Professional Development Cafés
Who:
- Speaker: David A. Wilcox, PhD, Medical College of Wisconsin
- Moderator: Juliana Alvarez Argote, MD, Medical College of Wisconsin
What: A Professional Development Café
Why: Join us to hear from David A. Wilcox, PhD, a professor in the department of pediatrics, division of hematology and oncology, at the Medical College of Wisconsin.
This presentation will describe the goal, strategy, background, and outcome of the first three patients enrolled in a phase I gene therapy trial for gene replacement of human FVIII in autologous hematopoietic stem cells (HSC). The treatment aims to induce long-term hemostasis for humans with severe hemophilia A with factor VIII delivered and secreted at the site of vascular injury from activated blood platelets. The presentation will focus on the analysis of Patient 1 who went from 10 bleeding events requiring rFVIII prophylaxis the year before gene therapy to zero bleeding events per year for more than two years after infusion of HSC genetically modified with platelet-derived FVIII, “Pleightlet.” This trial remains open for enrollment.
Learning Objectives
Following this event, attendees will be able to:
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Describe the role of factor VIII in blood clotting and its relevance to hemophilia A
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Discuss the significance of using platelet-derived factor VIII in gene therapy for hemophilia A
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Outline the key steps involved in conducting a first-in-human clinical trial for gene therapy
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Analyze the potential benefits and risks of gene therapy for severe hemophilia A patients
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Compare platelet-derived factor VIII gene therapy to current standard treatments for severe hemophilia A
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