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AABB CellSource - April 2017

 
AN UPDATE ON CELL THERAPY NEWS FROM AABB
April 2017

IN THE NEWS

Deadline to Submit Abstracts for AABB Annual Meeting Approaches

The AABB Selection of Abstracts Committee will accept abstract submissions through April 27 for the 2017 AABB Annual Meeting — to be held Oct. 7-10 in San Diego. Submissions from both members and nonmembers are welcome. Abstract categories include scientific and administrative categories in cellular therapies, transfusion medicine and patient blood management. Accepted abstracts will be published in a special supplement of the September issue of “Transfusion.” AABB will present 10 Outstanding Abstract Awards for Trainees based on abstract quality and scientific merit. Recipients, who will receive a $500 award, will be recognized for their achievement at the meeting.

Save The Date! Scientific Symposium Offered Prior to AABB Annual Meeting

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.

Cellular Therapies Certificate Program Opens for Enrollment

The AABB Cellular Therapies Certificate Program, produced in partnership with the George Washington University, is now open for enrollment. The self-paced, online program is 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 study and successfully work through the modules, complete each assessment with a score of 80 percent or higher and fill out a program evaluation will receive a certificate of completion. The program also provides 37 continuing education credits (CMEs).

Registered Attendees of 15th International Cord Blood Symposium Offered Tour of Blood Bank

The 15th International Cord Blood Symposium (ICBS), to be held June 8-10 in San Diego, brings together world-renowned experts in umbilical cord blood and tissue research and regenerative medicine. In addition to the scientific program, attendees can register for a private tour of the San Diego Blood Bank on June 9 from 5:30-7:30 p.m. The tour includes a welcome by blood bank CEO, David Wellis, PhD, a presentation on the blood bank “Then and Now” by Rob Tressler, PhD, VP of Laboratories, a look at the donor center, labs and hospital services, and a discussion on the bank’s role in blood banking, stem cell research, cord blood activities and more. The evening event, complete with light snack, is made possible by support from BioSafe. Transportation will be provided. Registration is complimentary but space is limited.

AABB Invites Readers to Join in First World Cord Blood Day

Expectant parents and health professionals across the globe are invited to participate in the inaugural World Cord Blood Day, to be held on Nov. 15. “We are excited about this opportunity to expand cord blood education worldwide while providing a platform for discussion focused exclusively on this valuable medical resource,” said Charis Ober, executive director of Save the Cord Foundation. Organized by Save the Cord Foundation, World Cord Blood Day is sponsored by Quick Specialized Healthcare Logistics. Inspiring Partners for the event include AABB, the Cord Blood Association, Be the Match (NMDP), World Marrow Donor Association (WMDA – Netcord) and FACT. As Inspiring Partners, these organizations will provide industry-specific and medical expertise. AABB will develop activities that highlight cord blood in connection with its social media.

World Cord Blood Day will also host a free, open virtual conference on November 15. The program will include introductory presentations appropriate for the public, as well as academic lectures designed for health professionals and led by renowned researchers and transplant doctors. In parallel, organizers will build cord blood awareness and encourage participation in the day’s events via social media. Register now to see how your cord blood bank can participate in this complimentary event at www.WorldCordBloodDay.org.

SLS Celebrates Six Months - La Subsección en Español del Centro de Terapias Celulares Celebra 6 Meses en el Mes de Abril

La Subsección en Español celebra sus 6 meses en este mes de Abril con una interesante conferencia acerca de proyectos de vigilancia en terapia celular a nivel mundial. La Subsección en Español ofrece a sus miembros la oportunidad de desarrollar una red de contactos con otros profesionales hispanos en el área de terapia celular. Al igual que las otras subsecciones del Centro de Terapias Celulares, el grupo se reúne mensualmente a través del internet para conectarse en conferencia telefónica y discutir temas técnicos y publicaciones relevantes, con el objetivo de desarrollar los recursos para los programas de terapia celular en las regiones de habla hispana. Las presentaciones de meses anteriores, que tratan acerca de bancos de cordón, células madre del tejido adiposo, el Estándar ISBT 128, procesamiento de tejido de cordón, y el proceso de traducir nuevas terapias celulares del laboratorio de investigación a la clínica, se pueden revisar aquí.

AABB Announces Updated Vision, Mission and Core Values

AABB announced that the association has updated Vision, Mission and Core Values statements. Members of the AABB Board of Directors believe the updated statements will position the association positively as it moves forward and serve as important guiding principles for the future.

REGULATORY AND GOVERNMENT UPDATE

AABB Posts its 2017 Advocacy Agenda

Every year, the AABB Board of Directors dedicates time to strategizing on the association’s advocacy priorities for the coming year. In 2017, AABB is dedicating its advocacy agenda to promoting policies that support two broad objectives: 1) ensuring that patients and health care providers have adequate, appropriate access to safe transfusion medicine and cellular therapies, and 2) promoting patient and donor care and safety in transfusion medicine and cellular therapies. AABB remains dedicated to its vision of making transfusion medicine and cellular therapies safe, available and effective worldwide. The 2017 advocacy agenda reiterates AABB’s commitment to this guiding principle.

Regenerative Medicine Advanced Therapy Program Opens; Designation May Speed Up Approval for Certain HCT/Ps

Center for Biologics Evaluation and Research (CBER) Director Peter Marks, MD, PhD, announced that the agency has begun accepting requests for the new Regenerative Medicine Advanced Therapy (RMAT) Designation. The RMAT designation expedites the approval process for regenerative medicines — cell therapies, therapeutic tissue engineering products, human cell and tissue products and certain combination products — meant to treat serious or life-threatening diseases or conditions. There must be at least preliminary clinical evidence that the product has the potential to address unmet medical needs for the disease or condition it is meant to treat. The sponsors of RMAT-designated products are eligible for increased and earlier FDA access and may receive priority review and accelerated approval, as well. Sponsors can apply for the new RMAT designation when they submit an investigational new drug (IND) application or amend an existing IND. CBER will respond to RMAT applications within 60 days of receipt. Additional information is available in FDA’s guidance, Expedited Programs for Serious Conditions – Drugs and Biologics.

CDC Releases New Information on Zika Risk to Blood and HCT/Ps in Florida

The Centers for Disease Control and Prevention identified a potential increased risk to blood and tissue safety from Zika virus (ZIKV), affecting donors in Florida's Miami-Dade, Broward and Palm Beach counties from June 15, 2016, to the present. In response, the Food and Drug Administration released Important Information for Blood Establishments and for HCT/P Establishments. FDA posted the notice to advise blood collectors of this new information. The agency is not suggesting "lookback" or recall activities for affected blood products. The new information will permit individual centers to determine if additional measures are necessary to address the possibility of increased risk from blood components collected in these three counties before blood donor screening tests for ZIKV were in place.

CDC also notes ZIKV's prolonged persistence in certain HCT/Ps, particularly semen. In addition, the tissues affected may include hematopoietic progenitor and stem cells from peripheral or cord blood, and reproductive and gestational tissues. CDC suggests that health care providers also consider the status of potential HCT/P recipients who may be at increased risk, such as immunocompromised individuals and women of child-bearing age — especially those seeking assisted reproductive technology treatments. CDC notes that the risk is small and does not require formal regulatory action from FDA. Additional information, is available in the FDA's guidance, Donor Screening Recommendations to Reduce the Risk of Transmission of Zika Virus by Human Cells, Tissues, and Cellular and Tissue-Based Products.

National Academy of Sciences Releases Report on Preparing for Future Biotechnology Products

On March 9, the National Academy of Sciences (NAS) released a report sponsored by FDA, EPA and the Department of Agriculture entitled, “Preparing for Future Products of Biotechnology.” Between 1973 and 2016 — according to the NAS website — the ways to manipulate DNA to endow an organism with new characteristics (that is, biotechnology) have advanced, enabling the development of new products. What will the likely future products of biotechnology be over the next 5-10 years? What scientific capabilities, tools, and/or expertise may be needed by regulatory agencies to ensure efficient and sound evaluations of the likely future products of biotechnology? “Preparing for Future Products of Biotechnology” analyzes the future landscape of biotechnology products and seeks to inform forthcoming policy making. This report identifies possible new risks, frameworks for risk assessment and areas in which the safety or risks of new biotechnology products are well understood.

Summary of October FDA Cell Therapy Liaison Meeting Available

AABB posted a summary of the Food and Drug Administration Cellular Therapy Liaison meeting, which took place at the association’s headquarters in Bethesda, Md. in October. The meeting included six sessions on issues relevant to cellular and gene therapy products. Presentations addressed the need for definitions of critical product and quality attributes — to enable comparability during development; introduced relevant FDA documentary standards and reference materials; highlighted the need for reference standards; and included discussions on transferring the manufacture of a product to an international location. Also covered were communicable diseases relevant to HCT/Ps and labeling of products intended for further manufacture with unique product and patient identifiers.

Transcripts Posted from the CBER Public Workshop: Identification and Characterization of Infectious Disease Risks of HCT/Ps

The Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) held a two-day public workshop, “Identification and Characterization of Infectious Disease Risks of HCT/Ps,” on Feb 8-9 to promote discussion on methods to identify and characterize infectious disease risks associated with human cells, tissues and cellular and tissue-based products (HCT/Ps). Transcripts of the Feb 8 and Feb 9 sessions have been posted on the FDA website.

RESEARCH FOCUS

Therapeutic Mesenchymal Stromal Cell-Derived Extracellular Vesicles Produced Using Platelet Lysate

Extracellular vesicles (EVs) which are released by mesenchymal stromal cells (MSCs) may contribute to biological processes such as tissue regeneration, immunomodulation and neuroprotection. To evaluate their therapeutic potential and future applications, researchers must first characterize EV content and production under defined medium conditions, devoid of xenogeneic substances and serum-derived vesicles. Addressing the apparent need for such a growth medium, Pachler et al have developed a medium formulation based on pooled human platelet lysate (pHPL). The medium is free from animal- derived xenogeneic additives and depleted of EVs. They found that 10 percent pHPL-based EV-depleted medium is appropriate for purifying exclusively human MSC-derived EVs. Researchers using this Good Manufacturing Practice-grade protocol can characterize and establish protein and RNA profiles of MSC-derived EVs, enabling them to identify active components in EVs for future clinical applications.

Stem Cells Replace Animals in Botox Research

Clostridium botulinum neurotoxins (BoNTs) are naturally occurring protein toxins that cause the severe and potentially life-threatening disease botulism. They are being utilized as cosmetics and as unique bio-pharmaceutical treatments for an increasing number of neurological and non-neurological disorders. Currently, the potency of biologically active BoNT for therapeutic use is primarily monitored by the murine LD50-assay. However, the assay is an ethically disputable test causing suffering and death of a considerable number of mice. An alternative to the widely used in vivo mouse bioassay, an in vitro BoNT detection assay was developed by Jenkinson et al using mouse embryonic stem cell-derived neurons (mESNs) cultured on multi-electrode arrays (MEAs). The mESNs form a neuronal network in culture showing spontaneous bursting activity based on functional synapses and express the necessary target proteins for BoNTs. Treating cultures with BoNT serotype A followed by incubating them with BoNT/A or Botox® led to a significant reduction of both spontaneous network bursts and average spike rate. These findings suggest that mESNs cultured on MEAs pose a novel, biologically relevant model that could be used to detect and quantify functional BoNT effects. This type of assay may accelerate BoNT research while decreasing animal use.

Toy Inspires Hand-Powered, Ultralow-Cost Paper Centrifuge

In a global-health context, commercial centrifuges are expensive, bulky and electricity-powered, thereby constituting a critical bottleneck in the development of decentralized, battery-free point-of-care diagnostic devices. Inspired by a toy, Stanford bioengineers have developed an inexpensive, human-powered blood centrifuge that enables precise diagnosis and treatment of diseases in areas of the world where it is difficult to operate standard equipment. This video shows the ultralow-cost (<20 cents), lightweight (2 g), field-portable centrifuge. The researchers designated the device a ‘paperfuge’ based on its inspiration: whirligig (or buzzer) toys. The paperfuge has thus far achieved speeds of 125,000 rpm (30,000 g) using only human power.

How to Get Better at the Things You Care About

In a recent TED talk, learning expert Eduardo Briceño explained why mistakes are welcome for learning. “Are you working hard but not improving? Well you are not alone.” Briceño reveals a simple way to think about getting better at all the things one does, whether it is work, parenting or creative hobbies. He differentiates between performance and improvement, and shares some useful techniques to help one keep learning and experiencing one’s progress.

EVENTS, OPPORTUNITIES, RESOURCES

AABB eCasts

AABB offers educational programs as eCasts, which are live online interactive programs that cover topics in transfusion medicine, cellular therapies and patient blood management. eCasts run for 90 minutes and include topics for learners of all experience levels. Registration is available for individuals and groups, and institutions can host eCasts for employees at one or more sites. Those who participate in group eCasts are eligible to earn continuing education credit. Those who are unable to attend a live eCast can purchase an On-Demand recording of the eCast session. Upcoming CT eCasts include:

05/11 Microbiology Lab in Cell Therapy Facility: Operational Aspects and Merits (#134)
06/08 Donor Safety and Selection of Donors (#140)
09/21 Zika Virus: Donor Screening and Handling CT Products in the Face of Emerging Viruses (#160)

PEP Offers Mentoring Programs for Experienced and Early Career Professionals

AABB’s Professional Engagement Program (PEP) offers experienced professionals the opportunity to make a difference to an early career professional through its mentoring program. This informal six-month program matches experienced mentors with mentees with less than five years of experience in the fields of transfusion medicine, patient blood management and cellular therapies. Participants expand their professional network, exchange information and ideas, and build lasting friendships. Members interested in participating in the program may apply by completing the application. For more information, visit the PEP website.

A Portal to the Cell

The new Allen Cell Explorer is a data portal that lets users explore publicly available data, tools and models of the Allen Institute for Cell Science. The portal provides an unprecedented view into the organizational diversity of human stem cells by combining large-scale 3D imaging data, predictive models, observations of cells, detailed methods, and cell lines available for use in labs around the world. Watch the announcement video.

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. David Stroncek, MD, presented two talks: “Translating Cell and Gene Therapies from the Research Laboratory to the Clinic” and "CAR T Manufacturing Challenges.” Vishnu Balaji, MD, presented “Stem Cells in Cancer Therapy.”

Regulatory Affairs: This subsection led by leaders Ljiljana Vasovic, MD, and Olive Sturtevant, MHP, MT(ASCP)SBB/SLS, discussed a variety of regulatory topics including the “FDA Guidance Agenda: Guidance Documents CBER is Planning to Publish During Calendar Year 2017,” x-ray scanners and cellular therapy products led by Lori Hanley, CMQ/OE, CQA(ASQ), MT(ASCP, National Marrow Donor Program/Be The Match, and Highlights of the Feb 8 FDA meeting “Identification and Characterization of the Infectious Disease Risks of Human Cells, Tissues, and Cellular and Tissue-based Products,” presented by Kerri Wachter, science writer, AABB.

CT Management: This group, led by subsection leaders Suzanne Dworsky, MBA, MT(ASCP), and Brian Jones, SBB(ASCP), discussed topics relevant to laboratory management. Dworsky presented “Participants’ Service Level Agreements” with template, as well as led a detailed discourse on capital equipment justifications and acquisition and laboratory organizational charts – important to running a ‘smooth’ CT facility.

Novel Therapies and CT Product Development: This subsection, led by Magali Fontaine, MD, PhD, and Richard Schaefer, MD, discussed human platelet lysate for manufacture of CT products, mesenchymal stromal cell–secreted chemerin, a BEST survey on “Novel Cell Therapy Processing,” and novel therapies in the news.

CT Quality Operations: Ed Brindle, MSc, MLT(CMLTO), and Deb Sesok-Pizzini, MD, MBA, hosted talks on “AABB Accreditation Common CT Non-Conformances Cord Blood and Somatic Cell Facilities” by Sarah Dickerson, MT(ASCP), and “Achieving Quality Through Data Management” by Kathleen Fortune, MT(ASCP).

CT Product Collection and Clinical Practices: Subsection leaders Tom Spitzer, MD, Jay Raval, MD, and Joseph ‘Yossi’ Schwartz, MD, MPH, presented journal club entries on the risks and potential benefits of stem cell therapies, as well as initiatives on cord blood and bone marrow quality.

CT Product Manufacturing and Testing: Ronit Slotky, PhD, MSc, and Yen-Michael S. Hsu, MD, PhD, hosted speakers on several topics. Flow cytometry is currently the only methodology capable of rapidly measuring the clinically useful CD34+ surrogate marker of graft adequacy in all sources of hematopoietic stem cells. D. Robert Sutherland, MSc, who helped to develop the CD34+ ISHAGE flow cytometry protocol and contributed on the topic in the AABB book “Cellular Therapy: Principles, Methods, and Regulations,” presented “Going Beyond ISHAGE.” Ika Prawoko, MT(ASCP), presented “Preparing to Transition From An Older to Newer LIMS Platform” and shared a few tools, including the “SOP Writing Guide” and “Flowchart Details Template.”

Cord Blood Subsection: Subsection leaders Salem Akel, PhD, and Gwen Epstein, BSC, RT, led several discussions, including “Passive vs Controlled-Rate Freezing Quality of HPC Products” presented by Akel.

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.

 

Editor: Christina Celluzzi
Contributors: Kathy Loper, Jessica Yozwiak

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