IN THE NEWS
AABB-CCT Participates in TERMIS-AM and World Stem Cell Summit
The AABB Center for Cellular Therapies hosted a session at the Tissue Engineering Regenerative Medicine International Society–Americas, or TERMIS-AM annual conference and exposition held Dec. 13-16 in Washington, DC. AABB representatives assisted with the organization and moderating of the Thematic Working Interest Group, or TWIG, titled “Stem Cells and Cell Therapies and Development Biology Cell and Signaling” which was divided into six different oral presentation sessions titled “Cardiac and Smooth Muscle Cell Differentiation and Function,” “Stem and Progenitor Cell-based Approaches to Musculoskeletal Tissue Therapies,” “Neurogenic Differentiation and Tissue Engineering Strategies,” and “Stem Cell Function, Migration and Trafficking,” among others.“ The next TERMIS-AM meeting is scheduled for 2016 in San Diego. AABB staff shown in image represented AABB Consulting Services at the exhibit booth.
AABB also hosted a session “Emerging Technologies, Standards and Applications in Cellular Therapy” at the World Stem Cell summit held Dec. 3-6 in San Antonio. Invited speakers included industry representatives MaxCyte, TERUMO BCT and ORIG3N.
Merger of Genetics Policy Institute and Regenerative Medicine Foundation
The Genetics Policy Institute, or GPI, producer of the annual World Stem Cell Summit, and the Regenerative Medicine Foundation, or RMF, have agreed to merge to leverage their resources in the advancement of stem cell research, regenerative medicine and other advanced technologies targeting cures. The combined organization will use the RMF name. “The merger of these nonprofit leadership organizations will create the leading comprehensive regenerative medicine support organization in the world. It ensures that we are in a position to advance the research and innovations that are creating the future of medicine,” said Bernard Siegel, GPI’s executive director who will hold that position for the combined organization. Siegel’s counterpart, RMF’s executive director, William Hinman, will hold an unspecified executive position in the merged organization. RMF’s founder and chairman, Anthony Atala, MD, will continue to sit on the board of directors of the new organization.
Researchers Make Blood Vessels Grow by Shining a Light on Skin
Researchers have found a way to stimulate the growth of blood vessels — an important part of healing — by shining ultraviolet light on the skin. Reported online in Nature Materials, García and colleagues impregnated a water-based gel, or hydrogel, with a molecule called RGD peptide, which signals cells to stick to and grow on new tissues. By irradiating this ‘system’ researchers affected immune responsiveness, allowing cells to grow around the peptide-impregnated samples in a murine model.
License for Phase I CAR T Product Candidate Targeting CD22 for Hematological Malignancies
Juno Therapeutics, which focuses on developing cell-based immunotherapies based on its chimeric antigen receptor, or CAR, and high-affinity T cell, has entered into an agreement to obtain a license from Opus Bio Inc. for a CAR-T cell product candidate targeting CD22. This protein is expressed on most B cell leukemias and lymphomas. According to Juno, its CAR and T cell receptor, or TCR, platform activates a patient’s own T cells so they attack cancer cells. Through genetic engineering, a gene for a particular CAR or TCR construct is inserted into the T cell enabling it to better recognize cancer cells. The modified T cells can be infused into the patient or frozen and stored for later infusion. The CD22-targeted CAR T cell product candidate was developed by the National Cancer Institute, or NCI, under a cooperative research and development agreement with Opus Bio. NCI began enrollment for a phase I trial evaluating pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia or non-Hodgkin’s lymphoma. The NCI-sponsored trial will enroll patients with CD22-positive cancers, with both CD19 positive and CD19 negative patients eligible for treatment.
New Stem Cell Class Discovery Could Speed up Research
Canadian researchers have discovered a new class of stem cells they say could be safer and better to use in medical research, which could eventually lead to new treatments for illnesses. Scientists have reported the ability to rewind adult cells into a primitive or "pluripotent" state that mimics embryonic stem cells. The research team of Dr. Andras Nagy described a new class of the cells in mice they called F-class after their fuzzy appearance. The cells could be used in biomedical research. Whether F-class cells can be safely used in the clinic is still not clear and remains years away from being answered.
Nobel Prize Proceedings: Highlights and Insights “Revealed” to the Public
Episodes of the Nobel Prize ceremonies and speeches are available. The episodes show prestigious events, and highlight the discoveries and contributions made to humankind. Nobel laureates from all subject areas — Medicine, Physics, Chemistry, Economics Sciences, and Literature — provide insight into what gives them drive and creativity, illustrating their potential impact globally and serving as a source of inspiration at many levels. The 2014 Nobel Prize in chemistry was awarded for the development of super-resolved fluorescence microscopy, which has made visible essential details and movements of the molecules of life. This microscopy technique has implications for other scientific and medical disciplines.
Nobel Dialogue on “Genetics Revolution” to Take Place Outside Sweden for the First Time
The first Nobel Prize Dialogue to occur outside Sweden will be held in Tokyo on Mar. 1, 2015. Organized between Nobel Media and the Japan Society for the Promotion of Science, the conference aims to bring science and society closer together. Inspired by Nobel Week Dialogue, which has taken place in Sweden since 2012 on the day before the Nobel Prize Ceremony, the conference will be a high‐level celebration of the power of ideas focused on the topic "The Genetic Revolution and its Future Impact." Event participants will explore the societal implications of developments in genetics, genomics and their potential applications focusing on personalised medicine, genetically modified organisms and human evolution. There also will be specific panel discussions addressing genetics and genomics in Asian countries. Participants can ask questions, at the venue and online, engaging in a dialogue that will continue after the conference closes. The ‘free-to-attend’ conference is open to anyone interested in participating in discussions with Nobel Laureates, other prominent scientists, key policy makers and opinion leaders. A webcast is planned.
Most Cancer is Beyond One’s Control
In a paper published in Science, scientists Christian Tomasetti and Bert Vogelstein from Johns Hopkins University describe a new factor, a tissue’s stem cells, that may explain as much as two-thirds of the difference in cancer risk between different tissues. The more likely a tissue’s stem cells are to divide and develop DNA errors or mutations leading to uncontrolled growth, the more likely that tissue is to develop tumors.
REGULATORY AND GOVERNMENT UPDATE
AABB Submits Comments on FDA Draft Guidance: Same Surgical Procedure Exceptions
AABB submitted comments on the Food and Drug Administration’s “Draft Guidance for Industry: Same Surgical Procedure Exception under 21 CFR 1271.15(b): Questions and Answers Regarding the Scope of the Exception.” The draft guidance was issued in an Oct. 23 "Federal Register" notice, and AABB developed comments with input from members of the Cellular Therapies Regulatory Subsection. The comments from AABB contain a request to add the phrase “or those of a CMS-approved accreditation organization” to the FDA’s requirement that “all hospitals that store autologous cells or tissues for subsequent application in the same patient must follow the guidelines on the Joint Commission on Accreditation of Healthcare Organizations.” In general, AABB was grateful for FDA’s clarifications put forth in the draft guidance document. A final guidance will be published after FDA has reviewed public comments.
FDA Issues Two Draft Guidance Documents Relating to HCT/Ps
The FDA published two draft guidance documents in late December. The draft guidance titled “Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) from Adipose Tissue: Regulatory Considerations” includes recommendations for sponsors, clinicians and those who manufacture and use adipose tissue to facilitate compliance with the regulatory framework for HCT/Ps. The "Federal Register” notice provides information on submitting comments to the FDA. The second guidance document published in the “Federal Register” is titled “Minimal Manipulation of Human Cells, Tissues, and Cellular- and Tissue- Based Products” and provides recommendations to help HCT/P manufacturers, healthcare providers and FDA staff meet the “minimal manipulation” criterion for HCT/Ps. Comments on both draft guidance documents will be submitted by AABB and readers are encouraged to submit comments for consideration to email@example.com by Feb. 10.
Cord Blood Transplantation: Past, Present and Future
Allogeneic hematopoietic stem cell, or HSC, transplantation is an important treatment option for patients with poor-risk hematological malignancies. Umbilical cord blood, or CB, is an effective source of HSC support and allows for ‘fast’ availability of frozen samples and avoids invasive procedures for donors. Current excitement in the field revolves around the development of safer techniques to improve homing, engraftment and immune reconstitution after CB transplantation. In their review in “Stem Cells Translational Medicine,” J. Munoz et al. discuss the past, present and future of CB transplantation.
Study Validates MSC Banking Protocol
Mesenchymal stromal cells, or MSCs, are a heterogeneous population that can be isolated from tissues including umbilical cord Wharton's jelly, or UC-WJ. An increasing number of clinical trials consider MSCs as a potential anti-inflammatory or regenerative medicine agent. Creating a repository of MSCs would increase their availability for clinical applications. A study by K. Chatzistamatiou, published in “Transfusion,” assessed the optimal isolation and cryopreservation procedures to facilitate WJ MSC banking. Cells isolated from UC-WJ using enzymatic digestion or plastic adhesion methods were studied for their efficacy, growth kinetics, immunophenotype, differentiation potential and response to freezing. Data showed that viable MSCs could only be isolated from fresh UC-WJ tissue, setting the foundation for clinical-grade banking.
Qualification of Academic Facilities for Small Scale Automated Manufacture of Autologous Cell-Based Products
Academic centers, hospitals and small companies are significant contributors to the development of autologous cell-based therapies. P. Hourd et al. share their experiences from the qualification of a new cell therapy manufacturing facility in a British academic setting in the journal “Regenerative Medicine.” The authors focus on streamlining the qualification effort, the challenges, project delays and inefficiencies encountered and the lessons learned.
Cranial Transplantation of Human iPS Cells Ameliorate Radiation-Induced Cognitive Impairment
Cranial radiotherapy is beneficial for the treatment of central nervous system malignancies. However, it frequently leads to progressive and debilitating cognitive deficits. Cancer survivors have little or no clinical recourse for the neurocognitive decline that adversely affects quality of life. Preclinical studies have demonstrated the feasibility of using human stem cell transplantation in the irradiated brain to ameliorate radiation-induced cognitive dysfunction. In an article published in “Stem Cells Translational Medicine,” M.M. Acharya et al. define windows for transplantation following irradiation that maximize cognitive benefits using induced pluripotent stem cell-human neural stem cells or iPSC-hNSCs. Animals were given an acute dose of radiation aimed only at their heads and grafted with iPSC-hNSCs at various time points following irradiation. Animals receiving stem cell grafts showed improved hippocampal spatial memory and contextual fear-conditioning performance compared to irradiated sham-surgery controls. Radiation-induced inflammation was significantly attenuated across multiple hippocampal subfields in animals receiving iPSC-hNSCs. These results expand findings demonstrating that protracted stem cell grafting provides improved cognitive benefits following irradiation that are associated with reduced neuroinflammation and may provide practical relief and a chance for improved quality of life.
Unlocking the Secrets of Our Cells
Reviewing materials or comments from researchers can offer a glimpse of the thought processes and research approaches that lead to scientific advances and clinical applications. A half hour video documentary recognizes four scientists whose work has enabled progress in medical research: the 2012 Nobel laureates in chemistry, Robert J. Lefkowitz and Brian K. Kobilka, and the 2012 Nobel laureates in medicine, Sir John B. Gurdon and Shinya Yamanaka. The laureates talk about their background, their work and the story behind their groundbreaking research. In addition, leading scientists explain how the Nobel prize-winning research has influenced their work and patients describe how they have been helped by these medical developments.
EVENTS, OPPORTUNITIES, RESOURCES
CT AABB Audioconferences and Webinars
Upcoming 2015 CT audioconference programs:
03/04 Significant Changes to the 7th Edition of Standards for Cellular Therapy Services (#154853)
04/22 ISBT 128 Implementation: Challenges and Rewards (#154857)
06/03 Donor Medical History Interview for HCT/PS (#154860)
11/18 Using Competency Assessments to Assure Quality (#154867)
2015 CT Webinar Schedule:
02/19 Reducing Microbial Contamination in CT Products (#1510)
03/05 Accelerating Cellular Therapies to the Patient (#1512)
03/26 Programs/Methods Used in Training Staff in CB/apheresis Collections (#1513)
05/07 Survey of New Applications: Experiences with the Spectra Optia, Terumo BCT Quantum and Miltenyi Prodigy (#1514)
07/23 Funding Strategies for Cord Blood Banks (#1515)
08/06 Technical Topic Medley-How Cellular Therapy Facilities Handle Daily Occurrences and Quality Issues with the Available Resources (#1516)
08/20 Workload Justification and Staffing (#1517)
09/24 Topical Application and Bio-printing of Cells for Regenerative Medicine Applications (#1518)
10/01 Platelet Rich Plasma in Regenerative Medicine (#1519)
10/15 Supplier Quality Agreements for Cellular Therapy Products (#1521)
A complete listing of past 2014-2015 educational events is available for purchase by visiting the website.
WBMT Symposium Presentations are Available Online
Presentations from the Worldwide Network for Blood and Bone Marrow Transplantation, or WBMT, 3rd Workshop and Scientific Symposium held Nov.14-16 in Capetown, South Africa have been posted to the WBMT website.
2nd Edition of HSC Transplantation Handbook for Clinicians Available
The highly anticipated second edition of Hematopoietic Stem Cell Transplantation: A Handbook for Clinicians is now available in the AABB Marketplace. This edition features an enhanced educational trainee experience, with new chapters on nonmalignant diseases, haploidentical transplants, photopheresis and health care financial considerations. Preorder your copy today. Publications will ship on Jan. 30.
FOR MEMBERS ONLY
The Latest Updates on CT Subsection Activities
Members of the AABB Center for Cellular Therapies’ subsections meet regularly via teleconferences and participate in interactive activities including the 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 encountered by those working in 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. This quarter the group enjoyed presentations on cord blood (CB) banking in different countries. Recent presentations included “Cord Blood Banking in Japan,” (Monoki Takanashi, MD, PhD), “Activities of ALLCORD-Seoul Metropolitan Government Public Cord Blood Bank,” (Sue Shin, MD, PhD), and a discussion of the “Singapore CB Bank” (Arun Prasath, MSc, MPhil).
Quality Operations: Angie Ondo MT(ASCP) (Johns Hopkins Hospital) gave a presentation titled “Environmental Monitoring.” For the holidays the group enjoyed their quality-focused version of the popular gameshow “Jeopardy.”
Product Manufacturing and Testing, or PMAT: Allison Hubel, PhD (BioCor, University of Minnesota), gave a presentation titled “Cord Blood: Markers for Storage Lesion.”
CT Management: This subsection created a short survey to gauge practices for day-to-day operational management of cellular processing in laboratories in the United States. Results of the “Management of Cellular Processing” survey provide an initial understanding or ‘snapshot’ of where participants fall along the spectrum of practice compared with their peer group. The spectrum of practice is based on the number of cellular therapy products manufactured. Information from the study may be useful in justifying approvals for additional personnel. Suzanne Dworsky, MT(ASCP) (MD Anderson Cancer Center) associate leader of the subsection, is available to discuss any ideas or CT Management subsection projects potentially stemming from the results collected from this survey. She can be contacted via email at firstname.lastname@example.org.
Regulatory Affairs: This subsection hosted several presentations this quarter including “Same surgical procedure exception under 21CFR1271.15(B): Questions and Answers Regarding the Scope of the Exception” by Fran Rabe, MS, CQM(ASQ) (University of Minnesota) and “Harmonising Standards in HSCT: AHCTA Initiatives” by Kathy Loper, MHS, MT(ASCP) (Senior Director, AABB Center for Cellular Therapies).
Novel Therapies and CT Product Development: Subsection leaders are always looking for volunteers to present articles, original research or techniques that may be useful to other members. Interested members are encouraged to participate in teleconferences, which serve as excellent practice for professional development or speaking in an informal setting among peers. This subsection held a series of journal article discussions on various topics. Magali Fontaine, MD, PhD (University of Maryland School of Medicine) led a discussion titled “Generation of Functional Human Pancreatic beta cells in vitro.” Pampee Young, MD, PhD (Vanderbilt University Medical Center), led a discussion on CellNet, a network biology platform that determines whether engineered cells are equivalent to their target tissues; diagnoses aberrant gene regulatory networks; and prioritizes candidate transcriptional regulators to enhance engineered conversion. CellNet can be used to improve direct conversion and to uncover properties of engineered cells. The subsection discussed how this tool may be useful for their own laboratory studies. Andrew Fesnak, MD (University of Pennsylvania) recently gave a presentation on “Designing a Novel Fellowship in Cellular Therapy” − a program designed to prepare physicians to be medical directors of cellular therapy facilities.