Overview
The CIBMTR operated the Moonshot Initiative, NCI-funded CIDR program from 2018 to 2023, which was key to developing a robust infrastructure for cellular immunotherapies. As the CIDR program sunsets, cellular therapies activities will shift under Adoptive Cellular Therapies (ACT), which will include other cell and gene therapies.
CIBMTR ACT 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.
Adoptive Cellular Therapies (ACT)
In addition to receiving data on transplant recipients, CIBMTR receives data about patients who received other adoptive cellular therapies. Most activity is focused on the use of chimeric antigen receptor (CAR)-T cells for the treatment of cancer, although this structure has the ability to capture information on any cellular product beyond CAR T cells and for the treatment of solid tumors and indications other than cancer. CIBMTR receives these data via a suite of CTED forms and continues to work with international registries to review and harmonize data collection globally.
Gene Therapy
CIBMTR also collects data in the gene therapy arena to serve the research community. CIBMTR Gene Therapy Working Group collaborated with an external task force comprised of national gene therapy experts to determine how to best leverage the CIBMTR infrastructure to efficiently collect data for long-term follow-up of patients undergoing an autologous HCT using a genetically modified product. CIBMTR developed a new Gene Therapy Product form and revised key disease-specific forms to capture information on the most common indications for these therapies.
ACT Stakeholder's Council
Responsibilities
- Acts as a specialized group in cell and gene therapies that provides input to the Advisory Committee and to CIBMTR leadership on relevant topics in the field.
- Assists 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
Membership
- ACT Council membership includes a CIBMTR Scientific Director; three members with ACT expertise (core); one Chair (who serves on CIBMTR Advisory Committee); at least three affiliate members depending on the topic being discussed; and ex officio members.
The affiliate members are assigned and approved by the Advisory Committee, including the NIH project officers.
- Ex officio members include:
- CIBMTR Leadership and invitees
- NCI project officer
- Consumer Advocacy Committee representative
Meetings
The ACT Council meets in person annually at the Tandem Meetings, or at the AcCELLerate Forum and by teleconference as needed.