Technical Applications Track Sessions

Technical Applications Track 1: Quality and Technical Agreements for Cell Therapy Facilities
Thursday 5/19/2011
Time: 7:45am - 8:45am

Do you know when you need to establish formal agreements and with whom? Do you struggle with putting agreements in place in your facility? Are you unsure about what content to include that’s mutually beneficial for your material and service providers and your organization? If you have often asked yourself these questions then this is the session for you. We will present approaches to developing QA and technical agreements for facilities in the US and EU, and provide practical guidance for drafting these documents. Having current agreements in place protects your organization, is good business, and is important to monitoring the overall quality of materials and services so critical to the manufacturing of your product.

Technical Applications Track 2: Safety Testing for Cell Therapy Products: Requirements, Relevance, and New Technologies
Thursday 5/19/2011
Time: 3:30pm - 5:00pm

One of the many challenges of bringing any cell therapy into the clinic and potentially toward commercialization revolves around the issue of safety testing.  Regional regulatory guidelines can vary, making it difficult to know what level of testing is required, as well as what kinds of test “methods” are acceptable.  As new cell therapies and associated cell manufacturing procedures evolve, existing safety testing needs to evolve as well.  There are many different types of “safety tests” related to manufacturing process monitoring, as well as to the purity, identity, potency, and “sterility” of the final product. However, current safety tests have limitations and don’t always provide information critical to the cell products intended use.  Moreover, safety testing can be very time consuming and costly, and thus any new technologies which can reduce the time and cost of testing, and harmonize the process, will be welcome in the cell therapy community.

This session will open with an overview of European and US FDA regulatory requirements for safety testing, which generally emphasize detection of microbial contamination, viral adventitious agents, and tumorigenic potential.  Selection and qualification of test methods, and regulatory expectations for product release will be covered as well.  Then, a study will be presented that demonstrates how molecular profiling of a cell product (MSC) during culture and expansion may reveal critical product attributes that classical tests for safety/potency are unable to measure.  This kind of "profiling" may delineate new families of biomarkers that are important for manufacturing effective cell therapies.  Finally, we will look to see what kinds of tools are evolving to make safety testing and product “characterization” more “timely”, more affordable, and more adaptable to the unique needs associated with novel cell types, novel manufacturing processes, and novel clinical applications.

Technical Applications Track 3: Environmental Monitoring: How much is enough
Friday 5/20/2011
Time: 7:30am - 8:30am

This session will attempt to answer this common question in cell therapy facilities by presenting two perspectives.  Lynn O’Donnell will discuss the approach used in a non-classified cell therapy facility in the U.S., while Philip de Vries will present monitoring in a GMP facility in the EU. Attendees can expect discussion of other common environmental monitoring questions as well, such as “Do I have to validate my EM Program?” and “How do I know how to set action and alert levels?”  Plenty of time will be allowed for audience questions and comments.

Technical Applications Track 4: CD34 enumeration, how and when 2011
Friday 5/20/2011
Time: 10:45am - 12:15pm

While most laboratories use the ISHAGE protocol to perform CD34+ cell enumeration in HSC grafts, differences in reagents, gating schemes, when and how perform viability testing as part of their analysis and great variability still prevails. This WS was designed to improve the quality of CD34+ cell assessments in HSCT grafts. The topics that will be covered include suggested improvements of the ISHAGE method and of quality assessment of the enumeration assay. The speakers, from both academia and industry are all well known with more than 20 years in the field, will discuss CD34 antibodies, PMT, compensations and gating (CD45 and CD34), different  FCM platforms,  viability evaluated by 7-AAD staining  and gating of dead cells and when viability assessment is absolutely necessary. Frank Preijers, PhD the Stem Cell Laboratory Director/Supervisor Immunophenotyping  Radboud University Nijmegen Medical Centre, Netherlands  will present quality assurance (EQA) schemes and effective in reduction of inter laboratory variation and improvement in assay accuracy and viability testing. Lubomir Arseniev, MD, PhD, MBA from Cytonet Hannover GmbH will discuss the techniques used for CD34 enumeration in industry, quality control assays as well as for the manufacturing procedures that are in compliance with the Ph, Eur. and the national German regulations Joanne Kurtzberg MD, PhD Professor of Pediatrics, Professor of Pathology, Director, Pediatric Blood and Marrow Transplant Program and Director, Carolinas Cord Blood Bank, Duke University Medical Center, Durham, North Carolina, USA will present a clinical approach of enumeration on fresh and thawed cord blood cells and share their experience with both CD34 and ALDH staining.

Technical Applications Track 5: Establishing GMP Facilities with QA oversight in Academic Centers for Phase I/II Clinical Trials
Friday 5/20/2011
Time: 3:30pm - 5:00pm

In recent years, cell and gene therapies have shown the potential to treat malignant diseases and improve degenerative disorders of many organs including bone, muscle, nerve, retina, skin, pancreatic islets, and blood vessels. But after identification of an appropriate clinical target, establishment of a novel cell or gene based therapeutic approach, and completion of pre-clinical studies, trans¬formation of these research breakthroughs into new therapies for treating human disease is often difficult. Advanced cell therapy development such as cell transplantation, adoptive immunotherapy, gene therapy and regenerative therapy mandate cGMP (current good manufacturing practices) grade cell processing to assure the safety and quality of ma¬nipulated cell products. Therefore, one of the first steps to take from the bench to the bedside is the design of a facility for the preparation of clinical ma¬terial for use in human clinical trials. With this come many issues, questions and concerns relating to operating an academic GMP facility.

There are a number of medical centers in several regions of the world that are active in cell and gene ther¬apy and consequently have designed and built facilities ca¬pable of performing cell engineering and vector produc¬tion using cGMP. In this workshop, there will be a discussion of how differences in the expectations of research investigators and the requirements needed for pre-clinical development of cellular products were identified and how these were addressed in an academic center and therefore resulted in successful early phase cellular therapy studies. Also to be discussed will be how sources of information within the institution, the professional community and regulatory agencies are developed and accessed so as to facilitate IND/IDE submissions as well as premarket licensure/approval submissions.

Technical Applications Track 6: The Essentials of Validation, Qualification and Verification
Saturday 5/21/2011
Time: 7:30am - 8:30am

Who’s the man behind the curtain?  Are you caught up in semantics and the confusion associated with when to validate vs when to verify? Come learn and be prepared to participate in this dynamic interactive workshop. All new equipment, processes, and products that are critical to the safety, purity, or potency of products need to be qualified and validated before use. Speakers will share examples of well formatted validation plans with the participants and discuss how to prepare and operate with the corresponding SOPs. Participants will review specific examples of equipment and process validations (i.e. cryopreservation and thawing; cell processing and flowcytometric quality control). Upon completion of this workshop, attendees will take away tangible verification and validation examples as well as resources that can be used when you get back to the office on Monday.

Technical Applications Track 7: ISBT 128 Labeling: We’ve implemented, and so can you!
Saturday 5/21/2011
Time: 10:45am - 11:45am

ISBT 128 is a coding and labeling system, which was originally developed for blood and blood products to improve quality, safety and traceability in blood banking by ISBT. Today the standard is managed by ICCBBA.

Since 2005, the Cellular Therapy Coding and Labeling Advisory Group (CTCLAG) supported by most of the worldwide operating organisations in the field of cellular therapy (including, ISCT, JACIE/FACT) has been working on the ISBT 128 coding and labeling of CT-products. In this session, a short overview of the current activities of the CTCLAG will be given. Furthermore, two user's will share their experience: Boris Calmels from France and Giovanni Cameron from Canada both will give technical information on their implementation project in different settings. In this session, you will also have the opportunity to ask technical questions, share your concern, suggest improvements and interact with other participants on detailed technical CT-related issues.

Technical Applications Track 8: Process Development: Optimization and qualification of processes for maintaining quality parameters of therapeutic cells
Saturday 5/21/2011
Time: 1:15pm - 2:45pm

Process Development is a unique discipline that is not taught in University and is typically learned on the job.  Because of this, references to best practices in Process Development are difficult to find in manuscripts or books.  This session will cover the development processes that span the range of manufacturing processes, including 1) Cell Culture processes and harvest (upstream processing), 2) volume reduction and washing of harvested cells prior to final product formulation, and 3) formulation, final container choice, filling and cryopreservation of final product.  A focus on the critical quality parameters of therapeutic cells that must be maintained at each stage will be addressed.