Terry Flotte, MD, a leading figure in gene therapy and Dean at UMass Chan Medical School, shares his journey from childhood inspiration to groundbreaking research. He discusses the pioneering work in AAV gene therapy for cystic fibrosis, emphasizing safety and technological evolution. Alongside ASGCT President Hans-Peter Kiem, MD, PhD, they explore the future of gene therapy, touching on exciting advancements aimed at treating genetic disorders. Flotte's passion for pediatrics and personal interests, including baseball and obscure films, add a delightful touch.
Dr. Terry Flotte's pioneering work with AAV vectors in gene therapy has significantly advanced treatments for genetic disorders, especially in children.
His personal experiences and mentor influences deeply motivated his dedication to integrating scientific research with patient care in pediatrics.
Deep dives
ASGCT Annual Meeting Highlights
The annual meeting of the American Society of Gene and Cell Therapy (ASGCT) is highlighted as an essential event for networking and knowledge-sharing among professionals in the field. Scheduled from May 16 to 20, the meeting will showcase over 1,700 research presentations, including various clinical trials and advancements in gene therapy. It offers both in-person and virtual attendance options, enabling a global audience to engage with expert insights. This event serves as a valuable platform for attendees to connect with peers and discuss groundbreaking research that is shaping the future of gene and cell therapy.
Dr. Flott's Inspiring Journey
Dr. Flott's background reveals a deep-seated passion for medicine stemming from personal experiences, particularly the loss of his father to a cardiac event during his adolescence. Raised in Louisiana, he pursued education at public universities, ultimately opting for Johns Hopkins for his residency due to its commitment to both clinical and academic excellence. Influenced by notable mentors, he realized the significant impact of integrating scientific research with patient care, which propelled him into pediatrics. This decision was cemented by his desire to improve human outcomes and provide better health solutions for families.
Pioneering Gene Therapy Research
Dr. Flott’s groundbreaking work revolves around the use of Adeno-associated virus (AAV) as a vector for gene therapy, which he employed in the first human trial for cystic fibrosis. This innovative approach led to critical advancements in the understanding of gene transfer and the role of AAV in targeting non-dividing cells. Despite facing initial challenges and public concerns regarding environmental safety during the early trials, the findings have paved the way for the expansive application of AAV in treating various genetic disorders. The shift in focus from integrating vectors to utilizing episomal vectors instigated a transformative understanding of gene therapy delivery systems.
Future Perspectives in Gene Therapy
Looking ahead, Dr. Flott envisions a landscape where gene therapies are prevalent for a myriad of genetic conditions, referencing the success of recent approvals for treatments related to hemophilia and spinal muscular atrophy. He emphasizes the necessity for enhancing the efficacy and delivery mechanisms of AAV to accommodate larger target populations while minimizing potential toxicity. Ongoing research aims to refine gene therapy technologies and possibly integrate gene editing approaches to tackle a broader spectrum of genetic disorders. Dr. Flott expresses a commitment to advancing research efforts that can address the needs of patients with rare genetic diseases, underscoring the importance of continued innovation in this evolving field.
Terry Flotte, MD, discusses the early days and possible future of gene therapy, the use of AAV to deliver GT to targeted sites in the body, and his inspiration behind becoming a physician, scientist, and medical school dean. He spoke one-on-one with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the sixth episode of Giants of Gene Therapy!
Dr. Flotte is the Provost, Dean, and Celia and Isaac Haidak Professor at UMass Chan Medical School, with faculty appointments in the Department of Pediatrics and the Horae Gene Therapy Center. He is also a practicing pediatric pulmonologist and a prolific researcher who has authored more than 280 papers and been the principal investigator of multiple clinical gene therapy trials. Dr. Flotte is the editor-in-chief of his field’s oldest journal family, Human Gene Therapy.
In 1995, Dr. Flotte was the principal investigator for the first human use of AAV vectors, in a trial in cystic fibrosis patients. He’s currently investigating the use of gene therapy for genetic diseases that affect children, including alpha-1 antitrypsin (AAT) deficiency and lysosomal storage diseases.
Dr. Flotte has been an ASGCT member since the Society’s founding. He’s served on the board and committees, and spoken at the ASGCT Annual Meeting and other events.