Volume 29, Issue 4
Table of Contents:
Perspectives: On Grant Writing
By Michael Verneris, MD
Over the past few weeks, I have been helping write the PTCTC grant resubmission to be a consortium site for the BMT CTN. There is no candy-coating it: This was an intense experience. And like many of the cooking shows on TV, it all came together in the end with only moments to spare. One small benefit of this experience was that it served as the inspiration for this entry. So here goes.…
I sometimes like to use quotes to get my point across, since they are often succinct and poignant. Thus, I went to Google to find a meaningful quote about writing an NIH grant. Not surprisingly, no such quote exists. I’m not sure what that says about grant writing, but, for me, grant writing is a foundational aspect of what I do—“Without money, there is no mission.” Grant writing is about more than just money. It’s about creating time to reflect on and vet my ideas, survey the literature, and consider the potential impact of completing planned studies. There are, of course, more practical aspects to consider as well, such as which techniques to use to accomplish the scientific goals, how the data will be interpreted, the risks, and alternative approaches. In short, grant writing forces me to slow down and consider the ideas and the plans meticulously. It is a mini journey of self-reflection and discovery.
The PTCTC resubmission compelled me to confront the strengths and weaknesses of our consortium, leading to a deeper understanding of the areas where improvement is needed. The PTCTC grant is all about the structure and inner workings of the PTCTC—something that I should have known fundamentally more about in any event. Answering some of the grant prompts forced me to evaluate how we are truly doing as a consortium. In particular, the PTCTC grant’s focus on mentorship made me realize that, while we are doing a decent job, we could probably be doing better. Likewise, the grant requirements on diversity, both with regard to providers and patients, made me recognize that, once again, we are doing pretty well, but could be doing more. These realizations would not have occurred without the scrutiny and introspection demanded by the grant-writing process itself.
Grant writing is complex and intense, and funding decisions are highly competitive. Perhaps not surprisingly, there is a large body of literature around grant writing, ranging from more obvious topics, such as how to do it better and how to teach trainees about grant writing, to more interesting assessments about time spent grant writing and the associated personal costs. For instance, the energy industry in Austria provides the following perspective on grant writing, i.e., preparing a new proposal takes about 50 working days; more than 90% of researchers perceive that they currently spend too much time preparing proposals; only 10% believe that the current funding approach had a positive effect on their research quality; and researchers report having limited trust in the objectivity of the proposal review processes.
A paper looking at Australian medical research grants (the National Health and Medical Research Council) showed that spending more time preparing a proposal did not increase the chances of success for the lead researcher. These same investigators showed that almost all researchers agreed that preparing their proposals took top priority over other work (97%) and personal commitments (87%). Almost all researchers agreed that they became stressed by the workload (93%) and restricted their holidays during the grant-writing season (88%). While there certainly is value in requiring investigators to provide a logical and compelling research plan, it has been argued that some investigators spend as much as a third of their professional time on grant-writing activities. Given the low paylines, this creates inherent inefficiencies. Thus, some have called for less time-intensive models for grant submissions, including a “fail quickly” model, which might free up time for other pursuits.
In conclusion, grant writing is a multi-faceted experience that extends beyond the pursuit of funding. It is a process that demands being critical of your ideas, finding weaknesses, and addressing them. It requires continuous learning, planning, and an intricate balance between efficiency and quality. The chaos and stress associated with grant writing are not mere obstacles; they can be catalysts for better science. Circling back to our PTCTC submission, it too was chaotic and stressful. I believe that my partners—Leslie Kean, MD, PhD, and Heather Stefanski, MD, PhD—and I pulled together a nice proposal, highlighting the PTCTC. We frankly could not have done it without the support of the NMDP/Be The Match staff who helped at every twist and turn. We all shared the work and had some fun along the way. While I’m very hopeful that it is funded, at the very least this exercise allowed me to reflect on the broader implications of the grant-writing process and cooperative group clinical research. The chaos and stress of grant writing are not unique to our situation; they are inherent in the process. However, they also bring moments of clarity and self-discovery.
Acute Leukemia Working Committee
- Christopher Hourigan, NHLBI-NIH, Bethesda, MD
- Filippo Milano, Fred Hutchinson Cancer Center, Seattle, WA
- Nelli Bejanyan, Moffitt Cancer Center, Tampa, FL
- Partow Kebriaei, MD Anderson Cancer Center, Houston, TX
- Mariam Nawas, University of Chicago, Chicago, IL
- Kristin Page, CIBMTR MCW, Milwaukee, WI
- Mei-Jie Zhang, CIBMTR MCW, Milwaukee, WI
- Dylan Lui, CIBMTR MCW, Milwaukee, WI
Acute leukemia remains the most common indication for allogeneic HCT. This is partially due to increasing numbers of older adults receiving HCT, along with improved access due to expanded donor availability. However, post-HCT relapse remains a major barrier. The increasing use of molecular profiling is helping to inform therapy and risk prognostication. CIBMTR has supported this effort by extending data collection to include questions regarding cytogenetics and molecular markers to allow for more informative and relevant transplant registry studies. In July 2023, CIBMTR started collecting the International System for Human Cytogenomic Nomenclature “strings.” This allows for more complete and standardized cytogenetic data to be captured. Additional planned changes specific to acute leukemia include updating the classification to reflect the World Health Organization 2022 guidelines and expanding the collection of MRD test details, including multiparametric flow cytometry, cytogenetics, fluorescence in situ hybridization, next-generation sequencing, and polymerase chain reaction. We will also start collecting data on mutations and molecular abnormalities associated with Philadelphia chromosome-like ALL. The emergence of post-HCT therapies to prevent relapse is another setting for updating data collection. We expect many debates and analyses proposed on these and other topics in the Acute Leukemia Working Committee. HCT indications, approaches, and comparisons with other therapies are all valid topics for study.
The success of the Acute Leukemia Working Committee predominantly derives from a dedicated team with collaborative input. Earlier this year, Nelli Bejanyan, MD, joined as co-chair taking over for Mark Litzow, MD, who completed his term. Filippo Milano, MD, PhD, has also joined as co-chair as part of the recent Working Committee restructuring. Committee leadership works closely with committee members, data managers, and transplant groups in both the US and abroad. Leadership develops and promotes the scientific agenda (established with input from committee members); determines priorities in the selection of high-impact studies; and ensures timely progression in protocol development, statistical analyses, manuscript development, and publications. The committee strives to improve quality and efficiency and is guided by the 3 principles established by CIBMTR’s Advisory Committee: Publish peer-reviewed papers of high scientific impact, complete studies within a reasonable time frame, and ensure inclusiveness and fairness within the study process. We are also committed to mentorship and are excited to work with our new Working Committee Leadership and Training Program Chair, Mariam Nawas, MD.
Our committee received more than 50 proposals for the 2023 Tandem Meetings of ASTCT and CIBMTR. These numbers reflect a rapidly evolving field and a high level of interest in the rich data on acute leukemia within CIBMTR’s Research Database. As a committee, we are eager to review and discuss new proposals from centers worldwide. Notably, we have restructured the working committees in response to changing trends in proposal submissions; the Acute Leukemia Working Committee will now receive proposals that include adoptive cellular therapies or compare graft sources as long as they focus solely or primarily on acute leukemias.
Number of Cases in the CIBMTR Research Database
|AML autologous HCT
|AML allogeneic HCT
|ALL autologous HCT
|ALL allogeneic HCT
View planned, in-progress, and completed studies and publications on the Acute Leukemia Working Committee webpage.
Lymphoma Working Committee
- Alex Herrera, City of Hope, Duarte, CA
- Craig Sauter, Cleveland Clinic Foundation, Cleveland, OH
- Mazyar Shadman, Fred Hutchinson Cancer Center, Seattle, WA
- Mehdi Hamadani, CIBMTR MCW, Milwaukee, WI
- Kwang Woo Ahn, CIBMTR MCW, Milwaukee, WI
- Manmeet Kaur, CIBMTR MCW, Milwaukee, WI
The Lymphoma Working Committee, one of the first established within CIBMTR, focuses on HCT and cellular therapy for both Hodgkin and non-Hodgkin lymphomas and has conducted numerous studies addressing a wide range of issues in the field for these diseases. Since the last CIBMTR Newsletter update from the Lymphoma Working Committee in May of 2020, the committee has published 20 peer-reviewed publications. Included in these publications were 4 manuscripts published in Blood or Blood Advances, 6 manuscripts in the Journal of Transplantation and Cellular Therapy (formerly Biology of Blood and Marrow Transplantation), and 2 manuscripts in JAMA Oncology. Additionally, the Working Committee has participated in the creation of guidelines for the role of HCT and cellular therapy in mantle cell lymphoma and diffuse large B-cell lymphoma, since the last newsletter update.
In the contemporary era, the committee’s relevance has only increased given the introduction of 4 commercially available CAR T products targeting CD19 non-Hodgkin lymphomas including large B-cell lymphoma, follicular lymphoma, and mantle cell lymphoma. Key investigational pursuits concerning the juxtaposition of cellular therapy and HCT for various non-Hodgkin lymphomas continue through the Lymphoma Working Committee. These studies carry gravity considering recent FDA-approved label expansions for both axicabtagene ciloleucel and lisocabtagene maraleucel in the second line for large B-cell lymphoma against the previous standard of salvage chemotherapy and HCT. Limitations of the randomized Phase III studies garnering the label expansion, paired with access complexities of CAR T cells for many patients, have served as material for contemporarily relevant committee investigation culled from real-world CIBMTR data.
Most recently in 2018, updated versions of lymphoma disease-specific forms were implemented to capture molecular testing information, such as identifying the presence of chromosomal translocations, and other molecular subtyping information, such as germinal center versus non-germinal center “cell of origin” in the case of diffuse large B-cell lymphoma. This additional information will allow the Lymphoma Working Committee to conduct more up-to-date and clinically relevant analyses in the future, across both HCT and cellular therapy.
Thanks to the considerable number of lymphoma patients treated with HCT and cellular therapy whose data are in CIBMTR’s Research Database, the Lymphoma Working Committee can provide information with the capacity to influence clinical practice in various transplant-related matters.
The Lymphoma Working Committee has been extremely active over the last few years, in large part due to the extensive data available in CIBMTR’s Research Database. The number of transplants for lymphoma added to the research database from 2000 through 2022 are listed in the table below. During the annual committee meeting at the 2022 Tandem Meetings, 6 new proposals were presented, and 1 was approved to be further developed and analyzed. More than 150 people attended the Lymphoma Working Committee meeting, and the great involvement from the transplant community helps the committee to produce timely and substantive research.
Number of Cases Added to CIBMTR Research Database, 2000-2022
| Allogeneic HCT
| Autologous HCT
| Allogeneic HCT
| Autologous HCT
View planned, in-progress, and completed studies and publications on the Lymphoma Working Committee webpage.
2024 Tandem Meetings
By Alicia Halfmann and Maira Brey
ASTCT and CIBMTR are pleased to announce that registration is open for the 2024 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR. We look forward to connecting with our international community of healthcare professionals at the Henry B. González Convention Center in San Antonio, Texas, February 21-24, 2024—and during the pre-conference events on February 20 and the post-conference events on February 25.
The 2024 Tandem Meetings will feature in-person and virtual programming, so everyone can experience the premier event in the evolving HCT and cellular therapy field.
What to Expect: Cutting Edge Science, International Experts, and Collaborative Community
Throughout the 2024 Tandem Meetings, leading experts in the field of transplantation and cellular therapy, from around the world, will present the latest developments and key insights to help healthcare professionals and researchers save and improve the lives of patients with blood-related disorders. See the online program.
The 2024 Tandem Meetings will also offer a variety of opportunities for attendees to network with an international community of the field’s healthcare professionals. See the list of events and receptions.
Please visit the 2024 Tandem Meetings website for all current details.
Scientific Program and Educational Tracks
The 2024 Scientific Organizing Chairs, Alan Hanash, MD, PhD, and Christopher Kanakry, MD, along with the scientific organizing committee and session chairs, have assembled an excellent program providing access to the latest scientific updates, new technologies, and innovative products in the HCT and cellular therapy field.
An outline of the Scientific Program Topics is listed below:
- Mucosal immunology and host/pathogen interactions impacting tissue damage
- How to select the best donor in an age of plenty
- Biology of relapse after transplant and cellular therapy
- Novel antigen discovery for transplant and cellular therapies
- Laboratory science in HCT and CAR T: What is the role of laboratory science in our organizations and in our meetings?
- Data partnerships and possibilities: Beyond the CIBMTR Working Committees
- Optimizing alternative donor platforms: Focus on GVHD prevention
- Advances in gene editing to cure human diseases
- Late effects and survivorship post BMT: Moving from description to prevention and intervention
- Harnessing the microbiome
- Clonal hematopoiesis and immune function
- Point-of-care models of cellular therapy: Improving accessibility
- Evolving landscape of treatment for B-cell ALL
- Statistical issues in the design and analysis of transplant and cell therapy studies
Confirmed Educational Tracks include administrative director, advanced practice providers, BMT CTN coordinators, clinical research professionals / data management, infectious diseases, IT, and informatics, nursing, pediatrics, and pharmacy.
Please also join us for:
- Mortimer M. Bortin lecture
- Oral abstract sessions
- Late breaking abstracts session
- ASTCT spotlight sessions
- And industry-supported Satellite Symposia, Product & Innovation Theaters, and exhibitors
- E. Donnall Thomas lecture
- Best abstracts session
- CIBMTR Working Committee meetings
- Meet the professor sessions
We look forward to seeing you at the 2024 Tandem Meetings in San Antonio, Texas!
Follow ASTCT and CIBMTR on social media, as well as the official hashtag, #Tandem24, for updates.
Working Committee Training and Leadership Program
The Working Committee Training and Leadership Program was first announced during the 2023 Tandem Meetings, and applications were accepted through mid-April 2023. We had an overwhelming response—receiving applications from 77 Early Career Investigators representing more than 65 distinct transplant and cellular therapy centers in 10 countries. The 8 participants selected all share a high level of dedication, achievement, and enthusiasm for clinical research. We would like to warmly welcome them to the program and are excited to be a part of their career development. Join us as we further highlight the participants in this edition of the newsletter and future editions.
Michelle Schoettler, MD: Morbidity, Recovery, and Survivorship Working Committee
I am a pediatric HCT faculty member at Emory University/Children’s Healthcare of Atlanta. As a clinical and translational scientist, I study early endothelial disorders after HCT, with a particular focus on transplant-associated thrombotic microangiopathy and sinusoidal obstructive syndrome. I am very excited about this program and feel fortunate to have this unique opportunity. I look forward to learning more about the process of completing a CIBMTR project, from proposal selection to publishing the manuscript. I’ve already gained insight into statistical approaches and study design by joining the weekly statistical meetings and working with the fantastic statisticians within our Working Committee.
“It has been wonderful having Dr. Schoettler join the Morbidity, Recovery, and Survivorship Working Committee. She has already contributed thoughtful and valuable perspectives and feedback on ongoing studies. She is also helping us to complete a long-standing study focused on patients who have received both solid organ transplants and HCT.”—Rachel Phelan, MD, MPH, Scientific Director of the Morbidity, Recovery, and Survivorship Working Committee
Hany Elmariah, MD, MS: Chronic Leukemia Working Committee
I am an assistant member in the Department of Blood and Marrow Transplant and Cellular Immunotherapy at the Moffitt Cancer Center. My clinical interests focus on allogeneic HCT and cellular therapy for myeloid malignancies. My research is primarily focused on novel strategies to improve haploidentical and mismatched donor therapies for patients with myeloid malignancies. CIBMTR’s Working Committee Training and Leadership Program has already provided many opportunities to meet leaders in the field and learn new strategies to advance research. As a member of the program, I look forward to this ideal opportunity to gain knowledge regarding CIBMTR operations, optimization of proposals, strengths of the data available, statistical methods, and to make meaningful contributions to CIBMTR’s mission.
“Dr. Elmariah has quickly become an integral part of the Chronic Leukemia Working Committee team. He is working on several ongoing studies and is developing several study concepts for consideration at the Tandem Meetings. He is very engaged in discussions and provides very helpful input.”—Wael Saber, MD, MS, Scientific Director of the Chronic Leukemia Working Committee
Brian Ball, MD: Non-Malignant Working Committee
I am an assistant professor at City of Hope. My overall career goal as a clinical and translational investigator is to develop more tolerable and effective therapies to improve outcomes for patients with myeloid malignancies and bone marrow failure syndromes. I am particularly interested in understanding the molecular determinants of post-transplant outcomes among patients with MDS and bone marrow failure syndromes receiving allogeneic HCT. After developing a greater understanding on the impact of inherited and acquired molecular abnormalities on post-transplant outcomes, I aim to apply this information towards developing more personalized approaches to transplant regimens. I am thrilled to be joining the Working Committee Training and Leadership Program and Non-Malignant Hematology Working Group. I am looking forward to working with leaders in the field and developing skills in accessing and analyzing CIBMTR data.
“Dr. Ball has been a great addition to the Non-Malignant Working Committee. We are looking forward to his expertise and insight on our upcoming aplastic anemia studies.”—Larisa Broglie, MD, MS, Scientific Director of the Non-Malignant Working Committee.
CIBMTR CRO Services: PRESERVE 1 Now Enrolling
NMDP/Be The Match and CIBMTR are excited to announce the opening to accrual of the PRESERVE 1 study, “A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies,” NCT05589896! This new study is the result of a collaboration between NMDP/Be The Match and Ossium Health Inc., an innovative bioengineering company specializing in the development of stem cell therapies1. The CIBMTR CRO Services team has been contracted in a full-service model capacity to manage this study.
Recent studies utilizing post-transplant cyclophosphamide as GVHD prophylaxis have demonstrated favorable outcomes following HLA-MMUD2,3. Ossium Health Inc. has developed an innovative process to recover bone marrow from deceased organ donor vertebral bodies and to cryopreserve the product to enable off-the-shelf shipment to an identified recipient. The goal is to provide an on-demand, adequate dose of bone marrow hematopoietic cells, which would enable the rapid treatment of patients, and potentially improve outcomes and expand access to HCT.
Over the next year, this study will enroll 12 participants, with acute leukemias, who do not have a suitable living donor but are HLA partially or fully matched to an available Ossium HPC, Marrow product. The donor is identified through the NMDP/Be The Match customer-ready product team by reviewing Ossium’s inventory of cryopreserved Ossium HPC, Marrow products in the NMDP MatchSource donor search and patient Case Management System. Participants can receive either MAC or RIC before infusion and will be followed for 1-year post-transplant. This study will primarily assess the safety and efficacy of the Ossium HPC, Marrow product.
We look forward to seeing the results of this first-in-human clinical trial.
1 Press release: https://apnews.com/press-release/marketersmedia/organ-transplants-7ff83f33efd023a4f3e37d8dc96a77ab
2 Shaw BE, Jimenez-Jimenez AM, Burns LJ, et al. National Marrow Donor Program-sponsored multicenter, Phase II trial of HLA-mismatched unrelated donor bone marrow transplantation using post-transplant cyclophosphamide. J Clin Oncol. 2021 Jun 20;39(18):1971-1982. doi: 10.1200/JCO.20.03502. Epub 2021 Apr 27. PMID: 33905264; PMCID: PMC8260905.
3 Shaw BE, Jimenez-Jimenez AM, Burns LJ, et al. Three-year outcomes in recipients of mismatched unrelated bone marrow donor transplants using post-transplantation cyclophosphamide: Follow-up from a National Marrow Donor Program-sponsored prospective clinical trial. Transplant Cell Ther. 2023 Mar;29(3):208.e1-208.e6. doi: 10.1016/j.jtct.2022.12.017. Epub 2022 Dec 27. PMID: 36584941; PMCID: PMC9992261.
A Tool to Enhance Donor Selection to Lower GVHD Risk
By Ray W. Sajulga Jr.
Personalized medicine requires leveraging a patient’s genetic makeup to make an informed decision that optimizes outcomes. Previous research identified the importance of the level of HLA-DP expression of a patient’s mismatched molecule in acute GVHD risk. When the donor’s HLA-DPB1 is mismatched against a high-expression patient HLA-DPB1, patients are at significantly higher risk of severe acute GVHD than when the donor’s HLA-DPB1 is mismatched against a low-expression patient molecule. To enhance donor selection, we collaborated with Effie Petersdorf, MD, from the Fred Hutchinson Cancer Center, to design a tool for automated assessment of low- and high-expression HLA-DPB1 mismatches.
In our study, we combined the existing T-Cell Epitope model with the new HLA-DP expression model to avoid high-risk mismatches from both models. To demonstrate the tool’s utility, we characterized the variation of 356,188 exon 2-exon 3 nucleotide sequences and 5,032 patients with an HLA-10/10-matched unrelated donor transplant with archived donor searches in the NMDP/Be The Match database.
Our results demonstrate that HLA-DPB1 exon 3 variation provides complete information regarding the level of expression of the allele. Exon 3 is most variable in Americans who identified as Asian (including Native Hawaiian or other Pacific Islander), Black (including African American). Furthermore, our results stress the importance of proactively accommodating searches for these non-White patients, who have more high-expression, high-risk alleles and fewer opportunities for expression-favorable (mis)matches. Our work shows the importance of typing both exon 2 (the antigen-recognition domain) and exon 3 to avoid high-risk HLA-DPB1 mismatches. By automating these complex and diverse models, we can strive towards personalizing transplants for all patients.
Study Contributors: Ray Sajulga, Yung-Tsi Bolon, Martin J. Maiers, Effie W. Petersdorf. View the study published in the Blood Advances journal on August 24, 2023.
Data Transformation Initiative
In the past year, DTI has worked to accelerate expansion of data capture by identifying ways that data can be collected more efficiently. CIBMTR continues to onboard and engage transplant centers and partners with DTI, and interest continues to grow. We now have more than 25 transplant centers and partners onboarded with DTI.
The team is working on additional functionality that allows data to be extracted more efficiently, utilizing tools that centers are already using. One of the main focuses of DTI has been data automation, with a focus on labs at diagnosis, neutrophil count recovery, and cytogenetics. We are continuously upgrading and improving our existing DTI systems, to find efficiencies in data collection and downstream data use for research purposes. Included in these upgrades are improvements to the CIBMTR Reporting App, such as temporal filtering and upgrading standards for data exchange.
As we move forward into the new year, DTI will continue to develop efficiencies for data collection, with the main goal of reducing the burden for our participating centers and their data managers. One of our next upgrades will allow for the capture of medication data via Fast Healthcare Interoperability Resources (FHIR) methodologies.
Adoptive Cellular Therapy and Gene Therapy Updates
Transition from CIDR to Adoptive Cellular Therapy Initiative
CIBMTR operated the Cancer Moonshot initiative, NCI-funded program from 2018 to 2023; CIDR was key to developing a robust infrastructure for cellular immunotherapies. As the CIDR initiative sunsets, cellular therapy activities will shift to the Adoptive Cellular Therapies (ACT) Initiative, which will include other cell and gene therapies.
CIBMTR’s ACT Initiative will continue to expand and optimize the database to appropriately capture data on new cell and gene therapy products and outcomes and oversee the research projects including the interaction with cell and gene therapy manufacturers.
The activities of ACT will be overseen primarily by the CIBMTR Advisory Committee, which will rely on a newly developed ACT Stakeholder’s Council (ACT Council) to provide additional input on important topics and initiatives in this field. The ACT Council will assist in being a liaison between different stakeholders and CIBMTR, i.e., including the cell and gene therapy community, professional societies, payors, and industry, to better position CIBMTR as a resource to the field.
The responsibility of the ACT Council is to serve as a standing taskforce to guide the Advisory Committee and CIBMTR leadership. Topics to be addressed by the ACT Council are selected and prioritized by CIBMTR’s Advisory Committee and can come from the CIBMTR Assembly, CIBMTR leadership, the ACT Council, and the Advisory Committee. Selected topics are reviewed, and recommendations are presented to CIBMTR’s Advisory Committee.
The ACT Council meets in person annually at the Tandem Meetings, or at the AcCELLerate Forum and by teleconference as needed.
ACT Council membership includes:
- Amanda Olson, MD, MD Anderson Cancer Center, Houston, TX
- John Wagner, MD, University of Minnesota Blood and Marrow Transplant Program, Minneapolis, MN
- Lori Henderson, PhD, NIH-NCI Government Agency Partners, Bethesda, MD
- Marcelo Pasquini, MD, MS, CIBMTR MCW, Milwaukee, WI
- Mei-Jie Zhang, PhD, CIBMTR MCW, Milwaukee, WI
- Meryl Selig, MS, CIBMTR Consumer Advocacy Committee Representative, Columbia University, San Francisco, CA
- Nirali Shah, MD, MHSc, NCI-NIH, Bethesda, MD
- Rayne Rouce, MD, Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX
- Rebecca Gardner, MD, Seattle Children’s Hospital, Seattle, WA
- Shannon Maude, MD, PhD, Children’s Hospital of Philadelphia, PA
- Sung-Yun Pai, MD, NCI-NIH, Bethesda, MD (gene therapy topics)
Status of Post-Authorization Studies
CIBMTR’s goals to advance cellular therapy have been to develop and provide one of the largest cellular therapy data repositories in the world and to focus on a continuing collaboration with both industry representatives and our colleagues in the cellular therapy field.
- Completed accrual for the following studies:
- Tisagenlecleucel (Novartis) study of pediatric ALL: 1,000 patients
- Axicabtagene ciloleucel (Kite Pharma) study with diffuse large B-cell lymphoma cohort of 1,500 patients and follicular lymphoma cohort of 300 patients
- Idecabtagene vicleucel (Bristol Myers Squibb) study of multiple myeloma: 1,500 patients
- Brexucabtagene autoleucel (Kite Pharma) study of mantle cell lymphoma: 500 patients
- Rapid accrual of recipients of B-cell maturation antigen-directed CAR-T cell products for the treatment of multiple myeloma
- Capture data on recipients of out-of-specification products using the infrastructure for long-term follow-up once these patients enter Bristol Myers Squibb’s expanded-access protocols
- A new project with Kite Pharma to run research studies on patients who receive Kite products under the auspices of a steering committee and alignment with Working Committee studies to avoid competition
Next Steps for Data Collection and Liaison with Industry Partners
- Developing similar research-focused contracts as was done with Kite Pharma, Novartis, and Bristol Myers Squibb. The objective is to create a path for collaboration across the stakeholders, CIBMTR, industry, and the community to address research questions in the field
- CIBMTR will not only continue to work to release appropriate data to the larger cellular therapy community (working through the embargo process) via CIBMTR committee work but will also include center directors and cellular therapy community members as part of steering committees that directly input and oversee the development of industry analyses
Adding Solid Tumors, Autoimmune Conditions
- CIBMTR plans to expand collection of data for indications such as solid tumors and autoimmune disease. In the solid tumor arena, the initial focus is emerging cellular therapies for the treatment of sarcomas, using T-cell receptor-edited cells. CIBMTR is currently updating the existing sarcoma forms in collaboration with industry partners and sarcoma experts. Additionally, CIBMTR is developing processes to create a rapid mechanism for solid tumor-specific domains that can be used for data capture in this field. Considering that cellular therapies will likely be used in different types of malignancies that share common targets, this challenges CIBMTR to rapidly develop improved data-capturing tools. Collaborations with oncology data repositories to use curated fields in this area are being sought as a solution to address this issue.
- Similarly, cellular immunotherapy is expanding to autoimmune diseases, with new industry partners reaching out to CIBMTR to seek assistance in capturing long-term follow-up in recipients of these therapies. Fortunately, CIBMTR has several autoimmune disease forms that may easily be repurposed for use in this setting.
CIBMTR has contracted with 2 gene therapy clients for 3 registry data studies expected to launch in late 2023 or early 2024. Bluebird bio is a Massachusetts-based biotechnology company that develops gene therapies for severe genetic disorders. They have contracted for a long-term 15-year follow-up study of cerebral adrenoleukodystrophy that will enroll at least 120 patients. Bluebird bio has also contracted with CIBMTR for a long-term 15-year follow-up study of beta thalassemia; the study will enroll 150 patients.
Vertex Pharmaceuticals is a worldwide biotechnology company with headquarters in Boston, Massachusetts, and London, United Kingdom, that concentrates on potentially transformative treatments for cystic fibrosis, pain, sickle cell disease, beta thalassemia, alpha-1 antitrypsin deficiency, APOL1-mediated kidney disease, Duchenne muscular dystrophy, and type 1 diabetes. Vertex Pharmaceuticals will work with CIBMTR on a long-term 15-year follow-up study of transfusion-dependent beta thalassemia or severe sickle cell disease.
De-Identified Cord Blood Research Dataset
To support the evidence and research pillars prioritized by the cord blood community, members of the Cord Blood Advisory Group and its Research/Regenerative Medicine Subgroup suggested that a broad, readily available observational cord blood dataset would support those who want to interrogate data independently. The members determined that aggregated, de-identified data that are currently available and shared with cord blood banks via CIBMTR would meet these needs.
This De-Identified Cord Blood Research Dataset is now available by request. To access the dataset:
Please direct any questions to Jenna Kohn, clinical data analyst II.
Share Your Research in Plain Language
By Jennifer Motl
These new plain-language summaries of CIBMTR research may help your patients:
Find more summaries on the Study Summaries for Patients webpage.
CIBMTR is supported primarily by Public Health Service U24CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI), and the National Institute of Allergy and Infectious Diseases (NIAID); U24HL138660 and U24HL157560 from NHLBI and NCI; OT3HL147741 from the NHLBI; 75R60222C00008, 75R60222C00009 and 75R60222C00011 from the Health Resources and Services Administration (HRSA); and N00014-21-1-2954 and N00014-23-1-2057 from the Office of Naval Research.
Additional federal support is provided by P01CA111412, R01CA100019, R01CA218285, R01CA231141, R01CA231838, R01CA262899, R01AI128775, R01AI150999, R01AI158861, R01HL155741, U01AI069197, U01AI126612, UG1HL069254.
Support is also provided by Be The Match Foundation; Boston Children’s Hospital; Fred Hutchinson Cancer Center; Gateway for Cancer Research, Inc.; Jeff Gordon Children’s Foundation; Medical College of Wisconsin; National Marrow Donor Program; PBMTF; St. Baldricks’s Foundation; Stanford University; University of Pittsburgh; and from the following commercial entities: AbbVie; Actinium Pharmaceuticals, Inc.; Adaptive Biotechnologies Corporation; ADC Therapeutics; Adienne SA; Alexion; Allogene; AlloVir, Inc.; Amgen, Inc.; Angiocrine; Astellas Pharma US; AstraZeneca; Atara Biotherapeutics; BeiGene; BioLineRX; Blue Spark Technologies; bluebird bio, inc.; Blueprint Medicines; Bristol Myers Squibb Co.; CareDx Inc.; CSL Behring; CytoSen Therapeutics, Inc.; DKMS; Elevance Health; Eurofins Viracor, DBA Eurofins Transplant Diagnostics; Gamida-Cell, Ltd.; Gift of Life Biologics; Gift of Life Marrow Registry; GlaxoSmithKline; HistoGenetics; Incyte Corporation; Janssen Research & Development, LLC; Janssen/Johnson & Johnson; Jasper Therapeutics; Jazz Pharmaceuticals, Inc.; Karius; Kashi Clinical Laboratories; Kiadis Pharma; Kite, a Gilead Company; Kyowa Kirin; Labcorp; Legend Biotech; Mallinckrodt Pharmaceuticals; Med Learning Group; Merck & Co.; Mesoblast; Millennium, the Takeda Oncology Co.; Miltenyi Biotec, Inc.; MorphoSys; MSA-EDITLife; Neovii Pharmaceuticals AG; Novartis Pharmaceuticals Corporation; Omeros Corporation; OptumHealth; Orca Biosystems, Inc.; Ossium Health, Inc.; Pfizer, Inc.; Pharmacyclics, LLC, An AbbVie Company; PPD Development, LP; REGiMMUNE; Registry Partners; Rigel Pharmaceuticals; Sanofi; Sarah Cannon; Seagen Inc.; Sobi, Inc.; Stemcell Technologies; STEMSOFT; Takeda Pharmaceuticals; Talaris Therapeutics; Vertex Pharmaceuticals; Vor Biopharma Inc.; WellSky; Xenikos BV. The views expressed in this article do not reflect the official policy or position of the National Institutes of Health, the Department of the Navy, the Department of Defense, Health Resources and Services Administration (HRSA) or any other agency of the US Government.