Ding lab reported that it had essentially gotten around … Next Page »

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San Diego-based Novocell has nailed down a second important patent on a valuable type of cell derived from human embryonic stem cells, which could end up forcing a lot of researchers to license technology from the company on Torrey Pines Mesa.

The company is announcing today it has secured a U.S. patent that protects its invention for a method of using endoderm cells for research when derived from human embryonic stem cells. A couple of months earlier, Novocell nailed down protection for a composition of matter patent on endoderm cells, a type of cell with wide-ranging potential. Endoderm cells can become pancreatic cells for use in diabetes therapies, or be turned into other tissues that make up the lungs, intestines, liver, thymus, and thyroid.

“This meets a long-standing need of major pharmaceuticals for human cells,” said Novocell CEO John West, in a statement.

Patents are a sensitive subject in the field of stem cell research—just ask the Wisconsin Alumni Research Foundation, which has taken heat for what some scientists consider onerous licensing terms for the right to use human embryonic stem cells developed at the University of Wisconsin. Yet exactly how Novocell’s patents might translate into revenue for the company, which is still at the earliest stages of research and development of new therapies, is hard to tell. It stands to reason that collecting licensing fees on patents like these would be part of its revenue model, since Novocell is still at least a couple years away from the valuation-building stages of bringing its first stem cell therapy for diabetes into human clinical trials.

West, who joined as CEO just a month ago, told me in an interview shortly after he started that the strength of the company’s intellectual property is one of the things that attracted him to Novocell. He definitely gave it a close look, especially since he’s someone who could probably have his pick of a lot of jobs, after he sold his last company, Solexa, a maker of gene sequencing machines, to San Diego-based Illumina for more than $600 million in 2007.

“It’s similar to Solexa, in that it has very promising scientific breadth, and now the question is how you take it commercial,” West says. There are still big hurdles … Next Page »

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The world’s largest drugmaker is turning to a San Diego-based biotech company to jumpstart its foray into the world of stem cell research. Pfizer (NYSE: PFE) has agreed to form a two-year collaboration with Novocell to advance the biotech’s work, which concentrates on turning human embryonic stem cells into pancreas cells that might one day help treat diabetes.

Under the deal, Novocell will get an upfront fee, research funding to pay the salaries of “a few” of its 40 employees, and potential milestone payments if the collaboration reaches certain goals, said Liz Bui, Novocell’s director of intellectual property. Novocell will also receive payments from Pfizer if any moneymaking product emerges from the collaboration.

Novocell formed a high-profile scientific collaboration last week with Japanese researcher Shinya Yamanaka, who has shown how to “reprogram” adult skin cells so they can turn into any other cell type in the body, the same characteristic of stem cells. But this is the first time Novocell has formed a collaboration with a Big Pharma company, Bui says. It’s an intriguing sign, because Pfizer and other major drugmakers have been slow to embrace human embryonic stem cells, which have sparked intense interest in academic labs, but haven’t yet advanced to their first human clinical trial. Last month, Pfizer opened a new center for regenerative medicine, and Novocell hopes that other Big Pharma companies will come knocking to learn more about how to do it themselves, Bui says.

“For Pfizer, this will help them stay on the edge of science,” Bui says.

Novocell, like other biotech companies pursing stem cells, has a long road ahead before it can enter clinical trials. It has shown an ability to coax human embryonic stem cells to become fully functioning pancreatic beta cells that secrete insulin in mice. It’s an effect that mimics what the pancreas normally does, said Ed Baetge, the company’s chief scientific officer, in a company profile I did last month.

The catch is that about 15 percent of the animals got teratomas, a type of tumor, which the company will have to prevent in its future work. For now, Novocell can take some comfort knowing that some of the deepest pockets in the pharmaceutical industry are shelling out at least a little spare change to help it crack daunting challenges like that.

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San Diego-based Novocell, a developer of embryonic stem cell therapies for diabetes, said today it has formed a partnership with one of the world leaders in stem cell biology, Shinya Yamanaka of Kyoto University.

Yamanaka dominated headlines a year ago when his team, along with James Thomson of the University of Wisconsin, showed they could coax adult skin cells to have all the properties of embryonic stem cells, which have potential to morph into any cell type in the body. This was a big deal, because in theory, it could circumvent much of the ethical controversy surrounding research that destroys human embryos in order to harvest stem cells.

Yamanaka’s team in Japan will combine his expertise with reprogramming cells with Novocell’s work to create human islet cells in the pancreas that can produce insulin for diabetics. The collaboration is at the basic research stage, and no commercial agreement has been signed, Novocell said.

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