Personalized medicine is a promising new approach to disease treatment. The ultimate in personalized medicine is a cellular therapy manufactured specifically for an individual patient using his or her own cells. But this autologous approach to generating immunotherapies has unique manufacturing challenges. Each patient receives an individual product batch, which needs to be manufactured, tested, and released. So thousands to tens of thousands of batches could be made for each indication every year. Given the personalized nature of these therapies,…
March 2011
From The Editor
Early on in the life of BPI we offered a few “closer†pages that we called “Defining Moments.†It was to be a forum for distinguishing between terms that are misused and for proposing new definitions for new approaches. In fact, because one particular topic came up again in a conference I attended this past January regarding the terms analytical and bioanalytical, we decided to revisit one of these entries from 2005 (with a few…
Cell Therapy Bioprocessing
The past 15 years have seen approval and commercialization of the first cell-based therapeutics, including cartilage repair products; tissue-engineered skin; and the first personalized, cellular immunotherapy for cancer. Those successes are outnumbered, however, by all too common product failures. Notable failures can be attributed to commercial concerns such as high cost of goods (CoGs) and technical hurdles such as inadequate characterization, high process variability, and loss of product efficacy when manufacturing is scaled up (1).…
Think Strategically for Design of Experiments Success
Global competition fueled by the power of information technology has forced the pharmaceutical and biotechnology industries to seek new ways to compete. The US Food and Drug Administration (FDA) has promoted quality by design (QbD) as an effective approach to speed up product and process development and create manufacturing processes that produce high-quality products that are safe and effective (1,2,3). Statistical design of experiments (DoE) is a tool that is central to QbD and the development of product and process…
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…
A 483 Primer
The words of George Santayana — “Those who do not remember the past are condemned to repeat it†— ring especially true for companies regulated under good manufacturing practices (GMPs). Learning from and reacting to lessons from past inspections (both your own and those of other companies) is one of the best ways to prepare for future inspections. Regular review and close study of 483 notices issued during inspections can be an efficient and accessible means of identifying and absorbing…
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…
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…
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.…
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…