Provoking Our Inner Stem Cells

A startup aims to spur the body's native stem cells to heal disease.

Fate Therapeutics, a startup based in La Jolla, CA, aims to harness the body's ability to heal itself by developing drugs that stimulate resident stem cells. Rather than developing cell transplants to replace diseased or damaged tissue, which is the focus of a great deal of stem-cell research, Fate is searching for molecules that can control the behavior of adult stem cells in different parts of the body. The two-year-old company began its first clinical trial in May of a novel molecule that could make cord-blood transplants more effective by enhancing the activity of the stem cells that create the blood and the immune system.

Caspar the Fish: By creating a transparent zebrafish, Harvard researcher Leonard Zon could watch fluorescently labeled hematopoietic stem cells from a donor fish repopulate the bone marrow of the recipient. The transparent mutant zebrafish, dubbed Caspar, provided a means for studying the effects of FT1050, an experimental drug now in clinical trials to boost the effectiveness of cord-blood transplants. Credit: Cell Stem Cell

The human body is full of adult stem cells--small populations of tissue-specific stem cells that are capable only of developing into the cells of their resident tissue, and whose job is to help maintain and repair that tissue. While they lack the flexible fate of embryo-derived stem cells, adult stem cells come in a variety of flavors, including those capable of making liver cells and immune and blood cells, among others. Fate Therapeutics believes that, with a little pharmaceutical prompting, these cells can be nudged to repair tissue and organ systems, or even fight back against cancer.

"[Adult stem cells] can be induced to proliferate, they can be induced to differentiate into the cell type they were destined to become, or potentially even induced to become something they weren't destined for that might be therapeutically relevant," says Paul Grayson, Fate's president and CEO.

To better understand how to activate and command adult stem cells, Fate has focused much of its research on induced pluripotent stem (iPS) cells, adult cells that have been reprogrammed back to an embryonic state and have the same flexible developmental potential as embryonic stem cells. Rather than trying to use these iPS cells to treat disease, Fate is using them as a discovery tool to learn more about which pathways are important for activating or inhibiting stem-cell development.

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"Fate's strategy is to try and take advantage of what we're learning about stem-cell biology to develop methods of using drugs to turn on or turn off stem cells," says David Scadden, one of the company's founders and director of the Center for Regenerative Medicine at Massachusetts General Hospital. Scadden and other top investigators from regenerative-medicine institutes across the country--including MIT, Scripps, and Stanford--were brought together by a group of venture capitalists who saw Fate Therapeutics as a way to quickly take their research to the forefront of stem-cell science.

Fate has raised $25 million in capital and is backed by three venture-capital groups. The company has been building its intellectual-property portfolio by licensing technology from different universities, focusing in part on iPS cell technology, as well as patenting the stem-cell discoveries of its founders and in-house scientists.