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SCS Licenses San Raffaele’s Stem Cell Line; Will Validate Its Use as Drug-Discovery Tool

Stem Cell Sciences this week announced that it has exclusively licensed a human muscle stem cell line from the San Raffaele Institute in Milan, Italy.
The line, developed by Giulio Cossu, director of San Raffaele’s stem cell research institute, will enable SCS to produce the cells at its automated cell-production facility in Cambridge, UK, SCS Chief Science Officer Tim Allsopp told CBA News this week.
The license gives SCS the right to develop the stem cells for drug discovery and toxicology applications, but does not give the company the ability to use the cells, which are a subset of pericytes, to develop human cell-based therapeutics.
Allsopp said SCS will also conduct a market analysis to identify potential customers for the muscle cell-based assays.
“SCS already has a fairly good idea of what the relative demand is from the drug-development community for this type of assay,” he said. “The company needs to scope that out a little further, so that within a short period of time it can accurately design the business model based on these cells.”
Allsopp said that this “evaluation phase” will last for nine months. “Toward the end of that nine-month period, SCS should have a far greater understanding of the efficiency with which it can differentiate the cells into muscle cells, and how easy they are to grow and to adapt to SCS’ platform technology approach, which is to grow stem cells in a serum-free, feeder-free, automated environment using robotics,” he explained.  
SCS would also like to develop a “ready-to-use” assay in a miniaturized multi-replicate format in which cells are aliquoted into the individual small wells of a multireplicate plate, and provide that assay for those interested in using it in drug discovery or development, said Allsopp.   
Uncharted Waters
San Raffaele’s Cossu has been working for some years on what are termed mesoangioblasts, which are a subset of pericytes, said Hugh Ilyine, SCS’ vice president and chief operating officer. SCS first became aware of Cossu and his work through the company’s network of academic scientists in Europe, Asia, and the US.
“In addition, SCS’ model, which looks at the early revenue and product-development potential of various types of stem cells, showed the company that pericytes represented another unique, patent-protected cell type that it could add to its growing catalog,” Ilyine said.
According to the terms of the licensing deal, Cossu and the San Raffaele Institute retain the right to decide who may work with them in the future to develop pericyte-based therapies. SCS’ license is limited to developing the cells into drug-discovery tools.
If during the evaluation period SCS is able to culture the cells and grow them into muscle cells, and also validate the assays, the company will consider exercising its exclusive option to take a full license to commercialize the cells, Ilyine said.                 
In the Beginning
This collaboration originated several years ago when SCS was working as a commercial partner with Cossu in an EU-funded consortium called Eurostemcell, Allsopp explained. At the research level, SCS became familiar with Cossu’s stem cells and performed basic research with the cells to try and grow them in serum-free culture media.
“That approach is one that we try and do with most, if not all, stem-cell types that we work with, particularly human stem-cell types,” said Allsopp. He said that cells grown in the absence of animal products such as fetal calf serum are more consistent in terms of quality and perform better than cells grown in these media.
It was during the Eurostemcell project that SCS realized how readily Cossu’s cells could be differentiated into muscle cells, Allsopp said. The Eurostemcell collaboration also took SCS to the point where it entered discussions and negotiations with the San Raffaele Institute, he added.  
The data generated by Cossu’s lab were proof that his cells were of a type that SCS would like to work with more extensively. “In particular, SCS could use the cells to make muscle cells,” said Allsopp. “We realized that a potential market existed for muscle cell-based assays using human striatal muscle, either from cardiac or skeletal sources.”   
This is the first licensing agreement for Cossu’s pericytes, said Daniela Bellomo, director of the San Raffaela Institute’s biotech transfer center, who added that the institute is looking to enter into additional agreements with other companies to develop these cells for therapeutic use.
Moving Forward
“I think this agreement is another indication that stem-cell research is actually moving forward to the next stage, which is from basic research to crossing the various hurdles to getting therapies to market,” said Eve Herold, director of public policy, research, and education at the Genetics Policy Institute.
Herold said that drug discovery applications are among the less written- and talked-about uses for stem cells because it is not quite as “glamorous as, for example, the use of stem cells to grow new organs.”
She said, however, that this is where a lot of exciting work is going on because people are doing more drug discovery/development and predictive toxicology than ever before.

“SCS’ model…showed the company that pericytes represented another unique, patent-protected cell type that it could add to its growing catalog.”

Another benefit of agreements such as this is that the resulting knowledge will reduce the need for animal testing and human stem cells in drug development, Herold said.
“Agreements like this will enable the [drug-development] process to bypass a lot of the stages in animal experimentation and go straight to humans,” she said. “So many agents are identified in animal research and then it turns out that the drug is toxic or is simply ineffective in humans.”
Ian Lyons, chief scientific officer of Stem Cell Innovations, told CBA News in an e-mail this week that “in general, the drug- and toxicity-screening business is in need of human cells of high quality. Human differentiated cells are usually limited in availability and quality, however.”
According to Lyons, stem cells offer a solution if they can be expanded sufficiently and then differentiated in controlled processes into uniform populations of cells that are similar enough to adult cells. Unfortunately, the technical developments needed to scale up production of the cells and to control their differentiation are not simple, he wrote. 
Although pluripotent cells such as embryonic stem and embryonic germ cells appear to be infinitely expandable, adult stem cells appear not to be, Lyons wrote. This will restrict the amount of cells that can be generated and put into master and working cell banks, which potentially limits the usefulness of such cells. 
Lyons pointed out that the whole differentiation process needs to be confirmed, and the phenotypes of the cells generated need to be shown to be an appropriate model of human tissue in vivo. After that production needs to be scaled up.

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