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AABB CellSource - April 2019

AABB Cellsource
An Update on Cell Therapy News from AABB  | APRIL 2019
HIGHLIGHTS
  • Annual Meeting Abstract Deadline This Week
  • CT Subsection Activity Updates
IN THIS ISSUE
EDUCATIONAL RESOURCES
Upcoming AABB eCasts in CT:
Registration for AABB eCasts 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.
Cellular Therapies Certificate Program Provides CME Opportunity for CT Professionals 
The AABB 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 an opportunity to earn continuing education credits (CMEs).
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Cellsource
 
Editor:
Christina M. Celluzzi, PhD, MS
 
Contributors:
Kathy Loper, MHS, MT(ASCP)
Jessica Yozwiak, MS
 
AABB Center for Cellular Therapies
4550 Montgomery Avenue, Suite 700, North Tower, Bethesda, MD 20814 +1.301.907.6977
 
Deadline to Submit 2019 Annual Meeting Abstracts Extended to April 19
AABB extended the deadline to submit abstracts for the 2019 AABB Annual Meeting to April 19. Abstract categories include various scientific and administrative topics in transfusion medicine and cellular therapies. Abstract preparation and submission guidelines are available online. Submissions from both members and nonmembers are welcome. Those whose abstracts are accepted are required to attend the 2019 Annual Meeting, to be held Oct. 19-22 in San Antonio, where they will have the opportunity to present their work to more than 4,500 attendees from throughout the world. AABB will publish accepted abstracts in a special supplement of the September issue of Transfusion.
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AABB-ISCT Joint Working Group Updates its Collaborative Projects 
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AABB and the International Society for Cell and Gene Therapy (ISCT) formed a joint working group (WG) in 2014 to collaborate on projects in the cellular therapy (CT) arena involving issues important to both organizations.  
 
The WG's first project team addressed issues surrounding the production and standardization of pooled human platelet lysate (pHPL), a topic motivated by the increasing need for a nonanimal-derived source of growth supplements for cell culture to support CT product development. At both the AABB and ISCT annual meetings in 2017, the pHPL project team presented information on standardization of pHPL production, measures for evaluation and pathogen-reduction treatment of pHPL products, and differences between pHPL and fetal bovine and calf serums. This project team has prepared a manuscript, "Gaps in the Knowledge of HPL Production as a Cell Culture Supplement for Cell Therapy," that summarizes their findings to be published jointly in the journals Transfusion and Cytotherapy.
 
The WG is currently working with a second project team on considerations for establishing a CT product stability program. The aim of a stability program is to determine a product’s shelf life and to define best practices for storage. Differences in product type and regulatory pathways often make this a challenging task. A goal of this project team is to create reference documents and/or other communication materials to help with the design of a stability program for hematopoietic progenitor cell (HPC) products, including a standardized ‘template’ for use in performing stability testing of HCT/Ps. The project team members represent a wide selection of subject matter experts from academic institutions and industry.
WMDA Celebrates Its 25th Anniversary
On Mar. 21-22, the World Marrow Donor Association celebrated its 25th anniversary with a conference in Noordwijk, the Netherlands. Over 230 members from 45 countries gathered to hear the latest developments on donor registries and cord blood banks. Sessions included presentations on cellular therapies (e.g., CAR-T cells), the influence of social media, engagement of ‘GenZ’ as voluntary donors, and the pros and cons of a global cord blood bank. Festivities ended with a big party on the beach. The WMDA looks back on two amazing days and is set to continue its important work to realize its vision to ensure patients worldwide have equal access to high quality cells for transplantation from donors whose rights and safety are protected.
AABB Posts its 2019 Advocacy Agenda
The AABB Board of Directors approved the Association’s 2019 advocacy agenda. This document will guide the association’s public policy and advocacy efforts for the year. AABB will focus on advocating for policies that support a sustainable blood system; access to cellular therapies; coverage and reimbursement for blood, blood products, transfusion medicine and cellular therapies; patient and donor care and safety in transfusion medicine and cellular therapies; and research to support access to safe, clinically effective transfusion medicine and cellular therapies. 
CT Subsection Enrollment is Benefit of AABB Membership
AABB offers a variety of benefits including membership in its CT subsections. AABB enjoys a global membership and offers 5 subsections covering a variety of topics spanning cord blood, regulatory, quality, management, clinical, and hot-topic issues to support the growing needs of its CT professionals. Participation is a way to cultivate a collegial and informed community. Visit the AABB website to learn more about how to become an AABB member and enroll in a CT subsection to get involved. 
 
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HRSA Seeks Comments on Stem Cell Therapeutic Outcomes Database Information Collection
The Health Resources and Services Administration (HRSA) is requesting comments regarding the burden estimate on specific topics that are listed in the March 7 Federal Register notice for the Stem Cell Therapeutic Outcomes Database. Information collected on the forms outlined in the ‘‘Total Estimated Annualized Burden Hours’’ section is needed to monitor the clinical status of transplantation and provide the Secretary with an annual report of transplant center-specific survival data. Comments are due May 6.
FDA Releases Guidance Document on Standards
FDA released its final guidance document, "Standards Development and the Use of Standards in Regulatory Submissions Reviewed in the Center for Biologics Evaluation and Research" in March. The purpose of this guidance is to describe the CBER recommendations on the use of standards in product development and control, as well as the use of such standards in CBER’s managed review process. CBER may, when it deems appropriate, take accreditation standards established by accreditation organizations, including AABB, into consideration when assessing compliance with CBER regulatory requirements. The guidance states, "CBER has, for example, recognized certain blood donor history questionnaires developed by AABB as acceptable for use by blood collection establishments to collect donor history information from donors of blood and blood components."
Zika Virus Transmission Risk, Color-Coded World Map Available on CDC Website
The Centers for Disease Control and Prevention (CDC) has updated information on its Blood and Tissue Safety web page, used to communicate epidemiological information about Zika virus (ZIKV) to the blood and tissue collection community. The web page includes a world map of areas with risk of Zika for countries and territories outside of U.S. states. The CDC has developed a new process to indicate risk for these areas that assigns one of four risk categories and shades each one with a specific color (according to FDA, “Red” or “Purple” indicates increased risk of ZIKV transmission):
 
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  • Country or territory with current Zika outbreak (Red)  
  • Country or territory with any prior or current reports of mosquito-borne Zika transmission (Purple)
  • Country or territory with the vector and no reported mosquito-borne Zika transmission (Yellow)
  • Country or territory with no mosquitoes that spread Zika (Green)
 
HHS Announces Renewed ACBSCT Charter
The U.S. Department of Health and Human Services (HHS) announced it had renewed the charter of the Advisory Council on Blood Stem Cell Transplantation (ACBSCT), effective Feb. 19. ACBSCT advises and makes recommendations to the HHS Secretary on matters related to the activities of the C.W. Bill Young Cell Transplantation Program and the National Cord Blood Inventory Program. The renewal, published in the Federal Register, authorizes the Council to operate until Feb. 19, 2021. 
FDA Strives to Stop Unapproved Stem Cell Products While Supporting Their Legitimate Development 
Stem cell therapies offer the potential to treat — or even cure — diseases or conditions for which few effective treatment options exist. In addressing its role in protecting public health by assuring safety, effectiveness and security of biologics, the U.S. FDA Commissioner and the Biologics Center Director released a statement on FDA’s continued efforts to stop stem cell clinics and manufacturers from marketing unapproved products that put patients at risk, while advancing the agency’s commitment to help move legitimate stem cell product development forward under existing agency regulations. The complete statement is available online. Despite regulations for stem cell therapy development, there is a false premise currently circulating that stipulates that a product originating in a person then manipulated and transplanted back for another purpose is not subject to FDA regulation. Stem cell products can create unique and serious risks. When a product undergoes more than minimal manipulation, its characteristics may be changed in significant and potentially dangerous ways.
 
FDA uses a variety of tools to advance an efficient path for the safe and effective development of novel regenerative medicine products and to help foster beneficial new innovations, including providing advice through CBER’s INTERACT meeting process and the use of expedited programs, such as Regenerative Medicine Advanced Therapy designation, for products that qualify.
 
The FDA continues to encourage sponsors to discuss novel clinical development approaches with the agency, as in the collaborative development model highlighted in its guidance, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.

 
A Temporal Analysis of FDA and the US Direct-to-Consumer Marketplace for Stem Cell Interventions
A plethora of businesses in the U.S. currently advertise a wide range of non-FDA-approved stem cell interventions. Knoepfler and Turner present a novel systematic temporal analysis of U.S. companies engaged in direct-to-consumer marketing of putative stem cell treatments. Between 2009 and 2014, the number of new U.S. stem cell businesses with websites grew rapidly, at least doubling on average every year. From 2014 to 2016, approximately 90-100 new stem cell business websites appeared per year. In contrast, from 2012 to the present, regulatory activity in the form of FDA warning letters has been limited. The authors discuss how these data point to a problematic disconnect between a rapidly expanding U.S. direct-to-consumer stem cell industry and limited FDA oversight of this marketplace and suggest that more consistent, timely and effective FDA actions are urgently needed (subscription required for full access).
Cellular Therapies in Trauma and Critical Care Medicine
Transfusionsuppltrauma2019_184574.pngTrauma and its secondary complications are leading causes of morbidity and mortality worldwide. Despite the large number of patients treated with traumatic injuries, there are few therapeutic options to decrease morbidity and mortality and to improve outcomes. In recent years, the field of cellular therapies (CT) and regenerative medicine has grown, generating interest in the potential to address many different types of health conditions. In trauma and critical care applications, CT is being investigated for both the acute and chronic phases of injury. Although still in its early stages of development, animal and human studies demonstrate the promise of CT for trauma‐induced conditions such as traumatic brain injury (TBI), spinal cord injury (SCI), organ failure (such as acute respiratory distress syndrome, or ARDS, and acute kidney injury, or AKI), orthopedic trauma, burns, and ischemia/reperfusion insults.
 
The special issue, “A Supplement to Transfusion: Cellular Therapies in Trauma and Critical Care Medicine,” includes a selection of papers focused on topics presented at the biannual conference, Cellular Therapies in Trauma and Critical Care Medicine (CTTACC).
The Value of the Post‐Thaw CD34+ Count With and Without DMSO Removal in the Autologous Stem Cell Transplantation Setting
CD34+ cell count correlates with engraftment potency after autologous stem cell transplantation. Assessment of CD34+ mainly occurs after apheresis and before cryopreservation with dimethyl sulfoxide (DMSO). The influence of post-thaw CD34+ cell numbers over time to engraftment is not well studied, and determination of post-thaw CD34+ cell counts is challenging for a variety of reasons. Rohner et al. describe their retrospective study in which they systematically assessed the value of post-thaw CD34+ cell counts in autologous grafts with and without DMSO removal in an article published in Transfusion (subscription required for full access).
The Lifetime of Cells
Cells regenerate at different rates during the aging process. A brief video, “How Old is Your Body?” illustrates the beauty of cells’ constant changes and may inspire the viewer to feel ‘rejuvenated.’
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CRISPR Described in Animated Feature
An organism's fundamental features can be changed in record time using gene editing tools such as CRISPR. Andrea M. Henle describes what CRISPR means and the science behind the technology in this TedEd narrated animation.
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Workshop on Preservation of Cellular Therapies
BioCor at the University of Minnesota is offering a class “Preservation of Cellular Therapies,” May 21-22 in Minneapolis. The course, offered in person and via simultaneous webcast for those unable to attend, covers a variety of topics, including fundamentals of cryopreservation, protocol development, regulatory issues, designing a storage facility, containers, reagents and equipment. Information is available online or by contacting biocor-pct@umn.edu.
Pilot Studies - Common Uses and Misuses
Question_Mark_and_Person50x50_184480.jpgThe goal of pilot work is not to test hypotheses about the effects of an intervention, but rather, to assess the feasibility/acceptability of an approach to be used in a larger scale study. A pilot study does not address the question, “Does this intervention work?” Instead it is designed to gather information to help determine, “Can I do this?”
 
To learn more, visit uses and misuses of pilot studies on the NIH website. For a rigorous definition, Moore et al. in their article discuss recommendations for the design, analysis and sample size justification of pilot studies in clinical and translational research.
What Informed Consent Means to FDA
The term “informed consent” may be mistakenly viewed as merely getting a research participant's signature on the consent form. FDA believes that obtaining a research participant's verbal or written informed consent is only part of the process. Informed consent involves providing a potential participant with:
  • Offering adequate information to allow for an informed decision about participating in the clinical investigation.
  • Facilitating the potential participant's understanding of the information.
  • Providing an appropriate amount of time for the prospective participant to ask questions and discuss the research protocol and participation with family and friends.
  • Obtaining the potential participant's voluntary agreement to participate.
  • Continuing to provide information as the clinical investigation progresses or as the subject or situation requires.
Visit the FDA website to learn more about informed consent.
ISBT 128 Lessons in a Quiz
Cellular therapy facilities registered with ICCBBA were asked to participate in a quiz to determine the educational needs of users regarding appropriate selection of ISBT 128 Product Codes. The 21-question quiz covered user information and selection of attributes, collection types, division codes, and product description codes. ICCBBA has made the knowledge-based questions available, including the rationale for the answer selections.

 
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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
The AABB Center for Cellular Therapies  encourages readers to explore information about the CT subsections and how they can get involved.

 
Newly realigned CT-CET and QRM Subsections Make Debut
Two newly-realigned subsections began their monthly calls in February:
  • Quality, Regulatory and Management (QRM) — which combined the former Quality Operations, Regulatory and CT Management subsections and
  • CT Current and Emerging Topics (CT-CET) — comprised of former subsections Product Manufacturing and Testing, Product Collections and Clinical Practices, and Novel Therapies and CT Product Development
 
CT-CET: The first meeting on Feb. 21 covered PBSCs for autologous transplant. On a subsequent call, a former AABB Annual Meeting keynote speaker, Daniel Kraft, MD, of the Singularity University, presented “The MarrowMiner: A Novel, Safe, Effective, and Minimally Invasive Device for the Harvest of Bone Marrow Cells.”
 
QRM: The first meeting was held on Feb. 26 for members to become acquainted with their new colleagues. Other discussions centered around FDA guidance documents, including, “Evaluation of Devices Used with Regenerative Medicine Advanced Therapies” and “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.” The former Quality Operations subsection survey on environmental monitoring of 361 products was reviewed.
 
Cord Blood: Discussion covered topics on a wide range of issues, including unproven stem cell therapies.
 
Asia Pacific Group (APG): The APG continued its diverse lineup of guest speakers. In recent months, APG members welcomed Erwin Cabana of ICCBBA who presented "ISBT 128: Overview and Implementation for Cellular Therapy," Peihua (Peggy) Lu, MD, of Lu Daopei Hospital and Lu Daopei Institute of Hematology, Beijing, China, who presented “CAR-T Treatment for Refractory or Relapsed (R/R) B-cell Acute Lymphoblastic Leukemia (ALL), and Aisha Khan, from the Univ. of Miami, who spoke on “Clinical Trials: Moving Cell-Based Therapies to the Clinic.”
 
Spanish Language Subsection (SLS): The SLS subsection is conducted entirely in Spanish. This quarter, members held journal club and welcomed guest presenter, Sandhya R. Panch, MD, of NIH, who provided a presentation, “Importance of Logistics in Cell Therapy Manufacturing.”


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