Just the FACTs, 2019 Volume 6
I recently arrived back from Melbourne and the International Society for Cell & Gene Therapy annual meeting. I would like to give a special thank you and recognition to Ngaire Elwood, FACT vice-president, one of the main organizers of this meeting of one of our two parent organizations. Patrick Hanley, PhD, chair of our Education Committee, chaired sessions and presented, as did many other FACT patrons! The breadth and depth of cell-based therapies is truly astonishing, and FACT is actively participating to assure the best possible products are delivered to our patients every day. I use the term “our” because FACT board members and inspectors are all active members of transplant, cord blood banking, and immune effector cell programs.
We had successful FACT training workshops and another excellent Quality Boot Camp in the days leading up to the meeting. For those who were unable to attend the annual meeting, the ISCT Regional Meeting in North America will be held in Madison, Wisconsin from September 12th – 15th. Join us for the FACT Cellular Therapy Inspection and Accreditation Workshop on Thursday, September 12.
Articles in this issue include:
- FACT Events at ISCT Were a Success!
- Help FACT Design Education Just for YOU
- Comments Requested About Needed Standards for Regenerative Medicine
- Webinar Series on Implementing Commercial CAR T-cell Products
- The Standards Coordinating Body Gives You a Voice
- New CARTOX App Assists Grading and Management of IEC Toxicities
- ASFA Releases Updated Practice Guidelines for Therapeutic Apheresis
- June is Cancer Immunotherapy Awareness month
- FACT Educational Opportunities
Sincerely,
Dennis Gastineau, MD
FACT President
FACT Events at ISCT Were a Success!
Chrissy Gordon: A Patient Perspective
FACT was honored to have its Cellular Therapy and Cord Blood Inspection and Accreditation Workshop in Melbourne, Australia opened by Ms. Chrissy Gordon, a recipient of an unrelated bone marrow transplant in 2000. Chrissy’s inspirational story of diagnosis, treatment, transplant, challenges, and successes over the past twenty years highlighted the importance of continuing to pursue improvements in the quality of patient care and advancing the promising developments in the field of cellular therapy. Diagnosed with leukemia in 1999 at the age of 28, Chrissy was treated in Melbourne, but her matched donor was found half way around the globe in Omaha, Nebraska where the donation was made at the University of Nebraska Medical Center. We first met Chrissy when she and her husband Dave visited Omaha to celebrate the 70th birthday of her marrow donor, Mr. Ken Vice of Lincoln, Nebraska.
Bone marrow transplant recipient Chrissy Gordon and her husband Dave shared her transplant story during the FACT Accreditation Workshop.
ISCT-FACT Quality Boot Camp 2019
The 2019 ISCT Annual Meeting in Melbourne kicked off during the Pre-Conference Day with a packed house at the ISCT – FACT Quality Boot Camp on Wednesday, May 29, 2019. Patrick Hanley, PhD presented timely information on incorporating immune effector cell activities into existing quality management programs. Guy Klamer, PhD discussed the importance of document control and common issues faced by cord blood banks. Participants were actively engaged during an interactive session with Phillip Johnson on risk management, and were presented with several case scenarios and asked to assess the risk. With validations being an important and challenging component of every cellular therapy facility, Robyn Rodwell, PhD and Annette Tricket, PhD provided details on the validation process and examples of their organizations’ results. FACT’s Quality Manager, Heather Conway, addressed the important subject of auditing, focusing on the minimum requirements of an audit report and the “Five Whys.” The day concluded with a hands-on exercise using the ISBT 128 Audit Tool for Cellular Therapy. The day was a great success and we look forward to future quality boot camps discussing core quality topics valuable to your organization. If you have ideas for future quality sessions, email your ideas to FACT@UNMC.edu.
Quality Boot Camp participants work together on an activity applying quality principles to real-world scenarios.
Help FACT Design Education Just for YOU
We would like to hear from YOU! FACT wants to provide the education that you need. Please help us develop education that is tailored to fit your needs by filling out this short 3-question survey.
Complete the Education Suggestions survey.
Comments Requested About Needed Standards for Regenerative Medicine
The Standards Coordinating Body (SCB) and Nexight are requesting comments on a report titled, Community Perspectives: Needed Standards in Regenerative Medicine. The report outlines prioritized areas in cell therapy, gene therapy, and tissue engineering. Comments are due by June 19, 2019.
Per the SCB website, “One of SCB’s goals since its inception has been to strengthen the identification and prioritization process for advancing regenerative medicine standards through greater community engagement, a goal we outlined in Realizing the Promise of Regenerative Medicine Therapies: Strengthening the Standards Development Process. The methodology we used to create the report on needed standards allowed us to implement many of these process improvements.”
This report was drafted under contract for the United States Food and Drug Administration (FDA) and has the potential to be influential in the direction the cellular therapy field takes toward standardization. Your comments as experienced professionals are critical to charting the path.
Send comments to Sarah Lichtner at slichtner@nexightgroup.com by June 19.
Webinar Series on Implementing Commercial CAR T-cell Products
The American Society of Gene & Cell Therapy (ASGCT) has partnered with FACT and the American Society for Transplantation and Cellular Therapy (ASTCT) to bring you the two-part webinar series, “Centers’ Experience with Implementing Commercial CAR T-cell Products.”
The first webinar will be on Thursday, July 25, 2019 at 11:00 am ET. Part one includes information from two centers regarding their journey to successful implementation of commercialized CAR T-cell products. Clinicians will address the challenges faced during the site on-boarding process, where they are in this process, commonly faced impediments to timely delivery of therapy to patients, and suggestions for centers initiating implementation of commercial CAR T-cell products. Topics in this webinar will include required training, maintenance of chain of custody, and mechanisms for reporting outcomes data. FACT accreditation will also be covered.
Part two will take place in September of 2019 and will include the perspective of a hospital administrator in regard to the following:
- Agreements with Manufacturers
- Reimbursement
- FACT accreditation
View meeting details and register
The Standards Coordinating Body Gives Cellular Therapy Programs and Cord Blood Banks a Voice
The Standards Coordinating Body (SCB) was formed as an answer to the Advancing Standards in Regenerative Medicine Act, and holds contracts with the Food and Drug Administration (FDA) to advise the agency on needed standards in regenerative medicine. It formally supports many standards development efforts in conjunction with other organizations. FACT is an engaged member organization and participates in SCB projects and provides regular feedback as requested by the SCB.
Did you know that the transplant and cord blood banking communities are critical to the SCB’s efforts?
Although “regenerative medicine” is a very broad term with connotations related to “new” or “novel” therapies, many of the SCB’s projects relate to issues in which FACT-accredited organizations have decades of experience. Accredited organizations are often the facilities that collect cellular starting material, perform contract manufacturing or supportive processing and cryopreservation procedures, or clinically support specialties not yet experienced in cell and gene therapies. Even those organizations that are not directly involved have had to evaluate, resolve, and manage solutions to the very challenges newer players in cell and gene therapies are struggling with now.
Please share your experiences and expertise with the Standards Coordinating Body!
Your input will assist the SCB with its mission, inform commercial manufacturers and other specialties on inherent differences in cell and gene therapies, and promote solutions that harmonize with existing Standards and regulations. The SCB’s outputs could directly impact your daily work and, more importantly, help expedite availability of therapies to patients sooner rather than later. Express your interest in participating in SCB projects to Dawn Henke, SCB Senior Technical Program Manager, at dhenke@regenmedscb.org.
The following are notable SCB initiatives that are likely of interest to many FACT-accredited organizations. There are many more that may be intriguing based on professional interests. For a full listing of projects and reports, visit the SCB’s website at https://www.standardscoordinatingbody.org.
- Cell Collection Standards for Cell Therapies: FACT, SCB, commercial manufacturers, and many other organizations have heard, loud and clear, that apheresis facilities are struggling with the increasing volume of effort required of variable collection and audit requirements. This project group is currently formulating surveys to distribute to industry and apheresis facilities to learn what needs could be reconciled and then standardized.
- Cryopreservation of Cells (PDA-led project): Many commercial manufacturers have sought best practices related to cryopreservation and have had to adjust those practices to fit the needs of their particular cellular therapy products. This project group is drafting a framework on which to establish cryopreservation procedures in the hope to standardize requirements when possible.
- Transportation Requirements of Cells for Therapeutic Use: Shipping and transportation have a direct and significant influence on chain of custody and chain of identify of cellular therapy products, and is of great interest to commercial manufacturers. This project group is working to identify gaps in current Standards. Due to the emphasis on shipping and transportation requirements in FACT Standards, efforts to remain harmonization with these existing Standards will prevent conflicting requirements.
- Community Perspectives: Needed Standards in Regenerative Medicine Report: This report, based on input from many stakeholders, outlines needed standards that could have the greatest benefit to the safety and quality of regenerative medicine products. The draft is currently available, and will be updated as needed based on comments the SCB received during its recent public comment period in May 2019.
New CARTOX App Assists Grading and Management of IEC Toxicities
By Sherry Adkins, RN, MSN, ANP-C and Kara Wacker, MBA, RAC
The CARTOX Mobile App for iPhone and Android, created by MD Anderson Cancer Center, is now available to assist clinicians with accurate and timely grading and treatment of toxicities associated with immune effector cellular therapy (IEC). The app is intended for physicians, nurse practitioners, physician assistants, nurses, and pharmacists who treat patients receiving IECs.
Sherry Adkins, RN, MSN, ANP-C, Advanced Practice Provider Supervisor, Lymphoma Research at the MD Anderson Cancer Center developed the idea of an app to allow for safer and more efficient care of the rapidly expanding population of patients receiving IECs. “We recognize that the app has helped our group improve the safety and efficiency of care for this group of patients,” explains Adkins. “We hope it will help other clinicians, particularly those who are just beginning to administer this therapy.”
Ms. Adkins led the implementation strategy with coding assistance from Darren Skeete and his team from the center’s Informational Technology (IT) department. Members of the MD Anderson CARTOX Committee, including Drs. Sattva Neelapu and Elizabeth Shpall, reviewed and tested the app. The process started in the Fall of 2017, and MD Anderson clinicians had access by August of 2018.
The app is based on published guidelines, and will continue to be updated as science evolves to reflect the most current practice guidelines. The initial version of toxicity grading was based on the MD Anderson’s CARTOX program guidelines, but has since been updated to reflect the ASTCT (formerly known as ASBMT) consensus grading. (Biol Blood Marrow Transplant. 2019 Apr;25(4):625-638. doi: 10.1016/j.bbmt.2018.12.758. Epub 2018 Dec 25. Review.)
Toxicity management is based on the CARTOX program’s guidelines, which were developed by a multidisciplinary, multi-institutional group of clinicians (Nat Rev Clin Oncol. 2018 Jan;15(1):47-62. doi: 10.1038/nrclinonc.2017.148. Epub 2017 Sep 19. Review). Toxicity management guidelines are currently being updated and the mobile APP will be updated to reflect those changes in the near future.
To download the app, search for CARTOX in the Apple Store or Google Play.
ASFA Releases Updated Practice Guidelines for Therapeutic Apheresis
The Special Issue Writing Committee of the American Society for Apheresis (ASFA) updated indications for evidence-based use of therapeutic apheresis (TA). The Guidelines on the Use of Therapeutic Apheresis in Clinical Practice – Evidence-Based Approach from the Writing Committee of the American Society for Apheresis: The Eighth Special Issue contains fact sheets summarizing the evidence for the use of TA for 84 relevant diseases and medical conditions.
Fact sheets related to blood and marrow transplantation included in these guidelines include graft-versus-host disease (GVHD), ABO-incompatible hematopoietic stem cell transplantation, and HLA-desensitized hematopoietic stem cell transplantation. Given the wide range of TA performed by apheresis facilities, review of all fact sheets is worthwhile.
Padmanabhan, A, Connelly‐Smith, L, Aqui, N, et al. Guidelines on the Use of Therapeutic Apheresis in Clinical Practice – Evidence‐Based Approach from the Writing Committee of the American Society for Apheresis: The Eighth Special Issue. J Clin Apher. 2019; 34: 171– 354. https://doi.org/10.1002/jca.21705
June is Cancer Immunotherapy Awareness Month
This month we recognize and celebrate June as the 7th Annual Cancer Immunotherapy Month™ hosted by the Cancer Research Institute. Patients, caregivers, advocacy organizations, health care professionals and industry partners, unite to raise awareness of the lifesaving potential of cancer immunotherapies. Every day, so many work to transform T-cell therapy including researchers who have helped pioneer this research and continue to make seminal advances in this growing field. This month is not only termed to promote general awareness, but also to provide educational growth and professional development opportunities for clinicians, researchers, and patients.
Below are some of the benefits of cancer immunotherapy: (Benefits of Immunotherapy (n.d.). Retrieved from: http://www.cancerresearch.org/immunotherapy/why-immunotherapy).
Cancer immunotherapy can work on many different types of cancer
- Immunotherapy enables the immune system to recognize and target cancer cells, making it a universal answer to cancer.
- The list of cancers that are currently treated using immunotherapy is extensive. See the full list of Immunotherapies by cancer type.
- Immunotherapy has been an effective treatment for patients with certain types of cancer that have been resistant to chemotherapy and radiation treatment
Cancer immunotherapy offers the possibility for long-term cancer remission
- Immunotherapy can “train” the immune system to remember cancer cells. This “immunomemory” may result in longer-lasting remissions.
- Clinical studies on long-term overall survival have shown that the beneficial responses to cancer immunotherapy treatment are durable—that is, they can be maintained even after treatment is completed.
Cancer immunotherapy may not cause the same side effects as chemotherapy and radiation
- Cancer immunotherapy is focused on the immune system and is often more targeted than conventional cancer treatments such as chemotherapy or radiation.
- Both chemotherapy and radiation damage healthy cells, leading to common side effects such as hair loss and nausea/vomiting. These side effects may be less likely with immunotherapy.
- Side effects of cancer immunotherapy will vary depending on which type of immunotherapy is used. They are usually related to stimulation of the immune system and can range from minor symptoms of inflammation (e.g., fever) to major conditions similar to autoimmune disorders.
To learn more about Immunotherapy visit: http://www.cancerresearch.org/immunotherapy/what-is-immunotherapy
View Cancer Immunotherapy Month Events in June 2019
Education Events
Using an Electronic Platform for Accreditation Preparation and Continuous Readiness Webinar
Wednesday, June 26, 11:00 am ET
Webinar
This webinar will be on June 26, 2019 at 11:00 am ET and is FREE OF CHARGE. Trisha Deason, MHA, MSN, RN, NE-BC, Eryn Draganski, AGCNS-BC, OCN, and Jay Maningo-Salinas, PhD, RN, NE-BC, CPHQ, FACHE, from Mayo Clinic Arizona, have graciously agreed to share their tool designed in Microsoft OneNote to be used for continuous readiness and compliance with FACT requirements.
Collecting supporting evidence for accreditation standards can be a daunting task and include a tremendous amount of resources (e.g., paper, binders, charts). Workflows may be inefficient if duplication of efforts occurs across personnel and departments. Use of an electronic source provides an updated mechanism to gather, retain, and share evidence, as well as an organized approach to present all supporting documents to on-site surveyors. Presenters will provide step-by-step instructions on how to build a OneNote FACT e-binder and organize evidence to be used for continuous readiness and/or as a platform to deliver evidence to the FACT survey team.
View meeting details and register online
ASGCT-ASTCT-FACT Webinar: Centers’ Experience with Implementing Commercial CAR T-cell Products – Part 1
Thursday, July 25, 11:00 am ET
Webinar
The first webinar in this two-part webinar series will be on Thursday, July 25, 2019 at 11:00 am ET. Part one includes information from two centers regarding their journey to successful implementation of commercialized CAR T-cell products. Clinicians will address the challenges faced during the site on-boarding process, where they are in the process, commonly faced impediments to timely delivery of therapy to patients, and suggestions for centers initiating implementation of commercial CAR T-cell products. Topics in this webinar will include required training, maintenance of chain of custody, and mechanisms for reporting outcomes data. FACT accreditation will also be covered.
This is part one of two in the Centers’ Experience with Implementing Commercial CAR T-cell Products series. Part two will take place in September of 2019 and will include the perspective of a hospital administrator in regard to the following:
- Agreements with Manufacturers
- Reimbursement
- FACT accreditation
View meeting details and register
FACT-SBTMO Cell Therapy Accreditation Workshop
July 31, 2019
Brasília, Brazil
This training workshop is designed to explain the requirements of the FACT-SBTMO joint cell therapy accreditation program in Brazil. FACT and SBTMO representatives will be in attendance to:
• Provide an overview of the FACT Standards and accreditation process
• Explain how to integrate quality management into clinical, collection, and cell processing programs
• Examine how to prepare for a successful inspection
• Summarize how controlling documentation can improve your program
• Review commonly cited Standards
• Outline the inspector orientation process
• Answer questions
This workshop is from 8:00 to 13:15 on Wednesday, July 31, 2019.
View meeting details and register
WMDA-NetCord & FACT Cord Blood Day at 2019 Cord Blood Connect International Congress
September 12, 2019
Miami, FL
The World Marrow Donor Association (WMDA) and FACT are teaming up to host the pre-conference WMDA-Netcord & FACT Cord Blood Day, a workshop that incorporates sessions on trends in cord blood banking and harmonization of banking procedures, a Quality Boot Camp track, and a FACT Inspector Training Track. FACT accreditation serves as an important mechanism to maintain across-the-board uniform processes that are effective and assist with ensuring efficient bank operations.
Topics will include:
- Global perspectives of the regulation of cord blood banking,
- WMDA serious adverse events and reactions,
- Analysis of CD34+ and CD45+ cells in thawed cord blood samples,
- Analysis of cellular viability in thawed cord blood,
- Significant changes to the NetCord-FACT Seventh Edition Cord Blood Standards, and
- Essential Quality Management Principles
The afternoon offers two tracks:
- WMDA-FACT Quality Boot Camp: Sessions will examine cord blood unit selection strategies and core quality management concepts including risk assessment, validation, and auditing. Come prepared with your quality-related questions to ask the quality experts facilitating the session.
- FACT Inspector Training: This track provides training to new FACT cord blood inspectors on performing an inspection from start to finish. Sessions are accompanied by exercises and group discussions to practically apply lecture concepts to real-world experiences. Attendees training to be an inspector may attend the entire Cord Blood Day FREE in addition to free access to all other on-demand and in-person FACT educational events. Inspector trainees are required to attend the FACT Inspector Training track.
This is an excellent opportunity to network with the cord blood community and learn from experts in cord blood banking. We encourage banks to send multiple personnel to be able to attend both the Quality Boot Camp and nominate a member of your team to train to become a FACT inspector! Register before August 31st to receive the special advanced rate!
FACT Cellular Therapy Inspection and Accreditation Workshop at the ISCT North America 2019 Regional Meeting
September 12, 2019
Madison, WI
This training workshop is designed to explain the requirements for FACT accreditation of cellular therapy programs. FACT representatives will be in attendance to clarify the intent of the FACT-JACIE Standards, provide inspectors tips for conducting inspections, and assist programs in organizing and preparing for the accreditation process.
Two tracks are available for this workshop. The Inspector Track will provide FACT inspectors guidance on how to assess compliance with the Standards. The Applicant Track will give programs and facilities insight into how to comply with the Standards and demonstrate compliance.
View meeting details and register
Significant Changes to the Seventh Edition NetCord-FACT Cord Blood Standards
October 23, 2019
Webinar
This presentation will be on Wednesday, October 23, 2019, at 11:00 am ET. Paul Eldridge, PhD, Director of Cell GMP Facility at UNC Bone Marrow Transplant and Cellular Therapy Program, will describe significant changes to the Seventh Edition NetCord-FACT Cord Blood Standards, and provide examples for effective implementation.
View meeting details and register
