IN THE NEWS
AABB Thanks San Diego Blood Bank, ICBS Attendees; Encourages Celebration of World Cord Blood Day
AABB wishes to thank all registrants and exhibitors who attended the 15th International Cord Blood Symposium (ICBS) in San Diego for their participation in a variety of events that included educational lectures, workshops, lunch discussions and a hugely popular evening tour of the San Diego Blood Bank. Special appreciation goes to David Wellis, PhD, CEO, Robert J. Tressler, PhD, VP, Cell Biology & Therapy, and the staff of the San Diego Blood Bank for their warm welcome and generous donation of their time.
While at ICBS, AABB caught up with a few individuals and recorded “Conversations on Cord Blood” –discussions on the importance of cord blood, the future of the industry, engagement of the public and celebration of World Cord Blood Day, an initiative of Save the Cord Foundation. As part of the celebration, organizations, health care professionals and expectant parents are encouraged to participate in activities in the months leading up to the Nov. 15 celebration. The day will also be marked with a free, live virtual conference on the website. As an “inspiring partner” in World Cord Blood Day, AABB invites members to participate in the celebration by joining the discussion on social media and posting events at their cord blood banks. Members can follow AABB on Facebook, Instagram and Twitter to discuss cord blood, share highlights from ICBS and describe their role or interest in cord blood practice and education, not only on World Cord Blood Day, but every day. For more information on global events visit www.worldcordbloodday.org.
CTSCC Elections — Call for Candidates
AABB’s Cellular Therapies (CT) Section is now accepting applications from current members of any CT subsection who are interested in seeking election to the CT Section Coordinating Committee (CTSCC). The Committee works collaboratively to lead subsections, set priorities and support members of the CT Section. Committee members gain leadership experience, industry knowledge and networking opportunities. Members who wish to be included on the ballot may send an email to email@example.com by Aug. 31 stating their intention to run and subsection areas of interest. The email should include a photo and a 250-word summary describing the member’s relevant experience and potential contributions to the CT Section. AABB will acknowledge submissions via email and post information from qualified candidates online. The association will hold elections via electronic ballot and announce the results during the CT Section Business Meeting at the 2017 AABB Annual Meeting (Oct. 7-10) in San Diego.
AABB Meeting News Blog Allows Attendees to Add Their Voices
The AABB Annual Meeting Blog contains insider tips, tools, news and reviews about the 2017 AABB Annual Meeting, to be held Oct. 7-10 in San Diego. The blog is the best place to find information to get the most out of the meeting or to follow the meeting from home. AABB will regularly update the blog before and during the meeting. AABB will also publish blog posts from attendees who write about their favorite activities and sessions. Contact firstname.lastname@example.org for additional information on how to add your voice to the AABB Annual Meeting Blog.
Special Rate Offered to AABB Members For Scientific Symposium
In lieu of a workshop before the 2017 AABB Annual Meeting, the AABB Center for Cellular Therapies has partnered with the Alliance for Regenerative Medicine to present an opportunity to attend the 12th Annual Cell & Gene Meeting on the Mesa Scientific Symposium for the discounted rate of $95. Attendees provide their own transportation to and from the event. The symposium, which will be held at the Salk Institute for Biological Studies in La Jolla, Calif., draws leading scientists, clinicians, life science business executives, patient advocates and government officials to examine and discuss the imminent scientific and ethical challenges facing cell and gene therapy research today. Showcasing panel discussions that explore topics ranging from basic science to revolutionary techniques, the meeting will also feature two keynote presentations from world renowned researchers, in addition to extensive networking opportunities and a scientific poster session. The symposium program will be posted at a later time. Last year’s agenda is available on the meeting website for those interested in the format and past presentation topics. Registration information is available online. AABB members should select Industry & General Admission when registering for the Scientific Symposium and then enter the code "AABB" when prompted to receive the discounted rate. Individuals interested in submitting a poster may do so.
NBF Announces Availability of Funding For Scientific Research Projects
The National Blood Foundation, AABB’s philanthropic arm, announced the availability of funding in 2018 for scientific research projects related to transfusion medicine and cellular therapies. The NBF will award grants for investigator-initiated original research in all aspects of blood banking, transfusion medicine, cellular therapies and patient blood management. Grants will support one or two-year research projects, with a maximum award of $75,000. The NBF will not provide awards to increase the funding available for currently funded research projects. The grants are intended to provide seed funding that allows the principal investigator to enhance preliminary data, which can be used to apply for larger grants. Applicants must include an application fee of $160 in the the application, unless the primary investigator is an AABB individual member. To be eligible, an applicant must be a doctor (MD or PhD), medical technologist, or transfusion medicine or cellular therapies professional. All applicants will be considered regardless of age, race, gender, national origin or religion. Visit the NBF website for complete program guidelines, information about past award recipients and application instructions. The application deadline is Dec. 1, 2017.
AABB-ISCT Joint Working Group to Present Evaluation of Human Platelet Lysate at 2017 AABB Annual Meeting
Pooled human platelet lysate (pHPL) has been used by an increasing number of cell therapy manufacturers and scientists as a non-animal derived source of cell culture growth supplement. It can replace bovine serum while providing a rich source of multiple growth factors and cytokines. Identifying measures to evaluate pHPL products and compare the use of FBS will be important to enable its use in scaled-up cellular therapies. To coordinate worldwide efforts, AABB and ISCT collaborated to form a joint working group to identify and facilitate projects of common interest to both organizations and their significantly overlapping membership. The working group’s first project (offered in a webinar) addresses pHPL, and it plans to discuss standardizing pHPL production, evaluating pHPL products and interpreting the differences between pHPL and bovine serum, as well as highlights in the field, at an Oct. 8 annual meeting session.
8th Edition of Standards for Cellular Therapy Services Available
AABB’s Standards for Cellular Therapy Services, 8th Edition is available for purchase. Significant changes in the 8th edition include expanded proficiency testing requirements to facilitate compliance outside of the United States; a new requirement that all accredited laboratories implement ISBT 128 labeling by July 1, 2018; a separation of donor suitability and eligibility requirements into two distinct sections; and a new standard requiring facilities to have policies, processes and procedures for donor screening and testing that address all relevant emerging infectious diseases. The 8th edition of CT Standards can be purchased as an electronic version, the AABB Standards Portal; a printed version; or a bundle of the two. The portal also offers a complimentary two-week trial that gives new users full access to the Standards and allows them to experience all the features aimed at improving the user experience including the ability to personalize a user profile that is specific to the activities performed at a given facility, a robust search function and direct links to additional information, such as relevant guidance. An "On Demand” eCast about the changes to the 8th edition is also available.
REGULATORY AND GOVERNMENT UPDATE
FDA Posts Updated HCT/P Inspection Information
The FDA released inspection information for HCT/P establishments for FY 2012-2016. The number of inspections in 2012 was 592, 2013: 671, 2014: 652, 2015: 598 and 2016: 636. The average hours per each inspection were 39.4, 40.1, 35.4, 34.4 and 38.1 for each FY year, respectively.
RMAT— How Does it Work?
The new FDA Regenerative Medicine Advanced Therapy (RMAT)-designation expedites the approval process for regenerative medicines. Medicines must meet certain criteria to qualify for the RMAT designation: The drug must be “a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations.” In addition, the drug must be “intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition.” Finally, there must be preliminary clinical evidence that “indicates that the drug has the potential to address unmet medical needs for such disease or condition.” The Office of Tissues and Advanced Therapies (OTAT) reviews RMAT request submissions and is required to notify the sponsor if they have received the RMAT designation within sixty days. RMAT requests are submitted with the IND, or as an amendment to an existing IND.
FDA Assay Predicts Ability of Mesenchymal Stromal Cells to Suppress Immune System Activity
Scientists at the U.S. Food and Drug Administration developed an assay that can predict the ability of human mesenchymal stromal cells (hMSCs) to suppress immune system activity. The study represents an innovative use of morphological features of cells that enables researchers to rapidly predict future cell behavior. hMSCs are cells derived from multiple tissue sources (such as bone marrow, fat, dental pulp and umbilical cord) that have the unique ability to generate cells associated with bone, cartilage and adipose tissue. There is also great interest in the cells’ ability to suppress unwanted immune system activation. The FDA finding is important because the ability of hMSCs to suppress immune system responses makes them potentially effective treatments for inflammatory diseases caused by undesirable immune system activities. Several of the FDA researchers, Matthew W. Klinker, Steven R. Bauer and Ross A. Marklein, presented their findings — published in PNAS — for AABB members of the Cellular Therapy Regulatory Affairs Subsection.
The Implications of CRISPR
Geneticist Jennifer Doudna, co-inventor of the groundbreaking gene-editing technology called CRISPR-Cas9, describes (in a past TED talk) this tool, which allows scientists to make precise edits to DNA strands. This technology could lead to treatments for genetic diseases but could also potentially be used to create so-called “designer babies.” Doudna reviews how CRISPR-Cas9 works — and asks the scientific community to pause and discuss its implications.
Stem Cell Trial Data Go Mostly Unpublished
Fewer than half of completed stem cell studies in humans are published in peer-reviewed journals, according to an analysis of regenerative medicine trials appearing in Stem Cell Reports. There is a gap between studies that have taken place and publication of the data. Fung et al. assessed publication rates for clinical trial results of innovative cell-based interventions. Assessment criteria included (1) characteristics and time to publication of completed trials; (2) quality of reported trials; and (3) results of published trials. The authors identified and analyzed publications from 1,052 novel stem cell clinical trials. They found that the quality of the reporting varied: 179 (45.4%) of 393 completed trials had published results; 48 trials were registered by known stem cell tourism clinics, none of which reported results. Completed non-industry-sponsored trials initially published more rapidly, but differences with industry-sponsored trials decreased over time. Most publications reported safety, and 67.3% (mainly early-stage trials) reported positive outcomes. A higher proportion of industry trials reported positive efficacy. Heightened patient expectations for stem cell therapies give rise to ethical obligations for the transparent conduct of clinical trials. Guidance documents do not establish legally enforceable responsibilities. Fung et al. suggest that researchers are more likely to comply with requirements when journals require studies to be registered than just reporting results in clinical trial registries. Clinical trial registries, therefore, need appropriate support for their efforts to monitor the status of ongoing clinical trials and the reporting of results to inform patients and decision makers of the development status of novel fields of biomedicine. Reporting guidelines need to be developed that are specific to early-phase clinical trials. Related issues — recently cited in the news — include the potential that many unapproved cellular therapy studies are being marketed to consumers by listing them on ClinicalTrials.gov.
Researchers Create Inner Ear Tissue Using iPSCs
Researchers have figured out how to make many different human cell types using induced pluripotent stem cells (iPSCs), which are cells taken from the body and re-programmed into a stem cell-like state. Recently, researchers created inner ear tissue using iPSCs, a process which potentially could be used to gain new insights into the causes of hearing loss and balance disorders. Koehler et al grew iPSCs into three dimensional balls of cells, identifying proteins that stimulated the iPSCs to transform into inner ear tissue. Realizing that timing was key, they determined when to apply the protein signal. Applying the signals at the wrong time could potentially generate another organoid, such as a brain instead of an ear. These iPSC-derived inner ear organoids may be a key step toward the goal of repairing hearing loss.
Becoming Better Listeners
In our increasingly noisy world, says sound expert Julian Treasure, "We are losing our listening." In this short, fascinating talk, Treasure shares five ways to re-tune our ears for conscious listening — to other people and the world around us.
The Transcendent Power of Music
You would not expect to read about music in a newsletter about cellular therapies, but this sound may raise a few questions about the power of sound on our cells. Sam Maher, a West Australian instrumentalist and drummer, is known for his unique playing style on the handpan — a rare, transcendental percussion instrument invented in Switzerland in 2000. This instrument has been found to have an unusual effect on humans from all over the world.
EVENTS, OPPORTUNITIES, RESOURCES
AABB 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 include:
09/21 Zika Virus: Donor Screening and Handling CT Products in the Face of Emerging Viruses (#160)
11/16 Facilities for Cord Blood Processing: Gowning-Cleaning Procedures (#172)
Cellular Therapies Certificate Program
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 cellular therapies. The program features 12 narrated modules covering scientific, operational and regulatory topics, which could fit into a training program. 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 37 continuing education credits (CMEs).
Easy Access to Resources for AABB Members
The AABB Member Resources page provides access to relevant news sources, educational programs, advocacy updates, networking and volunteering opportunities, and more. Visit the page to discover new ways to build a network of colleagues, investigate opportunities for professional development and keep up with the latest advances in transfusion medicine, cellular therapies and patient blood management.
FOR MEMBERS ONLY
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. Palanisamy Murugan presented “Establishment of MSC Master Cell Bank from Umbilical Cord Tissue.”
Regulatory Affairs: This subsection, led by Ljiljana Vasovic, MD, and Olive Sturtevant, MHP, MT(ASCP)SBB/SLS, welcomed Matthew Klinker, PhD, Steven Bauer, PhD, and Ross Marklein, PhD, of the FDA, who presented their published work “Morphological features of IFN-γ-stimulated mesenchymal stromal cells predict overall immunosuppressive capacity.”
CT Management: This group, led by subsection leaders Suzanne Dworsky, MBA, MT(ASCP), and Brian Jones, SBB(ASCP), discussed the components of good laboratory management.
Novel Therapies and CT Product Development: This subsection, led by Magali Fontaine, MD, PhD, and Richard Schaefer, MD, discussed the results of a BEST collaborative survey. The survey aimed to identify the essentials of manufacturing practices to be recommended for specific cellular therapy products at different stages of manufacturing (i.e., collection, ex vivo manipulation or expansion, distribution or preparation for infusion). The survey will direct the surveyors to the specific type of products they are producing in their laboratory and give them an opportunity to collaborate with other labs on the validation of their manufacturing processes.
CT Quality Operations: Ed Brindle, MSc, MLT(CMLTO), and Deb Sesok-Pizzini, MD, MBA, hosted talks on a variety of topics, including benchmarking, non-conformances and highlights from recent professional meetings.
CT Product Collection and Clinical Practices: Subsection leaders Tom Spitzer, MD, Jay Raval, MD, and Joseph ‘Yossi’ Schwartz, MD, MPH, presented journal club and discussed the Letter to the Editor, “Transfusion practices for bone marrow harvests: a survey analysis from the AABB Bone Marrow Quality Improvement Initiative Working Group,” which was produced by several subsection members and representatives from a variety of other organizations. The letter appeared recently in Bone Marrow Transplantation (www.nature.com/bmt).
CT Product Manufacturing and Testing: Ronit Slotky, PhD, MSc, and Yen-Michael S. Hsu, MD, PhD, hosted speakers on several topics. Lawrence S. Lamb, MN, PhD, presented “Choosing and Monitoring the Right Manufacturing Environment for Your Cell Product.”
Cord Blood Subsection: Subsection leaders Salem Akel, PhD, and Gwen Epstein, BSC, RT, welcomed Indresh Kaur, PhD, who presented her article, “Comparison of two methodologies for the enrichment of mononuclear cells from thawed cord blood products: The automated Sepax system versus the manual Ficoll method.” Members of the subsection who attended ICBS in San Diego had the opportunity to meet face-to-face to share insights.
Spanish Language Subsection (SLS): Spanish-speaking members are encouraged to join the SLS. Celina Montemayor, MD, PhD, and Angel Guerra, MD, moderate this subsection, which is conducted entirely in Spanish. The SLS projects page showcases content that has been presented to the subsection.