No aspect of biopharma better epitomizes the goals and promise of delivering personalized medicine to treat and perhaps even cure intractable and disabling diseases than the emerging fields of cell and gene therapy. Adding to the excitement surrounding these technology-driven treatment approaches are their recent advance to the marketplace and increasing examples of progress in meeting clinical-scale production and regulatory challenges and achieving commercial success.
The progress and opportunities, obstacles and solutions, and optimistic projections and cautionary portents were all part of the science, technology, economic analysis, and insights presented, debated, and discussed at the recent “Phacilitate Cell & Gene Therapy Forum”.
Mahendra Rao, Ph.D., director of the NIH Center for Regenerative Medicine and head of the Laboratory of Stem Cell Biology at the NIH, led off the meeting and highlighted a key distinction from previous years: the growing number of commercial products now available for regenerative medicine.
Success begets new challenges, however, and Dr. Rao identified some of the issues this new reality is creating for young biotechnology companies. These may include how they ship their product, how to make a profit, how to deal with reimbursement and insurance issues, and how to manage competition.
The unique characteristics of cell therapies present new hurdles for this fledgling industry sector: individualized products prepared in small and even single doses; entirely or largely manual processes that need to be automated; and the challenges associated with reducing the cost of goods and leveraging economies of scale.
Other critical factors related to the actual products include storage, transport, time- and temperature-sensitivity, and time-out-of temperature specifications; chain of custody, overall quality control issues; and logistics considerations across the supply chain.
The main take-home message was to start thinking about factors related to commercialization of cell therapy product early in its development, and at every step along the way—including, for example, the potential impact of a change in a process, protocol, raw material, or analytical method.
Also emphasized by several presenters was the inevitable move toward automation, in the short-term at least aimed primarily at allogeneic cell therapies. Automated processing of cells and cell-based products will be essential for achieving optimal quality control and standardization, designing closed, disposable process streams, eliminating the risks associated with manual intervention and open systems, and reducing costs.
Moving to the Market
A roundtable discussion among a group of industry executives who have navigated the process of cell therapy commercialization focused on the question, “what does a commercially successful cell or gene therapy look like?”
One example was CartiStem®, developed by Medipost, and approved in 2011 in Korea for cartilage regeneration in the treatment of traumatic and degenerative osteoarthritis. The product has been used in about 250 patients but is not yet reimbursable. CartiStem is in Phase II trials in the U.S.
John Maslowski, vp scientific affairs at Fibrocell Science, which markets the aesthetic autologous human dermal fibroblast product azficel-T (LAVIV®) to improve the appearance of wrinkles, spoke about the labor-intensive nature of current autologous cell manufacturing processes.
Geoff MacKay, president and CEO of Organogenesis, described the company’s semi-automatic manufacturing process in place to produce its recently approved Gentuit™ product, composed of allogeneic human keratinocytes and fibroblasts, for oral tissue regeneration around teeth and implants.
For TiGenix, key hurdles in bringing to market its ChondroCelect® autologous cartilage product for the repair of single symptomatic defects have included lack of regulatory harmonization, and the need to reduce the cost and increase the effectiveness of therapy.