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AABB CellSource - January 2016

 
AN UPDATE ON CELL THERAPY NEWS FROM AABB
January 2016

IN THE NEWS

BEST Requests AABB Member Input on Survey

The BEST Cellular Therapy Team is considering a project to investigate viability testing of cryopreserved and thawed cord blood products. To help determine the project’s feasibility and utility, a project team led by Minoko Takanashi, MD, PhD, of the Japanese Red Cross, has developed a survey of 10 to 28 questions. The team asks AABB members to participate in the survey, which should take about 10 minutes to complete. Responses are requested by Feb 1.

AABB and ISCT Create Joint Working Group to Explore Common Interests

AABB and the ISCT have established a Joint Working Group, formed as a collaborative effort to identify and facilitate projects of common interest to both organizations and their overlap of members. The working group comprises three AABB and three ISCT representatives, with at least one member of each from their respective boards of directors. The first project will tackle preparation of pooled human platelet lysate, or HPL, to address the increasing need for a non-animal derived source of cell culture growth supplement to support cellular therapy product development.

Global Survey Assesses Training and Evaluation of Cell Processing Staff

Data from a recent survey developed and analyzed by the Alliance for Harmonisation of Cellular Therapy Accreditation, or AHCTA, reveal that cell processing facilities combine a variety of methods to train and evaluate personnel. Surveying different types of cell processing facilities from around the world, ACHTA’s goal was to develop recommendations on staff qualifications, training and competency — with an emphasis on employees responsible for processing minimally manipulated products. The analysis, published in “Cytotherapy,” could be helpful to both new and established facilties in decision-making on training and competency programs.

Induced Neurons — Valuable New Tool for Studying Aging?

Researchers have developed a new way to create brain cells that resemble those in older people’s brains. These cells may help scientists study age-related diseases like Alzheimer’s and Parkinson’s. Dr. Gage et al. from the Salk Institute Laboratory of Genetics developed a technique to convert skin cells directly into neurons that were shown to retain the signatures of aging — providing a valuable tool for study. While the work only tested the effectiveness of brain cells, a similar method may help researchers generate heart and liver cells, as well.

Changing Understanding of the Cell Cycle May Lead to New Treatment Approaches

Ground-breaking new research suggests that the current scientific understanding of the human cell cycle may need revision. Science students generally learn that DNA replicates in the synthesis or S-phase (between G1 and G2). However, in their article appearing in “Nature” Minocherhomji et al. show this is not always the case because some regions replicate only after the cell enters mitosis, or cell division. These findings and reconsiderations of current thinking could lead to the development of new therapeutic approaches targeting different types of cancers.

Regenerative Medicine Gets Japanese Drugmakers’ Attention

Takeda Pharmaceuticals and other Japanese drugmakers are increasing their involvement in regenerative medicine and stem cell research, viewing these areas as a future growth market. Plans include research with induced pluripotent stem cells to develop treatments for cancer, heart failure and diabetes. Sumitomo Dainippon Pharma is reportedly involved in a partnership with Hitachi that aims to develop “mass-produced” — rather than patient-specific — stem cell treatments for Parkinson's disease. Fujifilm acquired Cellular Dynamics to manufacture stem cells, and Tokyo-based Astellas Pharma is buying U.S. biotech Ocata Therapeutics for access to its stem cell treatment for age-related macular degeneration.

REGULATORY AND GOVERNMENT UPDATE

President Signs Stem Cell Reauthorization Act Into Law

President Obama signed the Stem Cell Therapeutic and Research Reauthorization Act of 2015 into law on Dec. 18. The new law provides $150 million for bone marrow transplant programs and $115 million for the National Cord Blood Inventory over the next five years. Specifically, the legislation authorizes $23 million per year for grants to public cord blood banks to promote cord blood inventory growth and the collection of diverse cord blood units, as listed on the Be the Match Registry. The registry, which is administered by the National Marrow Donor Program, currently comprises 11 million adult volunteer bone marrow donors and 193,000 donated cord blood units.

Public Hearing on Draft Guidances Relating to Regulation of Human Cells, Tissues, and Cellular or Tissue-Based Products

The FDA announced a one-day public hearing to obtain input on four draft guidances relating to HCT/Ps. The public hearing is April 13 at the FDA White Oak Campus. More information can be found in the Federal Register.

Homologous Use of Human Cells, Tissues, and Cellular and Tissue-Based Products; Draft Guidance for Industry and Food and Drug Administration Staff

The draft guidance document provides HCT/P manufacturers, health care providers, and FDA staff, with recommendations for applying the criterion of ‘‘homologous use’’ as it applies to HCT/Ps. The interpretation and application of the homologous use criterion and related definitions have been of considerable interest to industry stakeholders since they were first proposed during the Agency’s rulemaking on HCT/Ps. Read the Federal Register document. Comments can be mailed to regulatory@aabb.org by March 18 to be compiled and submitted by AABB.

RESEARCH FOCUS

Adipose-Derived MSCs Prevent Systemic Bone Loss in CIA

Rheumatoid arthritis, or RA, is an autoimmune disease characterized by chronic inflammatory synovitis leading to joint destruction and systemic bone loss. Inflammation-induced bone loss is mediated by increased osteoclast formation and function. Current anti-rheumatic therapies primarily target suppression of the inflammatory cascade but achieve limited or no success in controlling progression of bone destruction. Mesenchymal stem cells, or MSCs, are involved in tissue repair and demonstrate immunomodulatory properties. MSCs show promise in treating various autoimmune and degenerative diseases. Their role in preventing bone destruction in RA is not yet understood. Garimella et al. investigated the effect of adipose-derived MSCs, or ASCs, on in vitro formation of bone-resorbing osteoclasts and pathological bone loss in the mouse collagen-induced arthritis, or CIA, model of RA. The researchers provide strong evidence that ASCs ameliorate inflammation-induced systemic bone loss in CIA mice by reducing osteoclast precursors and promoting immune tolerance.

Regeneration of Full-Thickness Rotator Cuff Tendon Tear Following MSC Injection

Rotator cuff tendon tear is one of the most common causes of chronic shoulder pain and disability. Evaluating gross morphology, histology and motion activity, Gi-Young Park et al. report the therapeutic effects of ultrasound-guided human umbilical cord blood-derived MSCs into the rotator cuff tendon of rabbits. The MSCs contributed to regeneration of the full-thickness rotator cuff tendon tear without surgical repair, demonstrating the effectiveness of local injection.

Current Technologies for Tissue Engineering and Stem Cell Biology

Stem cells have great potential in many therapeutic areas. With conventional cell culture methods, however, it is difficult to achieve in vivo-like microenvironments in which a number of well-controlled stimuli are provided for growing highly sensitive stem cells. Microtechnology-based platforms offer the advantages of high precision, control, scalability and reproducibility and enable imitation of the complex physiological context in vivo. This capability may fill the gap between the present knowledge about stem cells and that required for clinical stem cell-based therapies. DoYeun Park et al. in their review in “Stem Cells Translational Medicine” discuss the various types of microplatforms on which stem cell microenvironments are mimicked, assigning categories based on practical uses for researchers. The review covers in vitro production of embryoid bodies and stem cell aggregates; factors controlling stem cell behavior; future research directions leading to scaled-up systems or organs-on-a-chip; regulation of induced pluripotent stem cells; and genetic states of stem cells on microplatforms.

Is Analysis of Apheresis Data Helpful for Production of Regulatory T Cells for GvHD?

In recent years, little has been reported on apheresis parameters such as yield, recruitment factor (RF) and collection efficiency (CE) regarding regulatory T cells (T regs) and other T-cell subsets in peripheral blood stem cells, or PBSCs. In their study appearing in “Vox Sanguinis,” Strobel et al. assessed the content of PBSC concentrates and related apheresis parameters. Donors, patients and PBSC concentrates were characterized for CD45+ cells, CD34+ cells, lymphocytes and an array of T-cell subsets. Cell yields, RF and CE for all cell types were calculated. Allogeneic data were compared to autologous data. Lower T-cell subset concentrations and T-cell yields were seen in autologous PBSCs compared to allogeneic PBSCs; however, of all cell types, RF and CE were higher in autologous compared to allogeneic PBSCs. T-cell subsets — especially Tregs — are a substantial part of PBSCs transplantation, as recruitment — or at least relevant enrichment — occurs during apheresis. The authors conclude that in large volume apheresis, the CE efficiency of Tregs is comparable to that of CD34(+) cells, while RF are even higher. Such apheresis data could be helpful for the production of Tregs to treat GvHD or to be correlated with clinical findings about prevalence and severity of GvHD.

Economics and Outcome After HSCT: A Retrospective Cohort Study

Most hematopoietic stem cell transplants, or HSCT, are performed in more affluent countries, mainly due to the treatment expense. The impact of economic factors on patient outcome has not been well-defined. Gratwohl et al. retrospectively analyzed a defined cohort of 102,549 allogeniec or autologous HSCT patients. Subjects received transplants in one of 404 HSCT centers in 25 European countries between 1999 and 2006. Researchers examined associations between center-specific microeconomic or country-specific macroeconomic factors and outcome. Center patient-volume and center program duration were significantly and systematically associated with improved survival after allogeneic HSCT and autologous HSCT. The data, reported in “EBioMedicine,” indicate that country- and center-specific economic factors are associated with distinct, significant, systematic and clinically relevant effects on survival after HSCT. These factors affect center expertise in long-term disease and complication management. The authors recommend that micro- and macroeconomic factors be integrated into clinical transplant practice for both individual patients and for the cooperative working between regional transplant centers, to improve outcomes through better patient selection and increased expertise in disease- and complication management.

EVENTS, OPPORTUNITIES, RESOURCES

AABB CT Audioconferences and Webinars

Information on upcoming 2016 CT audioconference and webinar programs will soon be posted on the AABB website. A complete listing of past webinars available for purchase can be viewed on the CT Webinar page.

Volunteer Opportunities for Early Career Members

AABB has created junior member positions on association year 2015-16 board committees. Serving on a committee will allow early career professionals to gain experience, contribute to important deliverables, network and learn more about operations and policy development. Members who would like to apply, or who know of others who might be interested in this opportunity for the 2016-17 association year, are encouraged to visit the AABB Committee Volunteer Opportunities Web page.

AABB’s Newest Book Release is ...

Cellular Therapy: Principles, Methods, and Regulations, 2nd edition
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 book can be reserved in the AABB Marketplace.

The AABB HUB is a Resource Too!

AABB welcomes members to experience the best features and functionality of popular social media platforms by joining or initiating a conversation on AABB’s online collaborative space, the AABB HUB. The AABB HUB offers members access to discussion groups including “Cellular Therapies.” Recent CT postings include flow cytometric enumeration and specific gravity and reference ranges of cord blood. Subscribers can receive email alerts when new threads are posted−replying to posts directly from their email inboxes. The AABB HUB enables participants to ask and answer questions, share ideas and network with colleagues from across the United States and around the world. Start connecting with your cellular therapy peers today.

FOR MEMBERS ONLY

The Latest on CT Subsection Activities

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 discussing a myriad of CT topics. The materials or “projects” produced by the subsections can be found on the AABB Center for Cellular Therapies Web page. Below is an update on these activities with web links to some of the materials. Click on the links provided to learn more.

Asia Pacific Group, or APG: This subsection, which accommodates members in the Asia Pacific time zones, has been discussing a variety of topics related to cord blood. APG conducts teleconferences on the second Wednesday of each month at 0400 UTC (universal coordinated time). Interested individuals are encouraged to enroll in this growing group. Slides on the following topics presented this past quarter can be found on the APG subsection projects webpage: “The Importance of Flow Cytometry in Measuring Viability and How it Affects Thawed Cord Blood Products” by Vasiliki Kalodimou, PhD, the “Role of Private Cord Blood Banks in Regenerative Medicine” by Arvin Faundo, MD, PhD, and “Improving the Outcome of UCB Transplantation Through Ex Vivo Expansion or Graft Manipulation” by Mitchell Horwitz, MD.

Regulatory Affairs: This subsection welcomed new Leader Ljiljana Vasovic, MD, and Associate Leader, Olive Sturtevant, MHP MT(ASCP)SBB/SLS. Activities included discussions of FDA’s Public Hearing on Draft Guidances Relating to the Regulation of Human Cells, Tissues, or Cellular or Tissue-Based Products (HCT/P); Minimal Manipulation of HCT/P; HCT/P From Adipose Tissue; Homologous Use of HCT/P; and Same Surgical Procedure Exception, to name a few. This subsection is a must for those dealing with regulatory issues.

CT Management: This subsection welcomed new Leader, Suzanne Dworsky, MBA, MT(ASCP), and Associate Leader Brian Jones, SBB(ASCP). Discussions revolved around a variety of topics related to efficient management of the laboratory, including 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 including one on “Oral Stem cells and Their Potential Use for Regenerative Medicine,” presented by George T.J. Huang, DDS, MSD, DSc.

MSCs are important cells holding increasing therapeutic promise. To address the potential of MSCs and gauge member interest in learning more about them, Richard Schaefer, MD, provided a brief overview and suggestions for relevant educational activities. Schaefer will lead MSC-focused discussions quarterly during subsection meetings. Those interested in learning more or contributing their expertise are encouraged to join the subsection. For a preview of what you might expect please visit the Subsection Projects page.

CT Quality Operations: ‘Veteran’ subsection leader Kathy Fortune, BS, MT(ASCP), joined new subsection leaders Ed Brindle, MSc, MLT(CMLTO), and Deb Sesok-Pizzini, MD, MBA, as they launched their first teleconference following the 2015 AABB Annual Meeting. Lisa Phillips-Johnson, MT(ASCP)SBB, CQA, CQM/OE, RAC, delivered a presentation “Nonconforming Product Assessment.”

CT Product Collection and Clinical Practices: Subsection Leaders Tom Spitzer, MD, and Jay Raval, MD, welcomed new Associate Leader Joseph ‘Yossi’ Schwartz, MD, MPH. Among several new initiatives, the group discussed their ongoing survey, “Autologous Red Cell Collection and Transfusion Practices for Allogeneic Bone Marrow Donor Harvests,” which has been distributed to medical directors of marrow harvest programs at different institutions. The group also welcomed Marisa B. Marques, MD, ASFA immediate past president, who discussed the new “Qualification in Apheresis Exam ASFA/ASCP.”

CT Product Manufacturing and Testing: Subsection Leader Lizette Caballero, MLS(ASCP)CM, hosted several well-attended presentation-discussion sessions. One presentation was delivered by subsection Associate Leader Vasiliki Kalodimou, PhD on “Quality Controls in Flow Cytometry CD 34+ Enumeration Schemes.”

Cord Blood Subsection: This subsection, hosted by Leader Salem Akel, PhD, and Associate leader Gwen Epstein, BSC, RT, discussed upcoming project ideas including potential surveys and tips for improving laboratory processes. Recent discussions included issues surrounding retention of maternal samples and CB WBC count by hematology analyzers.

 

Editor: Christina Celluzzi
Contributor: Kathy Loper

AABB Center for Cellular Therapies
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