Unleashing the Promise - Pharmaceutical Executive

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Unleashing the Promise
An up-close look at some companies that may soon bring stem cell-based therapies to market.


Pharmaceutical Executive



The Select Few
Umbilical cord blood These cells were isolated from umbilical cord blood, and such blood is particularly advantageous because of its newness. Studies show that MLPCs have the plasticity to generate into adipocytes (fat cells), as well as osteogenic (bone-forming), mesenchymal (stromal cells found in bone marrow), and neural cells. From a clinical standpoint, this is advantageous, especially considering that the range of differentiation with bone marrow tends to be limited.

One year after BioE shared its news about MLPCs, they announced an even more significant development: cloned adult stem-cell lines that are similar in many ways to embryonic stem-cell lines. These cloned stem cells are derived from single-cell umbilical cord blood MLPCs and will soon be available for pharmaceutical R&D applications. "We're growing up these cells and will offer genetically characterized cell lines to researchers by the end of June 2005," says Mike Haider, BioE's president and CEO.

Despite their remarkable plasticity, MLPCs are normal cells. This means that, although they do not have some of the downsides of aberrant embryonic stem cells, they eventually die. However, they can continually be re-cloned. In fact, at this moment, BioE has developed more than 50 individual cell lines, each of which is capable of reproducing hundreds of millions of cloned cells.

Processing system At the core of BioE's research efforts is its proprietary antibody-based cell-processing technology, the patent-pending PrepaCyte System. "This product is on track to become the first FDA-compliant cell-separation product of its kind," Haider says. It may also be the key to unlocking two major challenges associated with stem cells, yield and stem cell identification. This system is designed to facilitate high-yield recoveries of MLPCs, and hematopoietic and non-hematopoietic cells from umbilical cord blood.

Here's how it works: PrepaCyte causes unwanted cells to settle to the bottom of the container, leaving desired cells in the upper fraction of the solution. This process allows stem cells to be easily removed for further processing before therapeutic use in humans, and it also results in increased quantities and higher purity of cells that retain their innate cellular functionality. (See "The Select Few".)

BioE's cell-separation technology is unique in that it is a biological process based on antibody-based negative selection, so there is no equipment or machinery involved. It all comes down to a solution full of monoclonal antibodies, which stimulate certain cells in the blood to express certain adhesion molecules on their surface.


Chris Calhoun, CEO, MacroPore Biosurgery
Haider uses the "needle in a haystack" metaphor to clarify the elegance of this process. "This is a completely closed-bag system that promotes total stability. In essence, you get rid of the hay without touching the needle," he says. Distinct advantages are volume reduction, cost-savings, structural stability, and cell-selection specificity for therapeutic uses, such as transplantation. In addition, working with an FDA-compliant cell-processing system makes it easier to commercialize stem cell-based applications and facilitate therapeutic use. "If the tools are FDA-compliant when a particular therapy becomes part of the standard of care, it's a much smoother transition for the entity that is marketing or delivering the therapy," Haider says.

Antibodies BioE's diagnostic technology also relies on antibodies. In the diagnostic platform, antibodies are used to detect both members of a binding pair, such as an immune complex, to facilitate early diagnosis. Of particular interest is their Alzheimer's disease (AD) diagnostic process. "Post-mortem analysis shows that free iron in the brain is very toxic," Haider says. "Our AD diagnostic is based on identifying the presence of the iron-regulatory protein 2 gene [IRP-2] in the brain. The goal is to develop a definitive diagnostic that could be implemented way before symptoms develop."

NIH has committed a $6.5 million grant to fund research for BioE's diagnostic, and there is currently a five-year, double-blind multicenter study taking place at Loma Linda Medical Center in California. BioE is well positioned to continue its fast-paced growth. In addition to grant funding, the company has just completed an $8.3 million round of private funding, bringing total funding since inception to $22 million—all from angel investors.


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