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AABB CellSource - April 2016

 
AN UPDATE ON CELL THERAPY NEWS FROM AABB
April 2016

IN THE NEWS

International Cord Blood Symposium 2016 to Offer Technical Workshops

The 14th ICBS, to be held June 9-11 in San Francisco, will bring together global experts in the fields of cord blood and the related areas of hematopoietic stem cell transplantation, banking and therapy. The symposium will host a range of activities. Plenary sessions will highlight advances in cord blood expansion, the latest applications of perinatal cells and cord-derived tissues, and clinical trial results of regenerative cord blood therapies. New this year, attendees will be able to choose among technical workshops on cord unit selection, cryopreservation of cord-derived tissues and other focused topics in informal and interactive roundtable discussions. To register for the symposium or submit an abstract for presentation, visit the ICBS website.

AABB Accepting Abstracts for 2016 AABB Annual Meeting

AABB invites researchers to submit abstracts for the 2016 Annual Meeting, to be held Oct. 22-25 in Orlando, Fla. Submissions from both members and non-members are welcome. Abstract groups encompass scientific and administrative categories. This year, AABB has added a new category: “Immunotherapies: Innate and Adaptive Immunity Cell and Plasma-Derived Therapies” to include all aspects of developing a cellular or plasma-derived immunotherapy product or tissue for clinical research. AABB will present $500 awards to 10 trainees who submit exceptional abstracts. Two individuals in each of the following five categories will receive awards: undergraduate, nursing or medical laboratory science student; specialist in blood banking certificate student; medical or graduate student; medical resident; and fellow or post-doctoral scientist. Abstracts are due May 4 at noon (ET). Selected abstracts will be published in a special supplement of “Transfusion” in September.

Alliance for Regenerative Medicine Releases 2015 Annual Data Report

ARM recently released its annual data report. Containing information provided by Informa, ARM’s data partner, the report details industry-specific statistics compiled from leading gene therapy, cell therapy and other regenerative medicine companies worldwide. The report includes information on total financings, partnering and dealmaking, clinical trials, key data events and ARM’s strategic priorities for 2016.

Miller Launches Insurance Product for Medical Expenses Related to Cord Blood Stem Cell Therapies

Miller Insurance Services, a leading broker in the London market, has launched a new product to assist with medical expenses related to cord blood stem cell therapies. The new insurance product fills a gap in critical illness insurance by covering medical expenses for insured patients who receive therapies associated with banked umbilical cord blood for specified medical conditions. AABB is pleased that Miller recognizes the value of AABB accreditation for cord blood banks.

GRID Helps to Identify Donors Globally

The World Marrow Donation Association, or WMDA, developed a system called the “Global Registration Identifier for Donors,” or GRID. GRID provides unique identifiers for potential donors to facilitate communication and to prevent errors in donor identification. Designed to be used by electronic process control systems, GRID includes a standard format for a human-readable version. WMDA has a Memorandum of Understanding with the ICCBBA, the international standards organization responsible for the management and development of the ISBT 128 Standard. ICCBBA will assign and manage the list of issuing organization numbers and support the development of associated standards documents. Representatives from WMDA and ICCBBA have been working together to develop the GRID structure. A WMDA GRID Implementation Working Group will assist with delivering and implementing this concept within the unrelated donor registry community. It will provide support and guidance to issuing organizations on all operational matters related to GRID, as well. The GRID implementation guide is available to assist issuing organizations in the transition to GRID.

CT Attribute “Non-Mobilized” to Be Discontinued in ISBT 128

ICCBBA’s Cellular Therapy Coding and Labeling Advisory Group decided to discontinue use of the term “non-mobilized” to describe ISBT 128 cellular therapy (CT) products. ICCBBA will remove the attribute from existing product descriptions and product formulas. The group notes that during the transition period, the attribute will appear in some descriptions but not in others. ICCBBA expects the change to be completed by the time it posts the updated ISBT 128 Product Description Code Database and the ISBT 128 Standard Terminology for Medical Products of Human Origin that are posted in May. Comments, questions and concerns may be addressed to iccbba@iccbba.org.

REGULATORY AND GOVERNMENT UPDATE

FDA Publishes Donor Screening Recommendations to Reduce Risk of Transmission of Zika Virus by HCT/Ps

The United States Food and Drug Administration, or FDA, published a final guidance containing recommendations for establishments that make eligibility decisions for prospective donors of human cells, tissues and cellular and tissue-based products (HCT/Ps). The March 2016 guidance, which supplements the August 2007 guidance, “Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products,” identifies Zika virus as a relevant communicable disease agent or disease and is meant for immediate implementation. It highlights the potential risk of transmission of Zika virus by HCT/Ps, including hematopoietic stem or progenitor cells from cord and peripheral blood, corneas, bone, skin and heart valves. The FDA recommends completing a medical record review for all prospective donors to confirm that they are free from risk factors for, or clinical evidence of, Zika virus infection. The recommendations are different for living and cadaveric donors of HCT/Ps.

FDA Publishes Guidance for Adverse Reactions Related to HCT/Ps Regulated Solely as 361 Products

The FDA published a final guidance, “Investigating and Reporting Adverse Reactions Related to Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Regulated Solely under Section 361 of the Public Health Service Act and 21 CFR Part 1271,” to provide establishments that manufacture HCT/Ps with recommendations for complying with 21 CFR 1271, Subparts D and E. Furthermore, this guidance provides general user instructions for reporting HCT/P-related adverse reactions using the MedWatch mandatory Form FDA 3500A. It provides recommendations for identifying which regulatory requirements apply to investigating and reporting adverse reactions, how to conduct an adverse reaction investigation, and how to appropriately report adverse reactions.

AABB Updates HPC DHQ Materials

The AABB Interorganizational Donor History Questionnaire (DHQ)-Hematopoietic Progenitor Cell (HPC) Task Force revised the DHQs and accompanying materials. The new version 1.5 HPC, Apheresis and HPC, Marrow DHQ; version 1.2 HPC, Cord Blood DHQ; and accompanying materials conform with the FDA’s March 2016 final guidance, Donor Screening Recommendations to Reduce the Risk of Transmission of Zika Virus by Human Cells, Tissues, and Cellular and Tissue-Based Products. These versions are for immediate implementation. Individuals may contact regulatory@aabb.org with questions or for additional information.

RESEARCH FOCUS

“Transfusion” Supplement ‘Primes’ Readers on MSCs

Speakers participating in the “Mesenchymal Stromal Cells: Are They Ready for Prime Time?” workshop — held prior to the 2015 AABB Annual Meeting — share summaries of their presentations, which were compiled in a supplement to the April issue of AABB’s journal “Transfusion.” The summaries cover a variety of topics important to understanding the clinical role of MSCs; how MSCs are characterized, manufactured, and assessed; and strategies to improve their therapeutic potential. The role of blood establishments as material suppliers, manufacturers and distributors for these new therapies is also addressed.

A Study with A ‘Twist1’: Predicting Activity of Mesenchymal Stem Cells

MSCs are being evaluated in a number of trials with broad-based therapeutic effects; however, they are currently not well-defined based on physical, phenotypic and functional properties. New findings from Phinney et al. link stem and effector function mechanistically to provide a unifying framework that models the functional complexity of the cell populations. The findings support the notion that stem and effector functions are coordinately regulated at the cellular level by the transcription factor called TWIST1. By examining levels of TWIST1 in different human donor populations, the researchers found that higher levels of TWIST1 produced more angiogenic effects - boosting new blood vessel growth, while lower levels produced more anti-inflammatory and immuno-suppressive effects. Manipulating levels of TWIST1 in both cells and animal models resulted in a predictable change in the functional attributes of the stem cells. Based on their findings, the scientists developed a Clinical Indications Prediction or CLIP scale, which predicts the therapeutic potential of MSCs for a given disease indication based on their levels of TWIST1. According to the researchers, the proposed CLIP scale appears to accurately predict indications and contra-indications which would direct whether the stem cells would be beneficial or not in a given therapy.

Wound Healing: A Brief Review of the Role of MSC-Based Therapies

MSCs possess properties that can be used therapeutically in cutaneous wound healing. Data indicate that MSCs accelerate wound closure, increase angiogenesis, promote resolution of wound inflammation, regulate extracellular matrix remodeling, and stimulate regeneration of skin characterized by normal architecture and function. Studies have employed novel methods to amplify MSC delivery and efficacy. In their review, Lee et al. describe the novel studies that show promise for the development of MSC-based wound-healing therapies and provide direction for further research in this field.

Rapid, ALDHbr-Based Cord Blood Potency Assay May Predict Engraftment

Banked unrelated umbilical cord blood, or UCB, is a valuable source of allogeneic cells for hematopoietic stem cell transplantation. The potency, or ability of a UCB unit to restore hematopoiesis following transplantation, is key. Characteristics used to assess potency include the total nucleated count, CD34+ cell content, and hematopoietic progenitor colony-forming units, or CFU. UCB potency is typically assessed pre-cryopreservation. Changes may occur following freezing, storage and thawing. Shoulars et al. described the development and validation of an in vitro flow cytometric assay measuring aldehyde dehydrogenase, or ALDHbr, content of a UCB unit segment to evaluate potency. High ALDH expression has been reported for precursor cells of various lineages, including hematopoietic cells. Using the CFU assay as the best lab surrogate for clinical engraftment, the researchers compared the correlation of CFU content of 3,908 segments with several flow parameters. The results suggest that the ALDHbr cell content is the parameter most highly correlated with CFU and that the ALDHbr content is likely the best parameter to predict the ability of a UCB unit to engraft. Shoulars presented some of the findings on a recent AABB cord blood subsection Web conference.

T Cell-Derived iPSCs Used to Model Neurological Disease

Due to the difficulties associated with examining the human central nervous system, researchers have commonly studied neurological diseases using animal models and immortalized neural cell lines. However, recent advances in technologies using human induced pluripotent stem cells, or iPSCs, have enabled scientists to model these diseases. By culturing patient-specific neural cells, investigators can elucidate pathogenic mechanisms and perform drug screening. T cells are an ideal source of patient-specific iPSCs because they can be easily obtained from samples; however, iPSCs retain an epigenetic memory relating to their cell of origin that restricts their differentiation potential. To overcome this, Matsumoto et al. developed a neurosphere-based robust differentiation protocol enabling T cell-derived iPSCs to differentiate into functional neurons. Neurons derived from T cell-derived iPSCs generated from a pediatric patient with Parkinson’s disease exhibited several Parkinson’s disease phenotypes. The researchers concluded that these cells are a useful tool for modeling neurological diseases.

Study Confirms That an Allergy Can be Acquired From Donor Stem Cells

The phenomenon of allergy transfer from an allergic donor to a non-allergic recipient via hematopoietic cell transplantation has been described by several reports; however, researchers could not yet conclusively show that the donors’ cells elicited the allergic reaction in the recipient. Garzorz et al. show for the first time that allergy transfer can be mediated by the donor's cells in a case presentation involving a 46-year-old male patient who — for the first time in his life — experienced two episodes of oral allergic syndrome upon kiwi consumption after having received myeloablative hematopoietic stem cell transplantation from his kiwi-allergic sister. The researchers used a variety of tests, including a customized kiwi extract, to prove the contribution of kiwi-specific T and B cells in both the kiwi-allergic recipient and donor and to suggest that this approach could serve as a model for future studies.

EVENTS, OPPORTUNITIES, RESOURCES

AABB CT Audioconferences and Webinars

Information on 2016 CT audioconference and webinar programs is posted on the AABB website.

Upcoming Webinars:
5/12 Perinatal Cells and the Landscape of Ancillary Cord Blood Banking Services (#1610)
6/22 Adipose-Derived Stem Cell Banking for Reconstructive and Regenerative Medicine (#1611)

Upcoming Audioconferences:
5/9 Expansion of Umbilical Cord Blood Stem Cells for Transplantation (#164950)
7/20 Hematopoietic Stem Cell Collection: Devices, Product Characteristics and Optimization (#164951)
9/28 Process Development & Challenges in Large-Scale Manufacture of MSC Therapeutics (#164952)
11/02 How to Develop Competency Tests for Training Programs (#164953)

Go Cellular! AABB’s Newest CT Book Has Arrived

Cellular Therapy: Principles, Methods, and Regulations, 2nd edition, is now available through the AABB Marketplace. Designed as a compilation of state-of-the-art practices and methods for developing and producing cellular therapy products, this manual offers several examples and templates for laboratory document preparation. The revised edition includes information on international regulation of cell therapy products; pre-clinical testing; early phase trial development; and biorepositories with operational, regulatory and ethical considerations for storage of research samples.

The AABB HUB Links to ‘Gravity’ and QC

AABB’s online collaborative space, the AABB HUB, serves as a platform for members to help each other. Members of the Cellular Therapies discussion group enjoyed recent exchanges on such topics as specific gravity of cord blood (Where do you find literature and how do you apply it in practice?), trypan blue viability assays (What are potential controls to include?), flow cytometry QC (What controls should you use?), and cord blood collection (Where can you find a training video?). The AABB HUB enables participants to ask and answer questions, share ideas and network with colleagues from around the world. If you have not yet tried it, take a peek and start connecting with your cellular therapy peers today.

FOR MEMBERS ONLY

The Latest on CT Subsection Activities

Asia Pacific Group, or APG: Accommodating members in the Asia Pacific time zones, this subsection discuss a variety of topics related to cord blood. APG holds teleconferences the second Wednesday of each month at 0400 UTC (universal coordinated time). Interested individuals are encouraged to enroll and participate. Slides from topics presented this past quarter can be found on the APG subsection projects webpage: “HLA Basics and Beyond,” presented by Sharon Adams, MT,CHS(ABHI)” and “Regulatory Convergence for Advanced Therapies – an APEC Initiative” presented by Srinivasan N KELLATHUR, PhD.

Regulatory Affairs: Leader Ljiljana Vasovic, MD, and Associate Leader, Olive Sturtevant, MHP MT(ASCP)SBB/SLS, led discussions on an assortment of regulatory issues, including the most recent draft guidances available for public comment. As a service to members interested in commenting on current guidance documents, AABB will accept, review and then submit collective comments to the FDA. Members are encouraged to contact the AABB regulatory affairs department, regulatory@aabb.org, with any comments prior to the deadlines on announcements to ensure a timely review by AABB. Past comments related to CT submitted by AABB can be found on its website. In addition to reviewing guidance documents, Olive Sturtevant presented “Zika Virus: A Brief Overview.”

CT Management: Leader, Suzanne Dworsky, MBA, MT(ASCP), and Associate Leader Brian Jones, SBB(ASCP), led discussions revolving around a variety of topics related to efficient management of the laboratory. Topics included product inventory management; clinical trial management from a laboratory perspective; staffing models; and compensation, information systems and communication systems.

Novel Therapies and CT Product Development: Subsection leaders Pampee Young, MD, PhD, and Magali Fontaine, MD, PhD, facilitated several ‘novel’ discussions welcoming a presentation “Chimeric Antigen Receptors (CARs)” given by Vijay Bhoj, MD, PhD, and Andrew Fesnak, MD.

MSC Workgroup: Richard Schaefer, MD, who leads the MSC Workgroup, presented information on potency testing. Materials discussed by the Workgroup can be found on the Subsection Projects page. Workgroup sessions are held quarterly as part of the Novel Therapies and CT Product Development subsection schedule. Members of the subsection interested in MSCs are encouraged to participate. In addition, Schaefer and co-editor Selim Kuci, MD, launched "Mesenchymal Stem/Stromal Cells – An update" on BioMed Central (Open Access Publisher) that lists a collection of state-of-the-art articles on MSC basic biology and translational and clinical aspects, with the goal of providing updates that highlight and broaden the current understanding of MSCs.

CT Quality Operations: Leaders Kathy Fortune, BS, MT(ASCP), Ed Brindle, MSc, MLT(CMLTO), and Deb Sesok-Pizzini, MD, MBA, led discussions on Zika virus — the donor history questionnaire, and improving quality by “doing the right thing even when no one is watching,” and other topics.

CT Product Collection and Clinical Practices: Subsection Leaders Tom Spitzer, MD, and Jay Raval, MD, and Associate Leader Joseph ‘Yossi’ Schwartz, MD, MPH, held a journal club discussion on hematologic malignancy and the effects of donor characteristics on survival following unrelated donor transplantation. They also reviewed preliminary results from their “Bone Marrow Collection Quality Improvement Initiative Survey,” which was developed with AABB members and representatives from ASBMT, ASFA, DOD, CIBMTR, and NMDP.

CT Product Manufacturing and Testing: Subsection Leader Lizette Caballero, MLS(ASCP)CM, hosted several well-attended presentation-discussion sessions. Lizette spoke on “Lessons Learned When Preparing for an Industry-Sponsored Clinical Study.” Vasiliki Kalodimou, PhD, presented “Gating Strategy in Cord Blood Samples” and Michael Ancharski, BS, ASCPCM, with Ronit Slotky, PhD, presented a talk “BD FACSCanto II Flow Cytometer Quality Control.”

Cord Blood Subsection: This subsection, hosted by Leader Salem Akel, PhD, and Associate Leader Gwen Epstein, BSC, RT, welcomed several speakers. Sumithira Vasu, MBBS, presented “Expansion of Large Numbers of CD56+ NK Cells from a Minute Fraction of Cryopreserved Cord Blood for Immunotherapy after Transplantation.”Joan Lorenz, MT(ASCP), spoke on “The Microbiology Lab in Cord Blood Bank Facility: Operational Aspects and Merits,” and Kevin Shoulars, PhD, discussed “Development and Validation of a Rapid, Aldehyde Dehydrogenase Bright-based, Cord Blood Potency Assay.”

 

Editor: Christina Celluzzi
Contributors: Kathy Loper and Brandon Sandine

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