Author Archive

Opportunities and Challenges in the Development of Stem-Cell Therapies

Stem cells are being developed to treat a diverse set of conditions, including spinal cord injury, amyotrophic lateral sclerosis, macular degeneration, Parkinsons disease, and Type I diabetes. But the challenges in moving from the laboratory to the clinic are formidable.  The California-based biotech company, Geron, pioneered clinical trials using embryonic stem cells, with a Phase I trial using oligodendrocyte precursors derived from embryonic stem cells to treat spinal cord injury approved in 2009. In November 2011, however, Geron announced it would be discontinuing all of its stem-cell development work, citing cost and regulatory complexity as factors in that decision.

At the BIO International Convention, a panel will convene to discuss the current landscape for developing stem cell therapies in a breakout session titled Opportunities and Challenges in Developing Innovative Stem Cell Therapies. BioPharm International spoke with one of the session’s presenters, Dr. Armand Keating, Epstein Chair in Cell Therapy and Transplantation, University of Toronto about the challenges facing developers.  According to Keating, funding remains the biggest challenge in moving from laboratory to clinic.  He points out that unlike small-molecule development, stem-cell development is still very much rooted in academia. The traditional sources of funding available to academics are ill-suited or inadequate to fund preclinical validation studies, manufacturing scale-up studies, or the clinical trials themselves. While an organization such as the California Institute for Regenerative Medicine is able to provide some funding   for early-stage clinical trials, later-stage trials will be far too expensive to be carried out at academic research centers, he says.


Clarification of statement in podcast: There have been approximately one million bone marrow transplants performed worldwide since 1959.

Implementing FDA’s New Process Validation Guidance

Amy RitterIn January, 2011, FDA released its guidance, Process Validation, General Principles and Practices, describing process validation practices based on quality-by-design principles. At the IBC Biopharmaceutical Development and Production conference held this week in Huntington Beach, California, scientists described how their companies have been working toward implementing the guidance for the production of biopharmaceuticals.
Vijay Chiruvolu, director of corporate validation at Amgen discussed his company’s current process validation practices. Amgen, he explained, was somewhat ahead of the game, having worked toward implementing a data-driven and risk-based approach to process validation before the guidance was finalized. For the past 10 years, Amgen has taken a lifecycle approach to process validation, by beginning to plan process validation strategies during the early phases of process design and by continuing to update the plan throughout the lifecycle of the product. Process validation, he explained, is not a once-and-done activity, but should be adjusted as process understanding improves over time, or as expected or unexpected process changes require additional validation.
Peter Calcott, president of Calcott Consulting, weighed in with what the guidance means for small companies. Big biotechs like Amgen can devote a lot of resources towards risk assessment, process understanding, and developing a risk-based process validation plan, but smaller entities that outsource some or all of their process development and manufacturing activities will likely not have the staff or knowledge necessary to implement the guidance on their own. Calcott said he is commonly asked how much effort a company should spend in implementing the new guidance, and his advice, he says, depends on what the company’s business model is and where in the development cycle they are. For example, companies in early stages of development that expect to sell the compound to a larger entity for development need not concern themselves with the guidance, with the expectation that process validation will fall to someone else. A company that expects to manufacture and perhaps market the compound itself, however, should invest the time and resources to develop a compliant process validation plan.

Producing Stem-Cell Aggregates Using Bioprinting

Amy RitterResearchers from Heriot-Watt University in Edinbugh and Roslin Cellab in the UK describe a method for producing uniformly sized spheroids composed of human embryonic stem cells in the Feb. 4, 2013 issue of the journal Biofabrication. Read more »

Balancing Supply with Quality

Amy RitterAccording to FDA, 75% of the products in shortage are generic sterile injectables; low-margin products that must be manufactured in specialized facilities and that must meet stringent quality metrics. Read more »

From Raw Materials to Lifecycle Management: IBC’s Biopharmaceutical Development and Production Week

Amy RitterAt the end of February, sunny southern California will be the site for a week-long conference devoted to the development and production of biopharmaceuticals. Read more »

FDA’s Position on Abuse-Deterrant Opioid Formulations Becomes Clearer

Amy RitterWith the release of a draft guidance on the evaluation and labeling of abuse-deterrent opioid formulations, FDA is one step closer to clarifying its thinking on acceptable formulations for this product class. Read more »

Purchaser of Unapproved Drugs Pleads Guilty

Amy RitterShortages of certain sterile injectables pushed some practices to source these products from unapproved distributers. Back in February 2012, counterfeit versions of Roche/Genentech’s cancer drug, Avastin, appeared in the US, prompting FDA to send letters to practices that had purchased drugs from several foreign distributers, including Clinical Care, Quality Specialty Products (QSP), Montana Health Care Solutions, and Bridgewater Medical. Read more »

Opioid Formulation: Out with the Old

Amy RitterEndo Pharmaceuticals, maker of the opioid medication Opana ER (oxymorphone HCl), is suing FDA to prevent generic manufacturers from entering the market with formulations of oxymorphone that are not abuse-resistant. Read more »

A Tough Time for FDA at House Hearings

Amy RitterYesterday, FDA Commissioner Dr. Margaret Hamburg testified before the House Energy and Commerce Committee on the fungal meningitis outbreak. The purpose of the hearing was to determine why contaminated medicines entered the supply chain so that future occurrences could be prevented. Read more »

Will FDA Gain More Oversight Over Compounders?

Amy RitterIn the wake of the meningitis outbreak caused by contaminated compounded medications produced by the New England Compounding Center (NECC), both the government and the public have been wrestling with the question of how this happened—how did a company producing substandard medicines slip through regulatory oversight? Read more »

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