Insights from the “Bridging the Gap” Webinar: Advancing Cellular Therapies
As the cell therapy industry continues to expand, one organization is leading the way in advanced therapies, ensuring that hospital programs for cell treatments, such as CAR T-cell therapy, follow rigorous protocols and measure outcomes. This group, the Foundation for the Accreditation of Cellular Therapies (FACT), is driven by volunteer inspectors, and led by a dynamic medical professional with decades of experience and an exceptional dedication to ensuring a high standard of patient care: Phyllis I. Warkentin, MD, FACT Chief Medical Officer.
Established in 1996, FACT is a non-profit organization that sets standards for excellence in medical and laboratory practices related to cellular therapies. It was co-founded by the International Society for Cell and Gene Therapy (ISCT) and the American Society for Transplantation and Cellular Therapy (ASTCT) with the aim of providing voluntary inspection and accreditation services in the field of cellular therapy.
The “Bridging the Gap ” Webinar on Dec. 1, 2023, explored the foundation’s history, recent activities, and initiatives for the future. The session featured a roundtable discussion and Q&A with Dr. Warkentin; Tom Whitehead from the Emily Whitehead Foundation; Kim Schuetz, an apheresis nurse and parent of a successful CAR T-cell therapy patient named Austin; Dr. Patrick Hanley, Chief & Director of the Cellular Therapy Program at Children’s National Hospital; and Azenta co-host Gil Edwards.
Read on for a summary of the key points the participants discussed.
Exploring Milestones and Collaborations
Dr. Warkentin provided valuable insights into FACT’s evolution, emphasizing its origins in the 1990s and collaboration with key organizations like ASTCT and ISCT. She highlighted the historical challenges FACT faced in managing expectations and relationships with regulators, including the importance of taking a proactive approach to influencing regulations and collaborating with regulators, particularly in the context of third-party manufacturers.
“In the 90s, bone marrow transplant was a becoming mature field, and as more and more programs were being formed, there was a perceived need among professionals in the field to have some kind of quality standard, which didn’t necessarily mean everybody doing everything the same way, but some way to measure our outcomes versus someone else’s outcomes, and help one another improve,” said Dr. Warkentin about the genesis of FACT.
The growth of FACT, from a volunteer-based initiative to a structured organization with international collaborations, was a testament to its commitment to advancing cellular therapies.
“The FDA had published some draft guidance about how they planned to regulate the field. There was some fear that overregulation might stifle research. That overregulation might limit access,” said Dr. Warkentin. “We believed that as professionals practicing in the field, we were probably in a better position to set what the standards should be.”
Today, FACT maintains internationally established standards, accreditation, and education as a core part of its initiatives. FACT is also working with third party manufacturers to establish qualification audits for sites that take part in any element of cell therapy, from cell collections to infusions. This strategic move aims to streamline accreditation processes, ensuring more qualified sites and ultimately improving patient access to advanced therapies.
Navigating Challenges and Charting Future Initiatives
During the webinar, Tom Whitehead emphasized the ongoing dedication of the Emily Whitehead Foundation in assisting families facing pediatric cancer. He highlighted the organization’s mission to connect individuals like Olympian wrestler Michelle Forzani with potential treatment options. Additionally, Tom underscored the importance of successful fundraising events, such as the Thanksgiving Turkey Trot and the Annual Gala, in raising awareness and furthering the organization’s mission.
Another highlight was the contribution of Kim Schuetz, an apheresis nurse and parent of a successful CAR T-cell therapy patient named Austin. Austin, the 21st pediatric patient in the original CAR T-cell trial, celebrated a remarkable 10-year remission milestone. Kim shed light on her dual role as a nurse involved in cell collections and a parent navigating the challenges of pediatric cancer treatment.
Dr. Warkentin made a compelling call to industry professionals, urging collaboration to streamline processes, reduce audit burdens, and facilitate more efficient quality assessments. She shared how FACT’s entry into grant applications marked a significant development, opening a new avenue for financial support beyond accreditation fees. She also emphasized the potential impact this may have on education and international outreach efforts, especially in countries with accredited programs but limited access to immune effector cell therapies.
A pivotal discussion point was the evolving landscape of cellular therapies, transitioning from self-contained transplant centers to standalone entities serving multiple clinical centers and pharmaceutical companies. Dr. Warkentin stressed the need for improved communication with industry stakeholders, advocating for a dedicated platform to effectively convey the depth and breadth of FACT standards.
The discussion also addressed the challenge of recruiting more volunteer inspectors for FACT, highlighting the ongoing need for education, mentorship, and curriculum development to ensure a robust peer-to-peer inspection process.
A few key takeaways from the webinar:
- Commitment to pediatric cancer support: Both FACT and the Emily Whitehead Foundation are united in their commitment to ensuring that more cancer patients are successfully paired with advanced treatment options.
- FACT’s global impact: FACT has evolved from a volunteer-based initiative to an internationally recognized organization collaborating with key entities to set global standards for cellular therapies.
- Streamlining accreditation: FACT has taken a proactive approach in addressing diverse accreditation challenges, with a focus on reducing audit burdens and enhancing patient access to advanced therapies.
- Collaborative industry approach: FACT continues to value collaboration with industry stakeholders to streamline processes, reduce audit burdens, and facilitate efficient quality assessments.
- Addressing inspector shortage: FACT faces a critical need for more volunteer inspectors and is committed to addressing this challenge through education, mentorship, and curriculum development.
Bridging the Gap Addresses Important Topics
The “Bridging the Gap Series” webinar provided a comprehensive overview of the current landscape of cellular therapies, accreditation challenges, and FACT’s efforts to enhance patient care and access to innovative treatments. The commitment of organizations like the Emily Whitehead Foundation and the collaborative initiatives led by FACT underscored the collective determination to advance cellular therapies and improve outcomes for patients worldwide.
As we reflect on the insights shared during this insightful event, it is evident that a collaborative and forward-thinking approach is crucial to addressing the evolving needs and challenges in the field of cellular therapies.
Want to know more? Register for upcoming webinars in the series (held monthly on the first Friday).
About the Author
Phyllis Warkentin
Phyllis Warkentin, MD, is a founding member and the chief medical officer of FACT (Foundation for the Accreditation of Cellular Therapy). Since the organization’s inception in 1996, she has served as a board member. Dr. Warkentin has also been the director of the unrelated bone marrow transplantation program at the University of Nebraska Medical Center since 1988 and a professor of pediatrics and pathology/microbiology at the University of Nebraska Medical Center since 1994. Her extensive contributions to the medical field, particularly in cellular therapy and bone marrow transplantation, showcase her dedication to advancing research and enhancing patient care.