An Update on Cell Therapy News from AABB |APRIL 2020
IN THIS ISSUE
Is Now the Time to Experience the Cellular Therapies Certificate Program?
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 CT. The program features 12 narrated modules covering scientific, operational and regulatory topics. Students who work through the modules, complete each assessment with a score of 80% or higher and complete a program evaluation form will receive a certificate of completion. The program also provides an opportunity to earn up to 37 continuing education credits (CMEs).
Registration for AABB
eCasts is available for both 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 credits.
On-demand recordings of eCast sessions are available for those unable to attend a live eCast.
Upcoming CT eCasts include:
Christina M. Celluzzi, PhD
Kathy Loper, MS, MT(ASCP)
AABB Center for Cellular Therapies
4550 Montgomery Avenue, Suite 700, North Tower, Bethesda, MD 20814, +1.301.907.6977
2020 AABB Annual Meeting Deadline for Abstract Submissions Extended to May 15
The health and safety of the AABB community is AABB’s highest priority. The Association continues to monitor the rapidly changing situation related to the COVID-19 pandemic. Currently, the AABB 2020 Annual Meeting is scheduled as planned for October 3-6 in Baltimore, and AABB is committed to maintaining all appropriate health and safety measures. Any changes to the status of the meeting as a result of new developments, federal travel restrictions and/or local, state or regional decisions to close venues will be communicated immediately.
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Labeling Standard Available for Public Comment
Working Group Seeks Feedback for Proposed CT Product Stability Program
The AABB and International Society for Cell and Gene Therapy (ISCT) Joint Working Group (WG) asks the CT community to participate in a survey created by its Program Stability Project team to aid in providing a roadmap and recommendations for the establishment of a cellular therapy product stability program. The WG, formed to develop projects of mutual interest, is looking to identify potential gaps and opportunities for developing stability programs for cryopreserved hematopoietic progenitor cell (HPC) products. The WG requests that only one member from any participating organization
complete and submit the survey by May 8.
Openings on USP’s Council of Experts and Expert Committees
Every 5 years,
U.S. Pharmacopeia invites qualified candidates — scientists, academicians, regulatory professionals, health care practitioners and others who work with medicines and foods — to apply to serve as decision makers on its Council of Experts and Expert Committees. Qualified candidates may also be invited to serve as advisors on expert panels that make recommendations to expert committees. USP volunteers are called upon to utilize their best personal, professional and scientific judgment to benefit global public health. Members have found the experience to be both meaningful and enriching. New this 2020-2025 cycle, USP has added an Advanced Therapies Expert Committee to focus on quality standards for cell, gene and tissue-based therapies. Interested subject matter experts in this area can find additional information and a membership application for Biological Medicines openings on the USP
website. Applications are due by May 8. For questions or assistance with an application, please contact
Early-Career Training During the COVID-19 Crisis
AABB has developed
packages of highly discounted resources to allow early-career professionals to continue their professional development remotely following multiple requests. Various programs have reported that their MT students, fellows and residents have had to cut their rotations and/or learning short due to the COVID-19 crisis. AABB is committed to supporting these early-career professionals and continuing to cultivate a well-trained workforce, even in the face of adversity.
Coronavirus Resources Available
Coronavirus Resources web page is available to provide AABB’s latest information and tools related to coronavirus disease 2019 (COVID-19) — including information from the Food and Drug Administration, AABB and partner organizations related to cellular therapy products and COVID-19. AABB continues to monitor the COVID-19 situation closely and update the page as new information becomes available. Several resources are
available in Spanish.
AABB Launches COVID-19 Convalescent Plasma Website
AABB launched a new website to share information with the public, blood collectors and clinicians about convalescent plasma from individuals who have recovered from COVID-19 (CCP). FDA recently announced new guidelines permitting the use of CCP as an investigational treatment for patients with moderate or severe COVID-19 infections. Many AABB-accredited blood centers are now collecting CCP. The website,
www.COVIDPlasma.org, includes information to help identify eligible CCP donors and a tool to assist those donors in contacting their local blood center or hospital blood collector to schedule a CCP donation. In addition, the website provides CCP resources for blood collectors and the blood community.
REGULATORY AND GOVERNMENT UPDATE
AABB Releases Two New FDA Cell Therapy Regulatory Resources
AABB released two resources to consolidate and allow ease of access to information from FDA for cellular therapy manufacturers and distributers. The first resource,
FDA Product Inquiry Avenues and Designations for Expedited Review, is a collection of FDA pathways for product inquiry and designations for expedited product review. The second resource,
FDA CT Guidance and Reference Documents, is a dated listing of FDA's guidance and reference documents regarding cell and gene therapy. Both resources include a reference to the February 2019
guidance for industry, "Expedited Programs for Regenerative Medicine Therapies for Serious Conditions," which discusses a collaborative approach to clinical trials.
FDA Issues Updated Information for HCT/P Establishments Regarding the Coronavirus Pandemic
update does not recommend that establishments use laboratory tests to screen asymptomatic donors of human cells, tissues, and cellular and tissue-based products (HCT/Ps) for evidence of COVID-19 infection. According to FDA, it appears that SARS-CoV2, the virus that causes COVID-19, has only been detected in blood samples of a small percentage of severely ill patients.
However, the HCT/P establishment's responsible person must evaluate prospective donors and determine eligibility as per 21 CFR 1271.50. Based on the limited information available at this time, establishments may wish to consider whether, in the 28 days prior to HCT/P recovery, the donor cared for, lived with or had close contact with individuals diagnosed with or suspected of having COVID-19 infection; or were diagnosed with or suspected of having COVID-19 infection. The agency noted that while the potential for transmission of COVID-19 by HCT/Ps is unknown at present, there have been no reported cases of transmission of COVID-19 via these products.
FDA is continually assessing available scientific evidence to determine whether SARS-CoV-2 testing is warranted on certain types of HCT/Ps used in manufacturing biological products. The agency will continue to monitor the situation and issue updates as information becomes available.
FDA Guidance Clinical Trials of Medical Products during the COVID-19 Pandemic: Guidance for Industry, Investigators, and Institutional Review Boards
FDA plays a critical role in protecting the United States from threats, including emerging infectious diseases like COVID-19. The agency is committed to providing timely guidance to support continuity and response efforts to the COVID-19 pandemic. FDA released a guidance,
FDA Guidance on Conduct of Clinical Trials of Medical Products during the COVID-19 Pandemic, to provide general considerations to assist sponsors in assuring the safety of trial participants, maintaining compliance with good clinical practice (GCP) and minimizing risks to trial integrity during the pandemic. Given this public health emergency, the guidance is being implemented without prior public comment, which FDA determined not to be feasible or appropriate (see section 701(h)(1)(C)(i) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) and 21 CFR 10.115(g)(2)). This guidance document is being implemented immediately but remains subject to comment in accordance with the Administration’s good guidance practices.
FDA Extends TRG Rapid Inquiry Program Through Oct. 31
FDA extended its temporary Tissue Reference Group Rapid Inquiry Program (TRIP) to help manufacturers of human cells, tissues, and cellular and tissue-based products (HCT/Ps) obtain a rapid, preliminary, informal, non-binding assessment from FDA regarding how specific HCT/Ps are regulated. The program began on June 12, 2019 and will end on Oct. 31, 2020. Information on how to submit a TRIP request is available on the TRIP
Toward a New Framework For the Development of Individualized Therapies
In a comment in
Nature Gene Therapy, Marks and Witten of the FDA Center for Biologics Evaluation and Research describe personalized medicine — selecting the appropriate previously developed treatment off the shelf to address an individual’s condition — in the era of molecular medicine. An individualized approach to product development will require the implementation of a supporting regulatory framework that differs from the current approach. The process to develop such a framework must be transparent and will require input from patients, academics, industry and other stakeholder groups. The authors describe other issues for these products, which include determining the quantity of supportive preclinical evidence needed prior to patient treatment, the production of quality product fit for purpose and understanding the relevant disease-specific clinical information that should be captured when patients are treated. Available online, their
full comment includes specific examples.
Indications for HCT and Immune Effector Cell Therapy: Guidelines from the ASTCT
The American Society for Transplantation and Cellular Therapy (ASTCT) has updated its
recommendations for indications for hematopoietic cell transplantation (HCT). The revised recommendations include new data and incorporate indications for immune effector cell therapy (IECT) where appropriate. Indications for HCT/IECT were categorized as follows:
- Standard of care, where indication is well defined and supported by evidence.
Standard of care, clinical evidence available, where large clinical trials and observational studies are not available but has been shown to be effective therapy.
Standard of care, rare indication, for rare diseases where demonstrated effectiveness exist but large clinical trials and observational studies are not feasible.
Developmental, for diseases where pre-clinical and/or early phase clinical studies show HCT/IECT to be a promising treatment option.
Not generally recommended, where available evidence does not support the routine use of HCT/IECT.
Periodic reviews and updates of these guidelines will be made as new evidence becomes available (subscription required).
Nov/CCN3 Enhances Cord Blood Engraftment by Rapidly Recruiting Latent Human Stem Cell Activity
A protein called NOV/CCN3, which is normally found at low levels in the blood, can be used to rapidly increase the number of HSCs in single umbilical cord blood units that are suitable for transplantation. Using cell cultures and mouse models,
Gupta et al. found that umbilical cord blood units exposed to NOV showed significantly more transplantation potential than regular samples. The frequency of functional HSCs in the sample increased six-fold following an 8-hour exposure. The researchers found they could rapidly manipulate blood stem cells to alter their state — changing non-functioning HSCs to functioning HSCs and enhancing cord blood engraftment potential. This finding offers a new strategy for improving blood transplants that may make available units that would otherwise be discarded. The next stage will involve clinical trials exploring how this process might benefit patients with blood cancers and other blood disorders.
New Technique ‘Prints’ Cells to Create Diverse Biological Environments
With the help of photolithography and creative use of programmable DNA, researchers have created a new technique that can rapidly “print” two-dimensional arrays of cells and proteins that mimic a wide variety of cellular environments in the body — be it the brain tissue surrounding a neural stem cell, the lining of the intestine or liver or the cellular configuration inside a tumor.
Scheideler et al. show that the new technique can be used to rapidly print intricate patterns of up to 10 different kinds of cells or proteins onto a flat surface. This technique could help scientists develop a better understanding of the complex cell-to-cell messaging that dictates a cell’s final fate, from neural stem cell differentiating into a brain cell to a tumor cell with the potential to metastasize to an embryonic stem cell becoming an organ cell.
Scientists Have Turned the Structure of the Coronavirus Into Music
Dozens of images of the novel coronavirus are available, and now scientists have come up with a way for people to hear it ? by translating the structure of its well-known spike protein into music. In this
musical representation appearing in the journal
Science, Marco J. Buehler says that, while we cannot see small nanoscopic objects like proteins or other molecules that make up virtually all living matter — including our cells, tissues and pathogens such as viruses — a computational algorithm allows us to make its material manifestation audible. Why set a virus to music? The new format can help scientists find sites on the protein where antibodies or drugs might be able to bind — simply by searching for specific musical sequences that correspond to these sites. This, the researchers say, is faster and more intuitive than conventional methods used to study proteins, such as molecular modeling. By comparing the musical sequence of the spike protein to a large database of other sonified proteins, it might be possible one day to find one that can stick to the spike — preventing the virus from infecting a cell.
Wiley Offers Complimentary Access to COVID-19 Research
Wiley is offering
complimentary access to all COVID-19-related research published in the company's journal portfolio. This includes content from AABB's
Transfusion journal, as well as from the American Society for Apheresis, British Blood Transfusion Society, British Society for Haematology, International Society for Laboratory Hematology, International Society of Blood Transfusion and International Society on Thrombosis and Haemostasis. AABB, Wiley and its other journal partners aim to help the community continue providing the best possible care for their patients.
Additional Reference DATABASES: The Centers for Disease Control and Prevention has listed
selected databases and journals on its website to help researchers find scholarly articles about COVID-19.
EVENTS, OPPORTUNITIES, RESOURCES
WMDA Service to Assist in Donor Selection Process Affected by Coronavirus Pandemic
The World Marrow Donor Association has posted information for search coordinators and transplant coordinators who are seeking information on changes in registry operations that have been affected by the coronavirus outbreak. To help with the donor selection process, WMDA has introduced a service,
WMDA Share. The WMDA has also established a cord blood support team for those needing urgent assistance with the selection process due to problems finding a suitable and available unrelated adult stem cell donor. Cord blood may be a good alternative if there are complications in the search process, as cord blood is quickly available, was collected before the COVID-19 outbreak and can be transported via cargo flights or by driver. To connect to the service, email Lydia Foeken at
NMDP Provides Resource on COVID-19 Impacts
The National Marrow Donor Program has a
web page providing up-to-date information on COVID-19 to all network partners. This information includes updates for transplant centers, donor centers, apheresis/collection centers and recruitment groups. A posting on its site under “Cord Blood Unit Considerations” includes an
educational presentation on cord blood thawing.
Preservation of Cellular Therapies: A Professional Short Course Available
The course “Preservation of Cellular Therapies” is being offered virtually through the University of Minnesota on May 18-19. The recorded lectures will be stored so that participants can watch them at their convenience.
Virtual VeloCTTI Master Protocol Expert Meeting to Support Master Protocol Studies
Recent events highlight the need for agile clinical trials that foster collaboration to address major public health threats and ongoing challenges related to the treatment of chronic and rare diseases. The Clinical Trials Transformation Initiative (CTTI) will host a virtual expert meeting Apr. 20-21 to test and refine CTTI tools to better develop master protocol studies in diverse therapeutic areas and global regions. CTTI is a public-private partnership co-founded by Duke University and FDA to develop and drive adoption of practices that will increase the quality and efficiency of clinical trials. The meeting overview and agenda are available
online, as is
registration for the meeting. Email Jamie Pollenz at
firstname.lastname@example.org with any issues.
CTTI Master Protocol Draft Resources ─ Open Comment Opportunity
CTTI requests feedback on draft resources created to help drive the appropriate use of master protocols in diverse therapeutic areas. These resources include a master protocols roadmap, an FDA engagement tool, a business and operations plan template, a protocol development template, a vendor assessment tool and a trial simulation tool. Information on the resources as well as an area for comment can be found
online. Feedback is due by April 24 at 11:59 pm ET and will help shape the final materials which will be available this summer.
2020 ICD-10-PCS Available on CMS Website
The 2020 ICD-10 Procedure Coding System (ICD-10-PCS) files contain information on the ICD-10-PCS updates for fiscal year 2020. The 2020 ICD-10-PCS codes are meant for discharges occurring from October 1, 2019 through September 30, 2020. PDF files are available on the Centers for Medicare and Medicaid
InfoEd Global Shares Library of COVID-19 Funding Opportunities
Additional COVID-19 Resources
Additional information on international regulatory news and resources is available on the Alliance for Regenerative Medicine
Best Handwashing Techniques Explained
Keeping one’s hands clean is one of the most important steps to prevent sickness and spreading contaminants to others. The CDC developed guidance for effective handwashing and use of hand sanitizer based on data from several studies. Visit the CDC
web page and read about the science behind the different handwashing recommendations used globally. There is also a video, “How to Handwash?” and “How to Handrub? With Alcohol-Based Formulation” produced by the World Health Organization.
Members of the AABB Center for Cellular Therapies' subsections meet regularly via teleconference to participate in interactive activities, including developing tools and reference materials, as well as discuss CT topics. For more information visit
The Latest on CT Subsection Activities
The AABB Center for Cellular Therapies encourages members to enroll in subsections to enrich their professional experience. Materials presented on the monthly calls can be found on the
CCT projects’ website.
All subsections (CT-CET, QRM, CB, APG and SLS) discussed the impact and challenges of COVID-19 on their practices and operations.
The Asia Pacific Group (APG) hosted Patrick Hanley, PhD, who spoke on “Overcoming Challenges in the Cellular Therapy Laboratory.” Members also discussed their survey responses on how to optimize their experience in the APG. Residents of the Asia Pacific region who are interested in being a part of AABB may find information on AABB
subsection enrollment online.