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AABB CellSource - April 2018

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AN UPDATE ON CELL THERAPY NEWS FROM AABB
April 2018
in the news
Bioethics Among Large Array of Topics Important to Cord Blood Industry To Be Presented at ICBS
CELLSOURCE_ICBS50.jpgThe International Cord Blood Symposium (ICBS), the premier event for the cord blood, cord tissue and regenerative medicine industries brings together top experts in cord blood banking and research. The 2018 program comprises lectures and workshops on a variety of topics including cord blood transplantation and outcomes; novel therapies and clinical applications; cord blood expansion; cord blood unit connection with industry partner end users; regenerative medicine applications of cord blood/tissue-derived, perinatal-derived therapies; and delayed clamping. Leading bioethicist Leigh Turner, PhD, will give a talk on “Unproven Therapies: From Do It Yourself Gene Editing to Stem Cell Tourism.” In the session, Turner will provide an empirical analysis of the current U.S. direct-to-consumer marketplace for stem cell interventions and discuss ethical issues related to the proliferation of clinics, the expansion of this market in response to deregulatory pressures, public mistrust of scientific institutions and regulatory bodies, and the emergence of biohacking and DIY gene editing. Early bird registration for ICBS ends April 30.
AABB encourages readers to consult the resource, “Unproven Therapies- A Guide for Patients” which defines cellular therapies, informed consent, clinical trials and includes patient resources.
Deadline to Submit an Abstract for the 2018 AABB Annual Meeting is April 26
callabstracts_copy.jpg?r=1523903579785The AABB Annual Meeting provides a forum for high-quality educational and networking events for health care providers from around the world in transfusion medicine and cellular therapies. Attendees will have the opportunity to publish and present their cutting-edge research to thousands of attendees at the meeting, to be held Oct. 13-16 in Boston, Mass. The deadline to submit an abstract is April 26. The AABB Selection of Abstracts Committee will consider submitted abstracts for oral or poster presentation. The Abstract Submission Guide provides information on the abstract process, including instructions for preparation and submission.
Standards Coordinating Body Brings Together Organizations to Support Standards Development
The Standards Coordinating Body (SCB) for Gene, Cell, and Regenerative Medicines and Cell-Based Drug Discovery brings together product developers, tools and service providers, professional societies, government entities and academic centers to support standards development. Through supporting, coordinating and providing education about the standards landscape and its "gaps" in the regenerative medicine community, the SCB aims to create a more harmonized compliance environment (both domestically and internationally). SCB's mission is to efficiently and effectively support and improve the cost, time and resources for regenerative product development. AABB participates in SCB. Find out more about SCB, its methods and the results of its coordinated efforts.
NBF Award Helps Launch Independent Research Career
National_Blood_Foundation.jpgThe National Blood Foundation (NBF), a program of AABB, plays an important role in helping researchers achieve their career goals. After receiving an NBF award, 2016 scientific research grant recipient Mobin Karimi, MD, PhD, came closer to accomplishing his research goal of understanding how manipulating immune cell receptor signals can be used to alter immune cell functions. The NBF grant allowed Karimi to complete several projects related to the discovery of a novel human activating receptor, natural-killer group 2, member D (NKG2D) splice variant and mechanistically investigate its role in an anti-tumor response. He recently discovered that truncated NKG2D expression plays a critical role in leukemia relapse rates and prolonged survival of allogeneic hematopoietic stem cell transplant (HSCT) patients. Karimi demonstrated that NKG2D expression by CD8+ T cells plays a significant role in mediating graft-versus-host disease (GVHD) and graft-versus-tumor (GVT) effects by promoting the survival and cytotoxic function of CD8+T cells.
 
Using a murine model, Karimi found that expression of NKG2D ligands is not induced persistently on normal tissues of allogeneic HSCT-recipient mice treated with anti-NKG2D antibody, suggesting that transient NKG2D blockade might be sufficient to attenuate GVHD and allow CD8+ T cells to regain their GVT function. In his research, short-term treatment with anti-NKG2D antibody restored GVT effects while maintaining an attenuated GVHD state. NBF funding helped to support this research, which facilitated further funding and the launching of an independent research program and career. AABB congratulates Karimi for securing a tenure-track assistant professor position in the Department of Immunology and Microbiology at SUNY Upstate Medical University. His long-term interest is in developing therapeutic strategies for immune cell-mediated diseases. Individuals interested in learning more about NBF and how to apply for 2019 awards are encouraged to visit the NBF website.
WHO Develops Searchable Notify Library to Share Didactic Information on Documented Adverse Outcomes Associated With Clinical Use of Medical Products of Human Origin
AABB Center for Cellular Therapy staff participated in the recent meeting of technical experts for the Notify Library project. The Notify Library is a joint global initiative, co-sponsored by the World Health Organization and the Italian National Transplant Centre, which serves as its Collaborating Center. The library supports the sharing of published vigilance information for teaching purposes and for greater public transparency on the use of medical products of human origin. It aims to be a comprehensive resource that describes all types of adverse reactions or events that might have teaching value and assist in estimating risk. The library has added an open access, searchable database.
Internationally Standardized Terminology for Regenerated Tissue Open for Public Comment
ICCBBA requests public comment on the consultation document “Internationally Standardized Terminology for Regenerated Tissue.”  Any comments or suggestions to enhance the document should be sent by May 11, 2018 to regentissue@isbt128.org or via the electronic form found on the ICCBBA website. The Regenerative Medicine Technical Advisory Group (RMTAG) will review all comments prior to finalizing the terminology.
REGULATORY AND GOVERNMENT UPDATE
AABB Releases 2018 Advocacy Agenda
CELLSOURCE_AABBlogo.jpgThe AABB Board of Directors approved the association's 2018 advocacy agenda. This document will guide the association’s advocacy efforts for the year ahead. AABB's advocacy efforts for 2018 will focus on advocating for policies that support two broad objectives: ensuring that patients and health care providers have adequate and appropriate access to safe transfusion medicine and cellular therapies and enhancing patient and donor care and safety in transfusion medicine and cellular therapies. AABB plans to advance these goals by focusing on several priorities, including the sustainability of the U.S. blood system; access to safe cellular therapies; adequate coverage and payment policies; appropriate regulation and availability of products and technology; and patient and donor care and safety.
CAR T Cells Make an Appearance on FDA D.I.S.C.O.
Sanjeeve Bala, MD, MPH, a medical oncologist and acting clinical team leader at FDA, and Abhilasha Nair, MD, a medical oncologist at FDA’s Oncology Center of Excellence, discussed the recent approval of tisagenlecleucel, marketed as Kymriah, on FDA’s Drug Information Soundcast in Clinical Oncology, or D.I.S.C.O. FDA posted a full transcript of the discussion online.
FDA Research to Improve Technique for Studying Multipotent Stromal Cell Morphology
FDA scientists demonstrated unexpected changes in the morphology (shape and size) of multipotent stromal cells (MSCs) when preparations of the cells interact with specific engineered culture systems. The finding is a step toward understanding how to accurately interpret studies that use such systems, called microfluidics platforms, so they can be used to support the development of safe and effective medical products based on MSCs. More information is available on the FDA website.
RESEARCH FOCUS
Quality of Life Improvement Following Islet Cell Transplant
regenerate_copy.jpg?r=1523893590603The results from a Phase 3 clinical trial show consistent and dramatic improvements in quality of life for people with type 1 diabetes with frequent severe hypoglycemia — a potentially fatal low blood glucose (blood sugar) level — following transplantation of insulin-producing pancreatic islets. Islet recipients also reported better overall health status after transplant, despite the need for lifelong treatment with immune-suppressing drugs to prevent transplant rejection. Researchers observed these improvements even among transplant recipients who still required insulin therapy to manage their diabetes. The trial enrolled 48 people with type 1 diabetes who had an impaired ability to sense drops in blood glucose levels and experienced frequent episodes of severe hypoglycemia despite receiving expert care. Previously reported clinical outcomes from the trial showed that islet transplantation prevents severe hypoglycemia and improves blood glucose awareness and control. The study was conducted by the NIH-funded Clinical Islet Transplantation Consortium. The full findings are reported online in the American Diabetes Association journal Diabetes Care.
“How Do I…” Feature in Transfusion Describes How Clinical Trials Are Managed in the Cellular Therapy Laboratory
The services required in clinical trials are variable, ranging from sample receipt, storage and issue to simple manufacturing, and require multiple clinical trials coordinators to communicate about staff availability, ability to perform the necessary steps in the protocols and fee schedules. Realizing the importance of having a process to stay organized, Tanhehco and Schwartz developed a standard operating procedure (SOP), published in Transfusion, that includes processes and forms to help manage clinical trials in the cellular therapy laboratory.
Regenerative Medicine Landscape Report Provides Overview of Standards
Nexight Group LLC and the Standards Coordinating Body (SCB), in which AABB participates, co-authored an FDA-funded report, “The Regenerative Medicine Standards Landscape,” to provide an overview of nearly 200 existing standards relevant to the cellular therapies, gene therapies and tissue engineering sectors of regenerative medicine. The report further assesses needs and opportunities for standards development in regenerative medicine, as identified by key stakeholders. The report will be regularly updated to provide the most comprehensive list of regenerative medicine standards and to identify emerging standards needs in response to the rapidly developing regenerative medicine field. Nexight Group welcomes suggestions for improving the report or information on other standards relevant to regenerative medicine; those with comments may send them to Sarah Lichtner at Nexight Group (slichtner@nexightgroup.com).
ARM and NAMCP Publish Study of Managed Care Medical Director Perspectives
The Alliance for Regenerative Medicine (ARM) and the National Association of Managed Care Physicians (NAMCP) Medical Directors Institute released a joint study of medical director perspectives on value demonstration and reimbursement for regenerative and advanced therapies. The study, "Ensuring Patient Access to Regenerative and Advanced Therapies in Managed Care: How Do We Get There?," identifies challenges and posits solutions to ensure the appropriate integration of these high-value therapies into the current U.S. health care system. The study also looks at various proposed alternative payment and financing models to enable sustainable patient access to these products.
EVENTS, OPPORTUNITIES, RESOURCES
AABB eCasts
CELLSOURCE_ecastsimage.jpgAABB offers educational programs via eCasts — live, online interactive programs covering topics in transfusion medicine, cellular therapies and patient blood management. Registration is available for individuals and groups; and institutions can host eCasts for employees at one or more sites. Participants in group eCasts are eligible to earn continuing education credit. On-demand recordings of eCast sessions are available for those unable to attend a live eCast.
 
Upcoming CT eCasts:
 
Cellular Therapies Certificate Program Provides CME Opportunity for CT Professionals 
CELLSOURCE_CTCERTIFICATEprogram.jpgAABB Cellular Therapies Certificate Program, produced in partnership with the George Washington University, is a self-paced, online program designed for professionals interested in expanding their knowledge of CT. The program features 12 narrated modules covering scientific, operational and regulatory topics. Students who work through the modules, complete each assessment with a score of 80% or higher and fill out a program evaluation form will receive a certificate of completion. The program also provides continuing education credits (CMEs).
AABB Posts Cellular Therapies Transfusion Articles Online 
CELLSOURCE_AABBlogo.jpgAABB compiled a collection of 40 articles from Transfusion, AABB's monthly peer-reviewed journal, as an online reference for cellular therapy professionals—especially those working to complete the AABB CT Certificate Program. The selection addresses the topics covered by the 12 modules in the AABB Cellular Therapies Certificate Program and will be a convenient resource for many members. This reference provides online links to complete journal articles.
United States Consensus Guidance Posted for the Uniform Labeling of Cellular Therapy Products Using ISBT 128
Some aspects of label design using ISBT 128 are internationally standardized, while others have been left for national authorities to determine. An example of an internationally standardized aspect is the placement and content of bar codes, while examples of nationally determined aspects are what text must appear on the label and whether certain information (such as a collection date) must be visible. The ISBT 128 Standard Technical Specification (ST-001) addresses aspects of the standard that are internationally defined. The purpose of the "US Consensus Guidance for the Uniform Labeling of Cellular Therapy Products Using ISBT 128" is to provide guidance for users, software developers and label vendors in the U.S. in those areas of CT product labeling that are not internationally defined.
NIH Provides Resource for Clinical Trial Recruitment
NIH_toolkitcopy.jpgRecruiting an adequate number of study participants is a major challenge for many clinical researchers. NIH has a recruitment toolkit that offers tips on how to promote participant recruitment and retention at each stage of a clinical trial.
Site Devoted to Compilations of Data On Cell Therapy Trials and Products
Cell Trials Data, a project of the Parent’s Guide to Cord Blood Foundation, was developed to offer a searchable database of all cell trial compilations that have been published to date. The business plan, explained on their website is to enable clients to outsource the effort of creating high quality cell therapy databases. The intended audience includes Pharma/Biotech companies, researchers, business analysts and anyone interested in compiling raw CT data for statistical analysis.
AABB White Paper Aims to Help Facilities Advance Quality Processes
"The Quest for Quality – Quality System Management in the Translational Research Facility," a white paper by AABB Consulting Services, delves into how to achieve and maintain the quality processes necessary to ensure the provision of safe, effective cellular products and quality patient care. A third-party, independent validation of an organization’s quality system can be useful. AABB’s consultants offer no-cost 15-minute consultations to assist organizations in developing quality systems and quality management plans. For additional information or to schedule a consultation, contact AABB Consulting Services.
A Map App to Track Stem Cells: New Imaging Tool for Recording Changes Over Time, Space and Function
The development of measurement tools for stem cell production is challenging, making it hard to determine what makes various new stem cell-related products safe, effective or high- quality. National Institute of Standards and Technology (NIST) scientists developed a system that combines video footage with high-power computation to facilitate evaluating, understanding and quantifying features of cell populations that could be used in related therapies or products. The new image analysis system, the Web Image Processing Pipeline (WIPP), is like a mapping app that allows a user to interact with microscopic views of macroscopic objects. The WIPP app lets users divide a cell culture into real locations that they can explore and reference using a system that simulates GPS points or the quadrants on a geographic map or mapping app. WIPP also allows users to examine a petri dish over time, from any vantage point they choose. A video demonstrating the app is available online.
FOR MEMBERS ONLY
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Members of the AABB Center for Cellular Therapies' subsections meet regularly via teleconference to participate in interactive activities, including developing tools and reference materials, as well as discuss CT topics. For more information visit www.aabbcct.org.

 
The Latest on CT Subsection Activities
Asia Pacific Group (APG): This subsection accommodates members in the Asia/Pacific region on the second Wednesday of each month at 0400 UTC (universal coordinated time) to discuss a variety of topics related to cellular therapies. David Stroncek, MD, Arun Prasath, MS, and Yen-Michael S. Hsu, MD, PhD, lead the discussions. 
Xiuzhi Susan Sun, PhD, presented “Advanced PepGel: a new tool for high performance cell culture, encapsulation, expansion, and translational cell therapies,” Wenchun Qu, MD, PhD, presented "Translational Regenerative Rehabilitation Therapies at Mayo Clinic." Yen-Michael S. Hsu, MD, PhD, who assumed role of subsection Leader in April, presented Clinical Translation of Vascular Niche for Hematopoietic Stem Cell Expansion and Transplant.  Upcoming topics include adoptive immunotherapies and transfusion-associated graft-versus-host disease. AABB invites all members of the APG to take advantage of this member subsection benefit and participate in these quality talks.
 
Regulatory Affairs: This subsection, led by Ljiljana Vasovic, MD, and Olive Sturtevant, MHP, MT(ASCP)SBB, SLS, CQA(ASQ), discussed the special report, "Balancing Safety and Innovation for Cell-Based Regenerative Medicine." Robert J. Tressler, PhD, MS, presented "The Future of Regenerative Medicine and Wound Healing," and AABB posted the presentation under the title Human Platelet Lysate - Potential Uses.
 
CT Management: This group, led by subsection leaders Suzanne Dworsky, MBA, MT(ASCP), and Brian Jones, SBB(ASCP), discussed information important to dealing with emergencies in the laboratory including policies, communication plans, business continuity planning, decisions on assigning essential/non-essential personnel and in- patient/out- patient coverage.
 
Novel Therapies and CT Product Development: This subsection, led by Magali Fontaine, MD, PhD, and Richard Schaefer, MD, welcomed Robert J. Tressler, PhD, MS, who presented Human Platelet Lysate - Potential Uses. Schaefer presented an overview MSC Therapies for GvHD in the Clinic- An Update. Upcoming meeting topics include MSC culture and xenotransplantation.
 
CT Quality Operations: Ed Brindle, MSc, MLT(CMLTO), and Deb Sesok-Pizzini, MD, MBA, hosted guest speaker Sallie Allman, MT (ASCP), CQA(ASQ), who presented Implementation of ISBT 128 Product Labeling at NMDP Centers.
 
CT Product Collection and Clinical Practices: Subsection leaders Tom Spitzer, MD, and Jay Raval, MD, presented case studies and journal club on a variety of topics, including plerixafor and granulocyte colony-stimulating factor mobilization of hematopoietic stem cells for autologous transplantation. Updates from workgroup studies- “Bone Marrow Harvest Transfusion Practices Study” and “HPC, Cord Adverse Events/Reactions Monitoring Study” were presented.
 
CT Product Manufacturing and Testing: Ronit Slotky, PhD, MSc, and Yen-Michael S. Hsu, MD, PhD, discussed various challenges in cell processing, including preparing for the next drug shortage. Mike Halpenny, MLT(CMLTO), was newly named subsection Associate Leader as Hsu assumes the subsection leader role of the APG. The subsection gratefully acknowledges Hsu’s contributions and extends a warm welcome to Halpenny.
 
Cord Blood Subsection: Subsection leaders Salem Akel, PhD, and Gwen Epstein, BSC, RT, discussed a variety of topics with speakers Arun Prasath, M. Phil, MSc, who presented Crossover Banking Model for Singapore Community, and Tami Yonke, MT(ASCP)SBB, MEd, who presented ISBT128 Labeling for Cord Blood.
 
Spanish Language Subsection (SLS): AABB encourages copy_CELLSOURCE_SLS.jpgSpanish-speaking members to join the SLS. Celina Montemayor, MD, PhD, moderates this subsection, which is conducted entirely in Spanish. The SLS projects page showcases content that has been presented to the subsection.
Editor: Christina M. Celluzzi
Contributors: Kathy Loper and Jessica Yozwiak
 
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