Cell Therapies

Building a Bridge to Commercial Success

The history of the biopharmaceutical industry is one of continual invention and reinvention, of business models that have adapted to weather uncertain product futures and shifting economic fortunes. Some of us followed the up-and-down (and often financially painful) progress of monoclonal antibodies toward their eventual commercial success — a wealth of experience to draw from as other classes of products make their way from laboratories and onto the market. The vast majority of regenerative medicines are still produced at laboratory…

Therapies of Tomorrow Require More Than Factories from the Past

Live cells are being incorporated as active agents and delivery vehicles for a broad range of emerging therapeutic strategies. Successful commercialization of a cell therapy requires more than proving its safety and efficacy to regulators. Ultimately a therapy must be commercially viable, allowing enough patients to be treated with an adequate financial margin to justify investment in it as a product. “Whether the cells used are universal (allogeneic) or patient-specific (autologous), it is unlikely to be wholly one or the…

Successful Commercialization Through Industry Collaboration

    Nearly a year ago, the International Society for Cell Therapies (ISCT) decided to integrate industry into its organization to build a stronger platform for commercializing therapies. Robert Deans, vice president of regenerative medicine at Athersys, was invited to serve as a leader of ISCT’s Industry Task Force, which aimed to identify industry roles in its organization. Within two months, the task force invited industry members and chartered a white paper (1) that described how ISCT should go forward.…

Opportunities in Regenerative Medicine

Capitol Hill fly-in days (see the last page of this issue) … A focus of Google Ventures (www.google.com/ventures) … A favored new investment arena for GE’s CEO Jeffrey Immelt, the recently named head of President Obama’s economic recovery advisory panel, and Life Technologies’ Greg Lucier … Hardly a day skipped without a major news publication covering some exciting aspect of the science … The provocative cover of Wired magazine’s (www.wired.com/magazine) November 2010 issue … It all sounds like the stuff…

Stem-Cell–Based Therapies

A recent review of therapeutics in clinical development revealed 68 stem cell-based approaches (1). The majority of those leverage a patient’s own hematopoietic stem cells; others are exploring use of mesenchymal, neural, or embryonic stem cells. Here I highlight new therapeutic applications of stem cells and explore advances in the areas of induced pluripotent stem cells (iPS cells) and process-scale production of stem cells. Both should create new opportunities for stem cell-based therapies. Types of Stem Cells Hematopoietic stem cell…

Technologies on the Cutting Edge

    On the brink of bringing exciting new therapies to commercialization, cell therapy developers are taking notice of how other companies are addressing processing and technical challenges. Here, leaders from Dendreon, Advanced BioHealing, and Pluristem describe their current cell therapy programs. And two organizations —the Alliance for Regenerative Medicine (ARM) and McLaughlin–Rotman Center for Global Health —provide details on the promises of regenerative medicine.   Cellular Immunotherapy   Dendreon’s Provenge (sipuleucel-T) cell therapy induces an immune response to aid…

Industrializing Stem Cell Production

Stem cells have potential as a readily available, consistent source of many differentiated cell types. This unique property can be leveraged both for therapeutic purposes and for facilitating and improving a number of drug discovery and development processes. Large-scale, “industrialized” production of human stem cells in tightly controlled conditions will be required to deliver the quantity and quality of cells needed to support clinical trials and drug discovery development activities (Figure 1). Achieving this level of production while meeting rigorous…

Manufacturing Convergence Technologies

    Many regenerative medicine products represent a convergence of pharmaceutical, biologic, and medical device technologies. Although such products could have a great impact on medicine, they often pose significant challenges for their developers, requiring companies to incorporate competencies from several technology sectors. By addressing commercial regulatory and manufacturing challenges at an early stage in product development, these companies are more likely to succeed in reaching their commercial goals. Exact regulations governing the manufacture of a convergent technology — or…

Fluorescence-Activated Cell Sorting for CGMP Processing of Therapeutic Cells

    Cell therapy using embryonic or adult stem cells for regenerative medicine is generating high interest in the global medical community and in the general population.Physicians and patients are looking to cell therapies as potentially curative treatments for diseases such as diabetes, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, Graft versus Host disease (GvHD), and cancer. Cell-based therapeutic products have been administered in clinics for nearly 90 years in the form of blood transfusions and for 50 years in the…

Stem Cells and Tissue Engineering

Stem cells are probably the most-discussed — and least understood — potential therapeutics biotechnology offers. Headlines in mainstream media tout their potential benefits and decry their ethical complications. Time magazine featured stem cells on its cover one week in February (1), and an ABC network drama depicted criminals selling stolen cord blood stem cells to the rich and vain as a high-end cosmetic treatment (2). It’s a safe bet that most nonscientists don’t know the difference between embryonic stem cells,…