Fibrocell’s autologous cell therapy Laviv was reviewed by CBER with significant help from CDER and CDRH, providing a model of how FDA can handle high-tech products that cross traditional boundaries.

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?

As a small company selling into the fragmented wound care markets, Integra was spending as much as it was making. As a platform biomaterials company, Integra faced partnership risk and stood to only get a cut of end-user revenues. It needed a source of direct revenues. A year ago, the company's new CEO began building a neurosurgery business through acquisitions, initially based on mature, revenue-producing products. The strategy seems to be working; the company reached profitability in the last quarter and now holds the third position in a fragmented neurosurgery market. But the incremental strategy that has brought Integra to profitability inherently has its limitations. Now that it has created a growth expectation, it needs to achieve critical mass, through increasingly larger acquisitions or home-run products.

Genzyme Corp is merging two of its subsidiaries, Genzyme Tissue Repair and Genzyme Surgical with a third company, Biomatrix Inc. in a bold bet on the promise of biosurgery--the combination of biomaterials and gene and cellular therapies with more traditional surgical instruments. Clinically, biosurgery will hopefully improve patient outcomes; commercially, it will differentiate surgical instrument makers whose products seem increasingly alike.