Cell therapy collaboration prepares to (fibro) blast off

Fibrocell Science, UCLA announce collaboration aimed at fibroblast research

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EXTON, Pa.—Fibrocell Science Inc. and the University of California, Los Angeles (UCLA) recently announced a scientific collaboration to research the conversion of dermal fibroblasts into other types of functional human cells that might have increased regenerative capacity.

Fibroblasts are cells that aid in the formation of connective tissue fibers, and both Fibrocell and Dr. James Byrne, assistant professor in UCLA's Department of Molecular and Medical Pharmacology, have considerable experience in the realm of autologous cells. Autologous cell therapy is based on using a patient's own cells for personalized treatment, and the collaboration will focus on this research and its potential for future diagnostics or treatments. Per the agreement, Fibrocell will provide funding, its knowledge and expertise in the area of fibroblast research and its proprietary platform technology in cell multiplication and expansion.

"We have a milestone-based agreement with UCLA over a three-year period of time," says David Pernock, Fibrocell's science chairman and CEO. No specific financial details were disclosed.

Byrne, who was appointed as a scientific advisor to Fibrocell as part of the agreement, will be leading the research group for the collaboration, which will take place at UCLA.

He says his group will be handling the research, analyzing the characteristics of the cells and how they could be used directly and indirectly once they've been "reprogrammed."

"My lab is focusing on the subpopulation in adult human skin cells and reprogramming those cells back into stem cells and using those as a means to achieve cellular therapy," Byrne explains.

Pernock notes that Byrne, whom he met at Stanford while Byrne was in the process of interviewing for his position at UCLA, was "the key reason" for the collaboration. Pernock says he contacted Byrne about the collaboration roughly a year ago.

"UCLA is on the forefront of cell science research, and Dr. Byrne is a renowned expert and researcher with pioneering discoveries in the study and re-engineering of human fibroblasts," says Pernock, adding that the collaboration is "very synergistic."

In collaboration with Dr. Renee Reijo Pera, Byrne is the first scientist to isolate a subpopulation of fibroblasts that can more easily be epigenetically reprogrammed into pluripotent stem cells, cells that can differentiate into other kinds of cells. Byrne also has experience in the conversion of human dermal fibroblasts into functional myocardial cells. Byrne says that the research is "amazing," citing one experiment in which skin cells were reprogrammed to become heart cells and "you could actually see the cells beating in the dish."

"When you see what was a piece of human skin cell become a beating human heart cell, it's one of the most amazing things I have seen," says Byrne.

Reprogramming human skin cells means that the skin cells could essentially become any of the 200-plus kinds of cells in the human body, according to Byrne. He explains that the concept of cellular therapy is that "any degenerative diseases resulting from tissue loss or damage could be theoretically cured." Byrne says that Parkinson's could benefit from this kind of research, adding that it also has applicability in diabetes and other secretory diseases. For the most part, though, he feels that "the degenerative diseases are the ones that would be most likely to benefit initially from the research."

"Any degenerative disease could conceptually be treated using this approach," says Byrne, adding that the benefit of using a patient's specific cells is that "the subpopulation from the patient's cells are genetically identical and won't be rejected by the patient's system."

Fibrocell hopes that through the collaboration, the parties will be able to expand both the current range of clinical applications to autologous cell therapy and their patented technology, which is capable of multiplying human dermal fibroblasts. He notes they are waiting on U.S. Food and Drug Administration (FDA) approval for the first indication and expect to hear from regulators by or before June 22, and adds they will "have the ability in the very near future to mass produce."

"It's early research, but ultimately, if it works, there's potential for diagnostics and there's potential for therapeutics," says Pernock.

The collaboration is still in its initial stages, but Byrne says he is anxious to get started. He adds that Fibrocell's research regarding fibroblasts one of the reasons he is looking forward to working with them, and he's "very excited to get going and get the work moving forward," an opinion that Pernock shares.

"I think it's a very exciting area of medicine," says Pernock. "And because of my company's work with autologous fibroblast cells, I think it's very important for us to be involved in this, because if it works it could represent a very significant advance."

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