The University of Minnesota said Wednesday it granted exclusive rights to a breakthrough stem cell technology to Athersys Inc., which plans to use it to develop therapies for treating a wide range of diseases. Dr. Catherine Verfaillie and other researchers at the university have shown that a specific type of stem cell isolated from adult bone marrow, known as Multipotent Adult Progenitor Cells, can be transformed into virtually any cell type.
Those include liver, lung, muscle, brain and kidney cells, according to a paper they published in the journal Nature in July. Use of these cells, which can be derived from a patient's own marrow, offers potential for treating diseases such as diabetes, Parkinson's, muscular dystrophy and inherited disorders, they said. Verfaillie's research caused a stir because it works with cells from adults, sidestepping ethical concerns that come with research on embryonic stem cells, which are obtained by destroying human embryos.
Verfaillie, director of the university's Stem Cell Institute, said Athersys' own technology promises to help commercialize a wide range of potential therapeutic applications based on MAPCs. In particular, Cleveland-based Athersys has developed a technology called RAGE, for Random Activation of Gene Expression, which allows researchers to rapidly discover and link a protein to its function. Combined with Minnesota's MAPC research, the company said it has the potential for identifying the proteins that induce these stem cells to "differentiate," or become various other cell types.
Combining Athersys's technology with Verfaillie's could benefit patients with many diseases who currently have few treatment options, said Gil Van Bokkelen, chairman, president and chief executive officer of Athersys. "There's a broad spectrum of opportunity here," Van Bokkelen said For example, he said, their research could lead to treatments for genetic defects or the failure of organs such as the liver. "In the near term we will focus on accelerating the clinical development of cell therapy treatments," Bokkelen said. "Our long-term goal, however, is to unlock the mechanisms responsible for the differentiation of stem cells."
As part of their efforts to advance MAPC research, the university and Athersys will make the stem cells available to other academic researchers. About 20 research institutions in the United States, Europe and Japan have signed agreements that give them access to the MAPC technology, they said. Those researchers are free to use the cells but Athersys would have the option to license any commercial applications, said Michael Moore, who's in charge of licensing the university's health technology.
Financial terms of the agreement were not released, but Van Bokkelen said the university will get an upfront payment and research financing from Athersys, a stake in the company and possible future payments and royalties. Some fear that exclusive agreements hamper progress by discouraging other researchers from getting involved, but Van Bokkelen said his company hopes to collaborate and share the returns.
Moore said the school decided to offer Athersys an exclusive license because nonexclusive deals tend not to attract as much corporate interest. University researchers and Athersys hope to start the first clinical trial using MAPCs in two years, probably to treat a rare genetic disorder. Van Bokkelen said the disorder they choose will depend on the progress of their research.
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