Cell & Gene Therapies
Cell and gene therapies are emerging therapies that represent a new treatment paradigm for many diseases including cancer. Friends of Cancer Research (Friends) supports alignment in the cell and gene therapy drug development space to ensure these innovative therapies make it to patients who need them faster. In cancer, current FDA approved cell therapies are indicated for treatment of certain hematologic malignancies. Our work focuses on enhancing the translation of evidence to support clinical development and adoption of these therapies for other patient populations and cancer types. Applying lessons learned from emerging clinical development programs and clinical data will help inform efforts to overcome obstacles and advance the next generation of cell therapies as well as gene therapies. Visit us on LinkedIn and X to stay up to date with #CGTFriends.
Recent approvals and clinical experience with cell therapies demonstrate their potential to improve outcomes for patients. Cell therapies, such as chimeric antigen receptor (CAR) T-Cell therapies, are a type of immunotherapy in which immune cells are engineered to help a patient’s immune system fight cancer. Despite recent advances using these therapies for treatment of hematologic malignancies, there are several unique challenges throughout drug development that limit widespread use. For example, cell therapies are often individualized, or developed for a specific patient, which requires novel approaches to development and manufacturing. Traditional regulatory frameworks and development processes must be adapted, and appropriate regulatory flexibility applied to enable more efficient, cost-effective methods for identifying safety signals and testing new candidate products as quickly and safely as possible.
Since the release of Friends’ first white paper related to cell and gene therapies in 2019, the FDA published several guidance documents incorporating concepts we proposed and continues to highlight advancing cell and gene therapies as a priority area of their focus:
- Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial (Draft Guidance, 2022)
- Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products (Draft Guidance, 2022)
- Human Gene Therapy Products Incorporating Human Genome Editing (Draft Guidance, 2022)
- Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products (Planned Draft Guidance, 2023)
These guidance documents provide cell and gene therapy developers and sponsors with the regulatory flexibilities that will ensure safe and effective products get to patients efficiently. As additional experience is gained and new challenges emerge related to the development and use of cell and gene therapies, Friends will continue to convene stakeholders to propose regulatory policy solutions that address these barriers to advance development and regulation of cell and gene therapy products. The goal of our work is to optimize development to support more efficient access, safe use, and enable broader application and access to the next generation of cell and gene therapies for patients.
How are we helping to find solutions?
Friends unites stakeholders from across patient advocacy, academia, industry, and the regulatory space to develop innovative approaches and flexible regulatory strategies to propose innovative solutions to speed innovation.
2019: Friends and the Parker Institute for Cancer Immunotherapy developed a white paper that identifies potential approaches to address development and regulatory hurdles encountered during early- and late-stage development of cell and gene therapies. The paper proposes applying investigational new drug application (IND) and manufacturing flexibilities during early-phase product development for streamlined regulatory submissions and evaluations of cell and gene therapy products. The paper also discusses approaches and data needed to determine whether a manufacturing change during late phase development will result in changes a product to safety or efficacy.
2020: Friends published a paper discussing how patient-reported outcomes (PROs) can be leveraged to inform patient experience and long-term safety of cell and gene therapies.
2022: Friends published a paper to address challenges related to reporting and characterizing emerging novel toxicities associated with the use of immuno-oncology therapies including cell therapies. The paper proposes evidence-based strategies for capturing and reporting cytokine release syndrome (CRS) and associated adverse events (AEs) which can support more consistent identification, monitoring, and management of CRS and enable accurate comparisons of these toxicities across therapies.
2023: Friends led multi-stakeholder discussions this year to evaluate how existing data can be safely leveraged across products and between versions of engineered cell therapy products to support more efficient development, iterative product improvements, and broader access to these therapies. Those discussions culminated in a public meeting and white paper to inform the development of the next generation of cellular therapies and support policy discussions between key stakeholders in the drug development process. As additional experience is gained using cell therapies, it will be important to evaluate the totality of evidence available to inform product modifications that support improvements to safety and activity.