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AABB CellSource - January 2017

 
AN UPDATE ON CELL THERAPY NEWS FROM AABB
January 2017

IN THE NEWS

Registration Opens for 15th International Cord Blood Symposium; AABB Issues Call for Abstracts

Registration for the 15th International Cord Blood Symposium (ICBS), to be held June 8-10 in San Diego, is open. AABB has also issued a call for abstracts for the meeting, which brings together world-renowned experts in the fields of umbilical cord blood and tissue research, and regenerative medicine. Information about registration rates, lodging and abstract submissions is available on the ICBS website. Abstracts will be accepted in the following areas: cord blood transplantation; cord blood and cord tissue banking; processing, expansion and business models; and non-hematopoietic stem cell transplant (non-HSCT) applications of cord blood, cord tissue and regenerative medicine. The deadline to submit an abstract is March 22. Information on the scientific program will be posted soon.

CT Section Welcomes New Faces to Coordinating Committee

Each year, the Cellular Therapies Section welcomes new members to its 16-seat Cellular Therapies Section Coordinating Committee (CTSCC) as veterans complete their terms. Members of the CTSCC, which helps to guide the association and its members on critical issues relating to cellular therapies, are elected by fellow CT Section members. This year, Magali Fontaine, MD, PhD, was elected to a second term and Mike Halpenny, MLT(CMLTO), Brian Jones, SBB, Ronit Slotky, PhD, and Missy Vogelpohl, MT(ASCP), MBA, were elected to a first term.

Transfusion Seeks Authors for “How Do I…” Articles

AABB’s peer-reviewed journal, “Transfusion,” is seeking submissions for its “How Do I...” section, which publishes expert opinions to clarify areas of practice and policy in transfusion medicine, blood banking, coagulation/hemostasis and cellular therapies. Individuals interested in providing materials for this section may submit a message through Manuscript Central with "How Do I ..." at the beginning of the title. Next, select “Brief Report” for the Manuscript Type. Questions regarding manuscript submissions can be directed to Beth Shaz, MD.

Feedback Sought on AABB Publications

AABB’s Publications Committee is considering producing a second edition of Cellular Therapy: A Physician’s Handbook to replace the Technical-Manual-derived Core Principles in Cellular Therapy. The committee requests feedback on the value of these publications. Comments may be sent to Jennifer Boyer at jboyer@aabb.org by Feb 1.

NHLBI Renews Production Assistance for Cellular Therapies

The National Institutes of Health’s National Heart, Lung, and Blood Institute announced the renewal of the Production Assistance for Cellular Therapies (PACT) initiative. PACT supports the development of novel cell therapy products through funding contracts at PACT processing facilities. The initiative has awarded five-year contracts to five cell processing facilities and a coordinating center: The Center for Cell and Gene Therapy, Baylor College of Medicine; the Center for Biomedicine and Genetics, City of Hope; the Interdisciplinary Stem Cell Institute Cellular Manufacturing Program, University of Miami, Miller School of Medicine; the Moffitt Cancer Center; the University of Minnesota, Molecular and Cellular Therapeutics; and The Emmes Corporation (a coordinating center). “We are very excited with continuing to support the cell therapy community and implementing new initiatives aimed at advancing the development of new cell therapies,” said NHLBI project director Lis Welniak, PhD.

PACT aims to promote the advancement of cellular therapies research in the regeneration of damaged/diseased tissues, organs and biologic systems, and in targeted treatments for serious diseases without effective therapies. The initiative will support novel translational research that advances the field of cellular therapies along IND-enabling pathways. PACT will begin accepting request-for-service applications Feb. 1. Additional information is available on the PACT website.

ICCBBA Changes Technical Advisory Group Name to Reflect Regenerative Medicine; Releases Consultation Document for Comment

The name of the Tissue Engineered Products Technical Advisory Group (TEPTAG) has been changed to the Regenerative Medicine Technical Advisory Group (RMTAG). Because "tissue engineered" has a specific meaning in European regulatory language, the group was renamed to prevent the incorrect association between TEPTAG and the rules applied by the EU Commission to human tissue engineered products. The two types of products under the governance of this TAG are regenerated tissues and seeded scaffolds. RMTAG has released the “Internationally Standardized Terminology for Regenerated Tissue and Seeded Scaffolds” consultation document for public comment. Comments may be submitted to regentissue@isbt128.org through Jan. 31.

ACOG Recommends Delayed Umbilical Cord Clamping for Healthy Infants

The American College of Obstetricians and Gynecologists (ACOG) issued a recommendation to delay umbilical cord clamping for all healthy infants until at least 30-60 seconds after birth, citing numerous benefits to most newborns. The latest Committee Opinion, which updates ACOG’s 2012 guidance, is based on recent research showing that while preterm infants are likely to benefit the most from the additional blood volume gained from the placenta, term infants can also benefit. These benefits may have a favorable effect on developmental outcomes.

WMDA, BMDW and NetCord Announce Consolidation

The World Marrow Donor Association (WMDA), Bone Marrow Donors Worldwide (BMDW) and NetCord were formally consolidated on Jan. 1. Operations and activities of BMDW and NetCord are now provided through WMDA. These include the BMDW Search & Match Service and NetCord, now a part of the WMDA Cord Blood Working Group. The newly consolidated organization has a combined mission to assure that the donation of marrow, peripheral blood and cord blood is safe and results in high-quality grafts for patients; to provide better service to transplant centers and registries searching for the best source of cells for their patients; to continue to share best practices for operating donor registries and cord blood banks; and to increase awareness of this combined activity. The leadership of the newly consolidated organization will provide more information as it becomes available. Questions can be addressed to Lydia Foeken, executive director of WMDA, at lydia.foeken@wmda.info.

21st Century Cures Act Set to Accelerate Medical Initiatives

The 21st Century Cures Act, which includes numerous provisions to speed the approval process for prescription drugs and medical devices in the United States, was signed into law by President Barack Obama in December. The Act requires the Food and Drug Administration to evaluate the use of “real-world” evidence to support new uses for previously approved drugs, and to support or satisfy post-approval requirements to confirm the safety and effectiveness of treatments. It will allow FDA to accelerate the approval of advanced regenerative and stem cell therapies. The legislation puts the U.S. on the same playing field as many other developed countries, including those in the European Union and Japan, which already have accelerated regulatory pathways for regenerative therapies.

Facebook Founder Looking to Create Facebook for Human Cells

The BioHub, a new $600 million center funded by Mark Zuckerberg, co-founder and chief executive officer of Facebook, has commenced its premier project: to facilitate a vast directory of human cells or “cell atlas.” The BioHub and its co-president, biophysicist Stephen Quake, who invented microfluidic devices used for cell analysis, are part of a consortium of global researchers who say mapping the millions of cells in the human body could help drug makers and scientists find new ways to treat disease. The BioHub wants to further technologies enabling scientists to analyze cells and their molecular contents directly in tissue samples. The BioHub is set to become the biggest funder of cell atlas technologies.

REGULATORY AND GOVERNMENT UPDATE

Comprehensive Ambulatory Payment Classification for HSCT

The Centers for Medicare and Medicaid Services (CMS) recently established a new Comprehensive Ambulatory Payment Classification (C-APC) for allogeneic hematopoietic stem cell transplantation (C-APC 5244). In response to comments received from stakeholders, including AABB, CMS agreed that it is preferable to use only claims with the CPT code for the transplant (CPT 38240) and the revenue code (0819) for the donor acquisition costs to calculate the payment rate for the new C-APC. CMS used this methodology to finalize a CY 2017 payment rate of $27,752, which is significantly higher than the proposed payment rate ($15,267). In addition, CMS updated the Medicare hospital cost report (Form CMS-2552-10) by adding a new cost center 77, “Allogeneic Stem Cell Acquisition,” to Worksheet A (and applicable worksheets) with the standard cost center code of “07700.” This new cost center will be used to record acquisition costs related to allogeneic stem cell transplants (defined in Section 231.11, Chapter 4, of the Medicare Claims Processing Manual).

Unrelated Donor Screening Materials Updated for HPC Products

FDA addressed the eligibility of HCT/P donors exposed to Human Derived Clotting Factor Concentrates [HDCFCs] in a recent guidance. The agency determined that deferring donors based on firsthand or sexual exposure to HDCFCs is no longer necessary to maintain recipient safety. In response to revised recommendations, Hematopoietic Progenitor Cell (HPC) Unrelated Donor History Questionnaires (UDHQ) have been revised. Questions relating to relevant communicable disease agents and disease risk exposure related to human derived clotting factors have been removed from both the HPC Cord Blood DHQ and the HPC, Apheresis and Marrow DHQ. For comments or questions on this guidance or the changes to the DHQs, please contact regulatory@aabb.org.

AABB International Regulatory Website Content Updated

The AABB Center for Cellular Therapies has updated and expanded its International Competent Authorities webpage. The updated content includes revisions to the legislative and regulatory framework for cellular therapy products in Canada and Japan, as well as additional information on South Korea.

RESEARCH FOCUS

CRISPR Gene-Editing Tested in a Person for the First Time

In a landmark clinical trial, Chinese researchers injected a patient with CRISPR-edited white blood cells in an effort to treat cancer. Due to its relatively low cost and high efficiency, CRISPR, as explained in this video, is revolutionizing gene-editing procedures. The Chinese trial enrolled 10 patients with metastatic non-small cell lung cancer, all of whom had previously been given a life expectancy of about six months and were non-responsive to current treatments. Researchers exploited the cut-and-paste mechanism of CRISPR to modify the patient’s own immune cells. Patients’ T cells were isolated and modified to turn off a gene responsible for programmed cell death (PCD-1). With this switch off, investigators hope that the released T cells are more adept at killing cancer than foreign drug compounds, minimizing the risks to the patient’s healthy cells. Results from this trial should inform us on how human bodies actually received CRISPR-edited cells and whether there are any unwanted side effects. The United States will likely soon approve its own trials with this technology.

Stem Cell Technology Mimics Salamander Limb Regeneration Ability

New advancements are being made in the field of tissue regeneration. In research published in PNAS, Chandrakanthan et al describe the generation of tissue-regenerative multipotent stem cells (iMS cells) by treating mature bone and fat cells transiently with a growth factor [platelet-derived growth factor–AB (PDGF-AB)] and 5-Azacytidine, a demethylating compound that is widely used in clinical practice. Unlike primary mesenchymal stem cells — which are used in clinical practice to promote tissue repair despite little supporting evidence — iMS cells contribute directly to in vivo tissue regeneration in a context-dependent manner without forming tumors. Modified cells injected into a damaged area stimulate regeneration, mimicking how salamanders exhibit plasticity to regrow lost limbs and tails. The researchers report that their method can be applied to both mouse and human somatic cells to generate multipotent stem cells and has the potential to transform current approaches in regenerative medicine. The science behind this new stem cell technology — how it works and how cells know what to generate and where — is still unclear. Scientist think that the cells lose their hard-wired identity, with the fat cell “forgetting” what it is and responding to the surrounding cells.

Researchers Develop Simple Method to Determine Purity of Adipose-Derived Stem Cell-Based Therapies

DuFrane et al describe a simple tool that may accelerate the delivery of safe and effective adipose stem cell (ASC)-based therapies to the medical community. The tool involves rapid testing release of specific growth factors, including vascular endothelial growth factor [VEGF], hepatocyte growth factor [HGF], insulin-like growth factor 1 [IGF-1], stromal cell-derived factor 1α [SDF-1α] and basic fibroblast growth factor [bFGF]. Notably, it can specifically discriminate fibroblasts and ASCs. Testing can be performed within 24 hours before the release of the ASC to patients.The team suggests the parameters of their tool could facilitate development of international standards.

Video Describes In vitro Model to Study the Central Nervous System

Fibroblasts can be used to create an in vitro neural model to replace animal testing. This video, produced by Johns Hopkins University, describes how fibroblasts removed from human skin can be reprogrammed into induced pluripotent stem (iPS) cells and differentiated into neural stem cells. The in vitro model has potential for use in a variety of studies exploring mechanisms in the central nervous system, toxicity and brain disease.

What Will Humans Look Like in 100 Years? Cell Therapies May Play a Role

Humans can evolve bacteria, plants and animals. Futurist Juan Enriquez questions the ethics of evolving the human body. In a visionary TED talk that discusses everything from medieval prosthetics to present-day neuroengineering and genetics, Enriquez sorts out the ethics associated with evolving humans. He also imagines the ways in which the human body will have to transform if future generations are to explore and inhabit places other than Earth.

First Prospective Trial of Unrelated Donor Bone Marrow Transplantation for Children with Severe Sickle Disease is Important First Step in Extending Curative Therapy

Children with sickle cell disease (SCD) experience organ damage, impaired quality of life and premature mortality. Matched donor bone marrow transplant for SCD is now well-established for children with severe disease. Can similar results be achieved in patients without HLA-matched family donors? In a phase 2 trial, the Blood and Marrow Transplant Clinical Trials Network Sickle Cell Unrelated Donor Transplant study, a reduced-intensity conditioning (RIC) regimen was adopted with bone marrow and cord blood grafts. Shenoy et al report their results of bone marrow transplant in the journal “Blood.” The primary objective was one-year event-free survival (EFS) after HLA allele-matched unrelated donor transplant. The conditioning regimen included alemtuzumab, fludarabine, melphalan and graft-versus-host disease (GVHD) prophylaxis. The trial met its one-year EFS, and the conditioning regimen was associated with significantly improved health-related quality of life. However, although the RIC provided successful engraftment in the majority of patients, the regimen cannot be considered safe for widespread use without modification due to regimen-related toxicity and a high rate of chronic GVHD, which was the predominant cause of mortality. The researchers suggest that future trials should focus on strategies that minimize the risk of GVHD and include stopping rules for chronic GVHD.

EVENTS, OPPORTUNITIES, RESOURCES

AABB Debuts eCasts

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

02/23 Apheresis Optimization in Gene and Immunotherapy Development in Non-Mobilized Patients and Donors (#113)
03/30 Expanded Use of Donor Lymphocyte Infusion (DLI) (#123)
04/05 Significant Changes to the 8th Edition of Standards for Cellular Therapy Services (#125)
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)

Easy Access to Resources for AABB Members

In a continuing effort to improve member benefits, AABB recently added a new consolidated Member Resources page. Members can explore opportunities for networking and volunteering, relevant news sources, educational programs, advocacy updates 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.

Biorepository Resource from the U.S. National Institutes of Health

The Biorepositories and Biospecimen Research Branch (BBRB) of the Cancer Diagnosis Program (CDP) provides leadership, tools, resources and policies in biobanking for the global biomedical research community to enable translational research and precision medicine. BBRB also develops biorepository standards and facilitates biospecimen science studies that form the basis of evidence-based practices to guide clinical cancer research and other biomedical studies which utilize biospecimens.

precisionFDA Aims to Harness the Power of Scientific Collaboration

Doctors have potentially at their fingertips information that allows them to individualize a diagnosis, a treatment or even a cure based on a patient’s genes. As part of the Precision Medicine Initiative, the FDA launched precisionFDA, an online, cloud-based portal that allows scientists from industry, academia or government and other partners to collaborate to foster innovation. The portal is also intended to assist in developing the science behind a method of “reading” DNA known as next-generation sequencing. Next-generation sequencing allows scientists to compile a vast amount of data on a person’s exact order or sequence of DNA. Recognizing that each individual’s DNA is slightly different, scientists can look for meaningful variances that can be used to predict someone’s risk of disease and potential response to treatment, and to assess his or her current state of health. Ultimately, the information gleaned from these differences could be used to design a treatment tailored to specific individuals.

FOR MEMBERS ONLY

The Latest on CT Subsection Activities

The subsections worked on proposal submissions for education sessions for the 2017 AABB Annual Meeting, to be held October 7-10 in San Diego, Calif.

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 cord blood. Sandhya R. Panch, MD, a 2016 NBF award grantee, presented “Optimizing Hematopoietic Stem and Progenitor Cell Quantities for Cellular Therapy,” and Ed Brindle, MSc, MLT(CMLTO), discussed “AABB CT Standards 7th Edition – Significant Changes.”

Regulatory Affairs: Subsection leaders Ljiljana Vasovic, MD, and Olive Sturtevant, MHP MT(ASCP)SBB/SLS, led discussions on different regulatory issues. Guest speaker Elina Linetsky, PhD, presented “Manufacture of Pancreatic Islets to Achieve the BLA: A Multi-Center Approach.”

CT Management: This group is led by subsection leaders Suzanne Dworsky, MBA, MT(ASCP), and Brian Jones, SBB(ASCP). The two leaders hosted interesting roundtable discussions on “Operational and Financial Management of Clinical Research Products not Billable to Third Party Payers” and “Managing Productivity in a Cell Therapy Lab Which Includes Standard of Care and Clinical Research” at the “Solve It! Scenarios in Cellular Therapy” session at the 2016 AABB Annual Meeting.

Novel Therapies and CT Product Development: This subsection formally welcomed Richard Schaefer, MD, to the leadership team alongside Magali Fontaine, MD, PhD. Pampee Young, MD, PhD, who completed her tenure as a member of the CTSCC, stepped out of the leadership role but will continue to participate in subsection and other AABB activities. The members gratefully acknowledge Young’s innovative contributions to this subsection. Schaefer presented “The Challenges of Translating Mesenchymal Stromal Cells (MSC) Products to the Clinic: (How) Can We Improve Their Therapeutic Potential in Regenerative Medicine?” and hosted a journal club leading discussion on a rich source of MSCs from bone marrow for clinical application.

CT Quality Operations: Ed Brindle, MSc, MLT(CMLTO), and Deb Sesok-Pizzini, MD, MBA, paid tribute to subsection leader Kathy Fortune, BS, MT(ASCP), for her years of subsection service and leadership. Fortune, who recently completed her tenure as a CTSCC member, helped establish the quality tone of this subsection, presenting many topics of interest and practicality in a professional and entertaining manner. Her “Jeopardy” game, customized for Quality Operations, was a holiday favorite. AABB looks forward to her continued participation in CT activities.

CT Product Collection and Clinical Practices: Subsection leaders Tom Spitzer, MD, Jay Raval, MD, and Joseph ‘Yossi’ Schwartz, MD, MPH, and guest presenter, Laura Cooling, MD, presented “Scenarios in Cellular Therapies” on peripheral blood stem cell transplantation conditioning regimens, transfusion management and infusion reactions.

CT Product Manufacturing and Testing: Congratulations were extended to Ronit Slotky, PhD, MSc who assumed the role of Leader of this subsection with her election to the CTSCC. Yen-Michael S. Hsu, MD, PhD, joined Slotky as Associate Leader. AABB would like to thank Vasiliki Kalodimou, PhD, for her leadership while upon her completion of her tenure on the CTSCC. Kalodimou plans to continue to be active on the subsection, exemplified by delivery of her presentation, “Characterization of MSCs- Are We Doing It Correctly?” Leading flow cytometry expert Robert Sutherland presented “Beyond the ISHAGE: Evolving Guidelines for CD34 enumeration.”

Cord Blood Subsection: Subsection leaders Salem Akel, PhD, and Gwen Epstein, BSC, RT, led discussions to develop topics for future presentations.

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. A new SLS projects page has been developed to showcase content presented to the subsection.

 

 

Editor: Christina Celluzzi
Contributors: Kathy Loper and Brandon Sandine

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