NMDP Creates Cellular Therapy Biovigilance System
The National Marrow Donor Program has created a new vigilance and surveillance system to capture serious adverse events associated with hematopoietic stem cell transplantation as well as product quality issues. The system, highlighted in the January 2012 issue of AABB News, is a module of NMDP's electronic data collection system called FormsNet. Data on licensed and unlicensed cord blood units may be reported into the module, which was developed in conjunction with the Center for International Blood and Marrow Transplant Research, or CIBMTR. The module may be used by any U.S. hematopoietic stem cell transplant center, for units both facilitated and not facilitated by NMDP, as well as non-U.S. establishments that have received NMDP products. NMDP is currently working on modifications to allow for the reporting of adverse events and complaints associated with all cell products.
Opportunities for Participation
New Leadership on CT Subsections
The CT Section elections, the results of which were announced at the 2011 AABB Annual Meeting, created opportunities for other members to participate in CT subsections and take on leadership roles. Kevin L. Bundy, BB, MLSCM(ASCP)SBB, the new leader of the CT Business Management Subsection, and Suzanne Dworsky, MBA, MT(ASCP), its new associate leader, will work together to guide this subsection's activities. The group has begun working on several new resources relative to business issues affecting the cellular therapy community. Having already completed work on business plan templates, which included the Creation of a Program Template and the Changing of a Business Model Template, this subsection will continue to address reimbursement issues, regulatory issues, funding sources for cell therapy program development and clinical trials, as well as the administrative issues related to such a program. Individual members interested in participating in this subsection should enroll here. Wade Atkins, MT(ASCP)SBB, leader of the CT Regulatory Affairs Subsection, will work with his new associate leader Federico Rodriguez, MT(ASCP)SBB, to guide their subsection on U.S. and international regulations, guidance and policies of various government agencies. Those who are interested in these topics are encouraged to enroll here.
Journal Club Continues to Spark Interest
The journal club has become a popular activity of the CT Product Collection and Clinical Practices Subsection. The first presenter of 2012 was Lee Clough, RN, HP(ASCP), who led a discussion on a recent Transfusion Journal article. Journal articles are selected by the volunteer presenter and journal club discussion is held quarterly. Don't miss out! Those who wish to participate in this subsection's activities, including journal club, are encouraged to enroll. The group collaborates via conference calls held monthly.
The Novel Therapies and CT Product Development Subsection held its first journal club during its February conference call. Doug Padley, MT(ASCP) led the discussion based on, "Peak serum: implications of serum supply for cell therapy manufacturing," by Brindley et al., appearing in Regenerative Medicine. To participate in this subsection's activities, enroll here.
Cord Blood Subsection Survey Aims to Gauge Training Needs for Development of Educational Tools
Effective training to optimize the purity, potency and safety of cord blood collections is important. The AABB Cellular Therapy Cord Blood (CB) Subsection is asking for your help in completing two short surveys to aid in the development of training tutorials to achieve this goal. Based on your experience, please click on the appropriate link to participate in the survey directed to cord blood banks and/or cord blood collectors. Results of the surveys are intended for use in the development of training tools for individuals involved in cord blood activities. If you know anyone who would be willing to participate in the surveys, please share the links. If you are interested in participating in the CB Subsection activities to help develop these tools or other projects related to CB topics, enroll via the AABB website.
Tips and Tricks
Do you have a special "trick" or practice that you would like to share that improves or enhances work performance, process flow, work environment, interaction with colleagues or just makes your job easier? If so, we would like to hear about it. Send your "tips and tricks" to firstname.lastname@example.org for consideration for inclusion in the next CT newsletter. Please indicate whether you would like to include your name on the submission if it is selected.
Engraftment Failure Checklist Available
From clinical and laboratory perspectives, identifying the causes that may have contributed to engraftment failure is an essential quality parameter. The CT Product Manufacturing and Testing Subsection has compiled a checklist of "Factors to Consider" that can be adapted in investigating engraftment failure. Presented in a simple format, items include clinical and laboratory considerations along with a few suggested references. This resource is available as an AABB member benefit. The subsection continues to address technical and operational aspects of the manufacturing and testing of CT products such as cryopreservation, cell separation and selection, automation, product characterization, assay development, validation and implementation. Individual members interested in participating in this subsection should enroll here.
The Alliance for Harmonisation of Cellular Therapy Accreditation, comprised of cellular therapy standard-setting organizations including AABB, has met regularly to develop a series of crosswalks comparing cellular therapy standards and definitions in different topic areas in an "easy to read" format. The crosswalks, organized by topic areas, have been posted on the AHCTA Web page. Recent postings include a comparison of standards on cell collection as well as objectives, scope and definitions to better understand the standards.
Learning Tool on FDA Guidance Document
Fran Rabe, MS, CQM(ASQ), leader of the Cord Blood Subsection, presented PowerPoint slides for discussion with subsection members to highlight examples of FDA reportable adverse reactions and deviations with respect to the new GTP FDA guidance. These slides have been posted to the subsection page as a learning tool. AABB individual members interested in the Cord Blood Subsection may join here.
FDA Issues Guidance on Current Good Tissue Practice Requirements
The Food and Drug Administration's Center for Biologics Evaluation and Research has released the guidance document titled "Current Good Tissue Practice (cGTP) and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)." This document finalizes the corresponding draft guidance dated January 2009. It provides HCT/P manufacturers with recommendations for complying with cGTP requirements under 21 CFR, part 1271, subpart D, and additional requirements under subpart E. The guidance is intended for any establishment that performs a manufacturing step during the recovery, processing, storage, labeling, packaging or distribution of an HCT/P. The guidance also provides recommendations for establishments that manufacture HCT/Ps that are regulated solely under section 361 of the Public Health Service Act, as well as the regulations in 1271 and the HCT/Ps regulated as drugs, devices and/or biological products under section 351 of the PHS act and/or the Federal Food, Drug and Cosmetic Act. An analysis of this guidance will be posted on the AABB website in the coming weeks and will be announced via AABB communication vehicles when available.
FDA Debuts New Website
In response to feedback, FDA updated its website to improve navigation and raise the visibility of the most requested content. FDA is currently working on improvements to its search engine, which would yield a more complete response to search queries.
Regenerative Medicine Journal Offered to AABB Members
AABB is offering members an online subscription to the Regenerative Medicine journal at a discounted rate. The award-winning Medline referenced journal, available for $30 as a six-issue online subscription, addresses important challenges and advances in regenerative medicine and stem cell research. Coverage includes bench-to-bedside translation and scale up of stem cell and regenerative medicine therapies. The subscription option is available with online membership renewal and in the AABB Marketplace. AABB member subscribers may click here to access the journal using their AABB username and password.
AABB Community: Connecting CT Professionals
AABB members continue to join the CT group within the AABB Community! Similar to other social media tools, the AABB Community provides individual members the opportunity to discuss important topics, post questions or comments, learn from one another and share technical and practical findings. In addition, the platform provides an avenue to help individual members network with colleagues. Join your colleagues and stay connected. Sign up for email alerts so you don't miss out on Community discussions. See the FAQs to learn more about setting up the alerts and other useful information.
Three cellular therapy-related audioconferences will be held in the upcoming months. "The Role of Clinical Trial INDs in Supporting the Biologics License Application," to be held March 7, will discuss the importance of product labeling claims and review the significance of IND primary and secondary endpoints. The March 14 audioconference titled "The Future of Bone Marrow vs. HPC" will describe different methods for HPC collection; review the side effects among HPC donors; and examine how the HPC source may affect transplant patient outcomes. "Challenges of Real-Time CT Product Evaluation," to take place April 4, will review how different types of cellular therapy facilities handle problems encountered during product testing, explore the various approaches to handling release of products in low staffing situations and compare strategies for exception release of cellular therapy products that fail to meet release criteria.
Stem Cells Engineered to Kill Cancer
A potential therapeutic approach and an important tool to help understand and optimize the human immune response to melanoma and possibly other cancers has been presented in Proceedings of the National Academy of Sciences. Using a humanized mouse model, Vatakis et al. show that human blood stem cells can be engineered to create cancer-killing T-cells that seek out and attack a human melanoma. Following tumor challenge, transgenic CD8+ T cells, in the absence of additional manipulation, limited and cleared human melanoma tumors in vivo.
Cord Blood Meets Its Match
The importance of matching at the HLA C locus has not been well defined for unrelated umbilical cord blood transplantation. The selection algorithm for umbilical cord blood units generally considers intermediate resolution HLA typing at A and B and allele-level typing at DRB1. Specific data presented by Eapen et al. in The Lancet Oncology aimed to establish the relative importance of additional matching at HLA C. Findings suggest that the present strategy for umbilical-cord blood unit selection should be reassessed; matching at HLA C for units that are matched at HLA A, B, or DRB1 or in the presence of a single locus mismatch at HLA A, B, or DRB1 should be included to minimize mortality risks.
Review Article: Cell Characterization in Process Development
Creating therapeutic products composed of living cells presents many challenges. A major challenge to commercializing cell-based therapies is developing scalable manufacturing processes while maintaining critical quality attributes (CQAs) of identity, potency, purity, and safety. In the January issue of Regenerative Medicine, Carmen et al. discuss these parameters including choosing assays and assigning their roles in support of a cell therapy product as well as cell characterization assays as tools for process development.
Review Article: Mechanical Control of Stem Cell Differentiation
Numerous studies have focused on identifying the chemical and biological factors that govern the differentiation of stem cells; however, recent research has shown that mechanical cues may play an equally important role. Mechanical forces such as shear stresses and tensile loads, as well as the rigidity and topography of the extracellular matrix were shown to induce significant changes in the morphology and fate of stem cells. Dado et al., in the journal Regenerative Medicine, survey experimental studies on the response of stem cells to mechanical and geometrical properties of their environment and discuss the mechanical mechanisms that accompany that response, including the remodeling of the cytoskeleton and determination of cell and nucleus size and shape.
Cord Blood: Biology, Immunology, Banking, and Clinical Transplantation
Cord blood transplantation and research are still evolving, having seen vast change since the first successful cord blood transplantation more than 20 years ago. Cord Blood: Biology, Immunology, Banking, and Clinical Transplantation, by Hal Broxmeyer, PhD, continues to advance the field by providing a solid understanding of its present status. This comprehensive resource incorporates the latest developments into 39 chapters by experts in various areas of practice. Topics include cord blood biology, engraftment and regenerative medicine.
New Digital Download Available: Core Principles in Cellular Therapy
The chapters of the Core Principles in Cellular Therapy digital download form a key resource for a distinct readership in laboratories who typically have not used the Technical Manual. Highlights include a revised section on quality control in the marrow chapter and new data on adhesion-blocking agents in apheresis collections. This economically priced resource is great for training medical staff.
Cellular, Tissue, and Gene Therapies Advisory Committee Meeting Transcripts read more »
FDA Advisory Committees' Tentative Schedule of Meetings for 2012
read more »
Biopreservation Core Resource at University of Minnesota read more »
CBER 2012 Draft Guidance Agenda for Blood and Blood Components and Cellular, Tissue and Gene Therapy read more »