Cardio3: Moving from Science Project to Business Model in Stem Cells
Cardiac stem cell therapy for regenerating diseased heart tissue has been long on promise but short on delivery. A Belgian incubator and start-up hope to finally bring this treatment to market.
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The debate over stem cells has produced a complex web of rules and regulations on both sides of the Atlantic. In the UK, the UK Stem Cell Foundation was established with the goal of attracting investors willing to support clinical research using stem cells, and to help translate basic research findings into clinically and commercially viable products. In the US, private firms have stepped into the gap created by a lack of federal research funding.
In heart failure, companies with expertise in gene or cell biology, or percutaneous delivery devices, see the first application where regenerative medicine could finally realize its promise. The enormous patient population, the high mortality of the disease, and the economics of treating it today provide a multi-billion dollar opportunity for which it's worth braving the complexity of cell therapy for tissue repair. Indeed, first-generation autologous heart cell therapies involve many different types of expertise resident in companies with different mindsets. Unknowns dog every component of the therapies on the level of basic biology. Still to be worked out: the right cell types; the optimal delivery route and device; when and at what dose cells should be administered, and in combination with which genes or drugs. Nevertheless, the great need in heart failure keeps companies dedicated to cardiac regeneration therapies. And as the cell therapy developers reveal new discoveries about the innate regenerative powers of the heart, drug developers are starting to move in, promising a much simpler approach than the combination products presently in the works.
With an increasing number of therapies derived from a patient's own cells, companies now have experience to draw on in designing commercial infrastructures for delivering these un-drug-like products. Indeed, armed with some small experience around how well they fit--or don't fit--into standard medical practice, cell-based therapies may finally be going commercial. And not only in terms of a transition from acute to chronic wound healing indications, where commercial success was first anticipated, but also as stem cell therapies, which would open up a multi-billon dollar opportunity in regenerative medicine, including replacements for organ and transplant surgery and cures for diabetes and neurological diseases such as Parkinson's Disease and Alzheimer's Disease, as well as autologous cancer immunotherapies. These companies are finding value in the experiences of earlier, technology-constrained companies whose attempts at developing cell therapies failed--Applied Immune Sciences and Systemix Inc., for example, both of which have virtually disappeared. Companies today are trying to overcome the challenges of product preparation, process standardization, and delivery. Other first generation cell therapy companies, like Genzyme Tissue Repair and Cell Genesys Inc., are still around. That a number of senior executives at today's leading-edge cell-therapy companies cut their eye teeth at those first-generation start-ups attests to their continuing belief in the broad commercial opportunities these technologies offer. But significant questions remain: Can companies solve the production economics of delivering products that require cell culture and expansion using a centralized, company-controlled laboratory model? The alternative, a decentralized technology transfer approach, would bring the product closer to the patient, but raises the question of whether the technology exists to standardize processes and satisfy regulators' quality concerns. Then, if these issues are solved, one must ask whether a hospital lab is the right setting for a commercial operation. Are there better commercial infrastructures, such as blood processing centers, with which companies could form alliances? And does the prospect of developing off-the-shelf, allogeneic-cell therapies-mass-produced stem cells that can pass the immune system undetected or vaccines derived from shared tumor antigens from cell lines, for example-threaten to obsolete autologous products before they become profitable?