AABB CellSource - October 2014

 
AN UPDATE ON CELL THERAPY NEWS FROM AABB
October 2014

IN THE NEWS

Seats Are Still Available for the CAR T Cell Therapies Workshop

It is not too late to register for the AABB and the University of Pennsylvania workshop, “CAR T Cell Adoptive Therapies: Product Engineering to Clinical Outcomes,” to be held on Oct. 24 at the Pennsylvania Convention Center, prior to the start of the 2014 AABB Annual Meeting. This all-day event will cover an introduction to T cell receptors, CAR T cell applications, CAR T cell manufacture for clinical use, CAR T technologies, needs of future therapeutic modalities and regulatory hurdles. Registration discounts apply to those attending the AABB Annual Meeting.

Presenters Sought for Cellular Therapy RAP Session

The chairs of the Cellular Therapies Research and Progress, or CT RAP, session are seeking presenters for the program, to be held at 6 pm on Sunday, Oct. 26 during the AABB Annual Meeting. Speakers at the session, “Stem/Progenitor Cell Expansion and Engraftment,” will address the challenge of obtaining sufficient engraftment of umbilical cord blood transplants. The interactive session will highlight new developments in the field and provide a shorter and less formal forum — as compared to regular educational sessions — for attendees to discuss their research. Interested individuals are asked to send an email message with their contact information.

AABB Annual Meeting Essentials for CT Attendees

'Ask an Assessor' at the AABB Center for Cellular Therapies Pavilion

Visitors to the Cellular Therapies Pavilion located in AABB Central at annual meeting will have an opportunity to schedule a discussion with an assessor to answer questions about CT Accreditation. “Office hours” will be posted on-site. Welcoming AABB staff will be available to provide information on enrollment in a CT subsection and the latest CT educational offerings including podcasts, webinars and audioconferences.

Tools Available to Map Out Annual Meeting Activities

There are a variety of resources for attendees to plan their CT activities at annual meeting. Hardcopies of the CT schedule at a glance will be placed at different locations around the meeting venue. A bulletin board displaying meeting information will be located on site. For those who prefer electronic delivery, the AABB Annual Meeting app for smartphone and tablet users is available for download. The app allows users to create an itinerary and stay connected with information and updates before, during and after the meeting. A multi-sync feature allows users to sync data, including “My Schedule” and “Exhibitor” bookmarks, between multiple devices using the apps.

CT Networking Events Will Keep Attendees in the Loop

Attendees can enrich their meeting experience with a variety of networking events including a Cellular Therapy Networking Reception and a SoCell Network roundtable session to discuss a myriad of CT topics. Those attending luncheons also will have opportunities to network — the CT Section Business Meeting and Luncheon for CT Section members to learn about CT Section activities and engage their colleagues, the Sizzling Topics Luncheon in the style of ‘point-counterpoint’ for attendees to debate ‘hot’ topics in a casual setting and the Abstract Poster Luncheon where attendees can browse the scientific presentations and hold discussion with the authors.

New Member/Attendee Breakfast

For first time meeting attendees and new AABB members, there will be an opportunity to network and get oriented to AABB and annual meeting activities during breakfast. Learn more about AABB CT membership ─what it can offer members and what members can provide to others.

REGULATORY AND GOVERNMENT UPDATE

AABB Posts New International Regulations Summaries and CT Quick Reference Tool

Summaries describing cellular therapy-related regulations in China, Malaysia, Hong Kong, and Colombia, have been added to the AABB International Competent Authorities Web page. These summaries also include links to applicable guidelines, resources and regulations.

Need to know the difference between when you should report an adverse event under an IND versus if a product is regulated solely as an HCT/P? AABB has developed a comparative adverse event reference sheet to assist members in understanding the different criteria and reporting requirements for adverse events and reactions for various product types. This tool provides members with valuable and easy-to-read information on the various adverse event reporting categories for cellular therapy products depending on whether they are subject to IND, BLA, and HCT/P requirements.

Cellular, Tissue and Gene Therapies Advisory Committee Meeting Scheduled in November

The Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee, or CTGTAC, will convene on Nov. 6 at the FDA’s White Oak Conference Center in Silver Spring, Md. At this meeting, the committee will discuss the draft guidance, “Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products,” and the “Dear Gene Therapy IND or Master File Sponsor Letter.” Meeting information is posted on the CTGTAC website and will be updated with additional agendas and meeting materials as they become available.

RESEARCH FOCUS

Chimeric Antigen Receptor Therapy For Cancer

Basic, preclinical and clinical advances spanning cellular immunology, synthetic biology and cell-processing technologies have paved the way for clinical applications of chimeric antigen receptor, or CAR-based therapies. This form of targeted immunotherapy merges the targeting specificity of monoclonal antibodies with the potent cytotoxicity and long-term persistence provided by cytotoxic T cells. Clinical trials have shown significant antitumor activity in neuroblastoma, acute lymphocytic leukemia and B cell lymphoma. Other trials targeting a variety of adult and pediatric malignancies are progressing. Identifying optimal tumor targets and manipulating cell- and host-associated factors to support expansion and persistence of the genetically engineered cells in vivo is the focus of ongoing work. This potential to target essentially any tumor-associated cell-surface antigen for which a monoclonal antibody can be made opens up a new arena for targeted cancer therapy. Several of the author researchers of a review on CAR therapy for cancer by D.M. Barrett et al. published in the journal “Annual Review of Medicine,” and other professionals in the field are scheduled to present at the CAR T cell workshop prior to the start of the AABB Annual Meeting in Philadelphia. Carl June, one of the leaders in this research, is the recipient of the 2014 AABB Karl Landsteiner Memorial Award and Lectureship. Dr. June will present “Engineered T Cells: A New Form of Transfusion Medicine” at the AABB Annual Meeting on Tuesday, Oct. 28.

Cell-based Therapies for Cardiac Disease: a Cellular Therapist’s Perspective

Stem cell-based therapies have the potential to fundamentally transform the treatment of ischemic cardiac injury and heart failure. Recent therapeutic approaches involve bone marrow-derived mononuclear cells and their subsets such as mesenchymal stem/stromal cells, or MSCs, endothelial progenitor cells, adipose tissue-derived MSCs, cardiac tissue-derived stem cells and cell combinations. In the journal “Transfusion,” Young and Schaefer review the clinical trial results to date — discussing challenges and operational issues in cell processing for cardiac applications.

Bacterial Diversity and Fewer Deaths in HSC Recipients

Graft versus host disease, or GVHD, remains the major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplant, or allo-HSCT, and limits its use as a curative therapy for malignant and non-malignant hematologic disease. The gastrointestinal, or GI, tract is affected in nearly all cases of GVHD. Research conducted by Y. Taur et al., published in the journal “Blood,” demonstrated that the diversity of the intestinal microbiota at engraftment is an independent predictor of mortality in allo-HSCT recipients. These findings indicate that the intestinal microbiota may be an important factor in the success or failure of allo-HSCT. Their work suggests that maintenance or restoration of intestinal bacterial homeostasis may prevent or control GI GVHD.

Human Stem Cell Clock Reset to Zero

Scientists have found a way to reset human pluripotent stem cells to a pristine state — a developmental state equivalent to cells found in an embryo before it implants in the uterus. Though previously achieved in mice and rats, scientists had to use a different approach in humans to induce short-term expression of two different genes, NANOG and KLF2, which is sufficient to cause the network of genes that controls the cell to ‘reboot’ and induce the native pluripotent state. Y. Takashima et al. reported these findings in the journal “Cell.”

Unsuspected Aspect of Immune Regulation Revealed: the Role of 'B Cells'

Until now, the immune cells known as 'B cells' have been thought to specialize only in the production of antibodies. A discovery by immunologists shows they also have a role to play in regulating another important aspect of the immune system — the development of regulatory T cells, or Tregs. This finding may benefit research into autoimmunity and transplantation. B cells inhabit the normal human thymus, suggesting a role in T cell selection. In their study, S.N. Waters et al. report in “The Journal of Immunology” that B cells can modulate thymic production of CD4+ Foxp3+ T cells — Tregs — and present a mechanism whereby intrathymic B cells, through the provision of cognate help, contribute to the shaping of the Treg repertoire.

EVENTS, OPPORTUNITIES, RESOURCES

CT AABB Audioconferences and Webinars Still to Premier in 2014

One ‘live’ CT audioconference program:

11/19 Top 10 Cellular Therapy Non-Conformances (#144830)

and several webinars in the AABB Center for Cellular Therapies webinar series in which experts share their knowledge and experience, answer questions from participants and provide helpful references remain in 2014. Economically priced for individuals, the hour-long, interactive webinars consist of 45-50 minutes of presentation followed by a 10-15 minute period for questions and answers. CME credits are available for the live sessions. Upcoming webinars include:

11/18 Efficient Generation of Cytotoxic T cells (#1422)Formerly scheduled for October 2
11/20 Key Elements of Cord Blood Processing (#1423)
12/16 Computer, Software and Spreadsheet Validation Cellular Therapy Lab (#1424)

For individuals who wish to see any of the past programs, a complete listing of the 2014 educational events is available for purchase by visiting the website.

Call for 2015 Annual Meeting Education Program Proposals

It is never too early to plan educational sessions for the coming year. The AABB Annual Meeting Education Program Unit is soliciting proposals for educational programs to be presented at the 2015 AABB Annual Meeting to be held in Anaheim, Calif. Consideration will be given to proposals for both 90-minute and three-hour programs. The deadline for submissions is Nov. 14.

FOR MEMBERS ONLY

Spotlight: CT Subsection Updates

Members of the AABB Center for Cellular Therapies subsections meet regularly via teleconferences and participate in an assortment of interactive activities ─ including development of tools and reference materials. The materials or “projects” produced by the subsections are located on the AABB Center for Cellular Therapies Web page. The following is a list of recent subsection activities.

Asia Pacific Group, or APG: This subsection, set up to specifically accommodate members in the Asia Pacific time zones, has been expanding its discussions to bring awareness to a variety of topics including unique challenges to the region. APG meets via telephone on the second Wednesday of each month at 0400 UTC (universal coordinated time). Interested individuals are encouraged to enroll. Salem Akel, PhD, (SSM Cardinal Glennon Children’s Medical Center) recently gave a presentation on “Current Thawing and Infusion Practice of Cryopreserved Cord Blood: the Impact on Graft Quality, Recipient Safety and Transplantation Outcomes” for the APG and Regulatory Affairs subsections.

Quality Operations Subsection: This subsection held several presentations this quarter including “Lessons Learned from FDA: BLA Experience” by Kathy Fortune, MT(ASCP) (SSM Health Care), and “Apheresis Equipment: Spectra Optia® vs. Fenwal Amicus®: Comparison Study” by Lizette Caballero, MLS(ASCP)CM (University of California San Francisco, UCSF).

CT Product Collection and Clinical Practices: This subsection enjoyed a presentation on “CIBMTR and Cord Blood Data” by Jennie Bloomquist, MS, and Mary Eapen, MD (CIBMTR).

Product Manufacturing and Testing, or PMAT: Presentations were given by subsection leader David Stroncek, MD (National Institutes of Health), on “Elutriation of Peripheral Blood Mononuclear Cells” and by Irwin Cabana (ICCBBA) “ISBT 128: From the Big Picture to Practical Issues.”

Cord Blood Subsection: Del Steckler, RN, BSN, MMA (NMDP) discussed “Strategies for Promoting Public Awareness of Cord Blood Donation.”

CT Management: This subsection created a survey to gauge practices for day-to day operational management of cellular processing laboratories in the United States. Results of the survey should provide a better understanding of where participants fall along the spectrum of practice compared to their peer group based on the number of cellular therapy products manufactured. Such information may be useful in justifying approvals for additional personnel. Click here to participate in the survey.

Regulatory Affairs Subsection: This subsection hosted several presentations this quarter: Lizette Caballero, MLS(ASCP)CM (UCSF), discussed “Materials Management,” and Fran Rabe, MS,CQM(ASQ) (University of Minnesota), provided a perspective on interpretation and compliance with “AABB Standard 5.4.6: Critical Materials,” of the AABB Standards for Cellular Therapy Services, 6th Edition.

Novel Therapies and CT Product Development: This subsection held a series of journal article discussions on topics including mesenchymal stem cells. Atta Behfar, MD, PhD (Mayo Clinic), presented research on cardiopoietic MSC-based therapies.

The subsections will be meeting face to face during the AABB Annual Meeting. Meeting times and locations can be found in the “CT Schedule at a Glance.”

 

Editor: Christina Celluzzi;
Contributors: Rafael Cassata, Naynesh Kamani, Kathy Loper, and Katherine Bricceno

AABB Center for Cellular Therapies
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