Interview: Optimizing the Cell Collection Network to Support the Commercialization of Cell and Gene Therapies
Featuring Amy Hines, RN, BSN, Director of Collection Network Management, Kimberly Kasow, DO, and Sandra Hoffman, MT (ASCP) SSB, CQA (ASQ)Download
Outcomes data and repository solution
One of the great challenges in early stage drug or cellular therapy development is to quickly gather quality data that can form the basis for launching a full-scale clinical development program. We can help you get the data you need.
Our clinical outcomes database is a powerful tool for your study design and development.
Our parent company, the National Marrow Donor Program® (NMDP)/Be The Match®, has been collecting cell therapy outcomes data for 30 years. We can provide clinical outcomes data on approximately 480,000 autologous and allogeneic unrelated and related hematopoietic cell transplants to support your research. The data is available through our research program, the CIBMTR® (Center for International Blood and Marrow Transplant Research®), in collaboration with Medical College of Wisconsin.
The CIBMTR Clinical Outcomes Database includes outcomes data on engraftment, incidence and severity of GvHD, remission rates, non-transplant related deaths and relapse associated with each pair.
In the early stages of therapy development, our team can provide insights into the potential outcomes of the therapy you’re testing based on our proven trial designs or your unique endpoints.
You can access more than 2.1 million sample aliquots for research from more than 50,000 donor-recipient pairs.
Since 1987, NMDP/Be The Match has been collecting research samples with informed consent to perform retrospective analyses on all unrelated hematopoietic cell transplants facilitated by NMDP/Be The Match. Related donor-recipient samples from a select number of transplants have been collected since 2007.
These blood or peripheral blood mononuclear cell (PBMC) samples from donor-recipient pairs stored in our Research Sample Repository are consented for research use, and available to support researchers and commercial drug developers who are advancing the field of medicine.
Our team locates samples from the repository that meet your specifications, and assists with collection of additional data.
Our outcomes data and repository solution provides:
Therapy Outcomes Data Collection and Analysis
- Provide access to outcomes data collected on 480,000 autologous and allogeneic related and unrelated bone marrow transplant cases with longitudinal follow up of survivors that includes:
- Outcomes at 100 days, 6 months and yearly post-transplant
- Transplant-related survival, neutrophil and platelet engraftment, acute and chronic GvHD, and relapse
- Conditioning regimens, GvHD prophylaxis, immune reconstitution, chimerism, infection, organ function, subsequent HSCT or DCI, new malignancies and cause of death
Biorepository Access for Research
- Provide access to more than 2.1 million blood or PBMC sample aliquots from 50,000 donor-recipient pairs that meet client specifications
- Supply sample aliquots that include:
- HLA-characterized samples with detailed demographics; subsets include additional genetic typing data (e.g., KIR) and donor infectious disease markers (e.g., HIV, EBV, CMV)
- Adult normal donor samples
- Paired recipient/donor/cord blood unit samples from related and unrelated hematopoietic cell transplants
- Complete, validated clinical outcomes data from the CIBMTR Observational Database
Immunogenetics and Bioinformatics Support
- Provide full spectrum of HLA matching capabilities, from restriction to histocompatibility
- Customize genomic study of donor-recipient pairs to meet client’s specific research needs
- Assist with collection of genetic data up to genome sequencing when needed
When your cell therapy relies on unrelated donors, we have the knowledge to help you identify the right donors.
Wei and our Bioinformatics team have critical expertise in the fields of immunogenetics, geospatial analytics and information technology that can inform and advance your allogeneic cell therapy platform from initial R&D toward commercialization.
Cell sourcing solution
We have unparalleled expertise to help you identify donor cells with specific biomarkers and guide you through the complex steps to acquire fully consented cells for research, clinical or commercial use.
Finding fully consented donor cells that meet your specific needs isn’t easy.
Through the Be The Match Registry®, our bioinformatics and immunogenetics team has access to data on nearly 16 million diverse U.S.-based registry members and 225,000 umbilical cord blood units with rare and common haplotypes.
Our team has unique knowledge in customized algorithm development, registry access and analysis, genetic markers and histocompatibility.
We can conduct bioinformatics analyses of the registry along many axes including: genetic markers (HLA, ABO, KIR), demographics (sex, age), geospatial analysis and others.
Whether you’re looking for cells representative of large segments of the population or those that are unique, we can filter and optimize specific criteria to help you identify the right unrelated donors or cord blood units to provide consented cells as you research and develop your cell therapy.
We have the experience to take you from identifying donors to acquiring consented donor cells that meet regulatory and legal requirements for research, clinical or commercial development.
After our team has helped you identify compatible cell source material, we assist in your protocol design and development, recruitment materials and regulatory compliance, and guide you through the institutional review board (IRB) process. We also help you manage your operational process, including cell collection and delivery.
Our mission is to save lives through cellular therapy. By extending our Registry and unique expertise to new and emerging cellular therapies, we can help save more lives and improve the quality of life for patients.
Our customizable allogeneic cell sourcing solution offers:
Immunogenetics and Bioinformatics Support
- Provide unique knowledge in customized algorithm development, registry access and analysis, genetic markers and histocompatibility
- Identify the most genetically compatible cell sources for early stage therapy development – including cytotoxic T-cell (CTL) therapies, induced pluripotent stem cell (iPSC) therapies, NK (natural killer) cell therapies and others – for all stages of development
- Offer customized matching using our peer-reviewed search engine, HapLogicSM, to model and optimize allogeneic cell banks
Regulatory Compliance Expertise
- Expertise in protocol development that ensures donor safety, informed consent and compliance with necessary regulatory standards
- Manage IRB protocol submission and review throughout the process
- Ensure compliance with U.S. and other applicable standards for cell therapy donor starting material
Diverse Source Material Access
- Manage the largest and most diverse donor registry in the world with 16 million HLA-typed donors
- Offer high-resolution HLA typing of rare and common haplotypes
- Provide a variety of cell types, including bone marrow, PBMC (mobilized) and whole blood
Personalized Donor Case Management
- Contact the potential donors to explain the opportunity and facilitate full consent for research, clinical and/or and commercial development
- Manage the donor workup process to ensure the donor is both medically suitable and willing to donate
- Perform necessary screening and testing for donor eligibility determination per FDA and other applicable regulations
- Schedule donation with our cell collection network and coordinate logistics after the cells have been collected
- Ensure the safety and privacy of donors by following strict internal standards, plus all applicable regulations from governmental agencies
Cell Collection Network
- Develop and manage a national network of apheresis and blood centers for diverse allogeneic product collection—including whole blood, apheresis products, mobilized peripheral blood stems cells or cord blood—that meets regulatory requirements as well as client specifications
- Conduct audits to ensure ongoing compliance
- Provide ongoing and ad hoc training and associated documentation to ensure continued compliance
- Manage the delivery of donor cells from the collection center to the research center
- Provide regulatory support to ensure compliance with FDA Good Tissue Practices (GTP) and Good Manufacturing Practices (GMP) regulations
When you need to ensure regulatory compliance at every step, our team offers the support you need.
Regulatory compliance is critical at every step of a cellular therapy’s development. Ann and her team understand the regulations involved in identifying and collecting cells from a donor or patient, and can ensure all regulations applicable to your therapy are met.