To provide access to therapy for patients who otherwise may not receive treatment, St. Jude Children’s Research Hospital and the World Federation of Hemophilia are collaborating to establish and facilitate a program to conduct a gene therapy clinical trial for treatment of severe hemophilia B in low- and middle-income countries.
The trial will help hemophilia patients who may not currently receive consistent treatment, said Glenn Pierce, M.D., Ph.D., WFH vice president medical.
Hemophilia B is an inherited bleeding disorder caused by a defect in the F9 gene, which encodes instructions for making the factor IX protein that helps blood clot. Currently hemophilia B is treated with infusions of factor IX to prevent or stop debilitating and life-threatening bleeds. The therapy is expensive and the replacement clotting factor is scarce in some countries.
“Presently, individuals in these countries have significant morbidity and mortality due to their hemophilia B,” Pierce said. “Gene therapy represents a new modality that can eliminate peaks and troughs of clotting factor replacement therapy. This results in circulating significant amounts of factor IX that should prevent most, or possibly all, bleeding episodes.”
St. Jude will sponsor and conduct a phase 2 gene therapy clinical trial for adult and older adolescent hemophilia B patients in low- and middle-income countries.
Researchers at St. Jude and University College London have collaborated to pioneer gene therapy for treatment of hemophilia B. The approach uses a re-engineered virus as a vector to ferry the correct factor IX gene into liver cells.
The proposed clinical trial will improve the understanding of how advanced technologies, such as gene therapy for hemophilia B, can move rapidly to countries with limited resources. The collaboration will also demonstrate whether gene therapy is a safe and feasible approach for hemophilia B treatment in settings where factor IX replacement therapy is limited.
The vector used in the planned clinical trial will be produced in the Children’s GMP, LLC, a Good Manufacturing Practices facility on the St. Jude campus. The collaboration with the WFH will speed potential clinical advances by facilitating interactions between local hemophilia treatment programs and the St. Jude investigators.
“We are delighted to work with our experienced colleagues at WFH,” said Ellis Neufeld, M.D., St. Jude executive vice president and clinical director. “They have decades of experience working in settings of varied financial resources and have already facilitated interactions with expert and enthusiastic hemophilia physicians and investigators in several countries.”
“We are pleased to support St. Jude in their efforts,” said Alain Baumann, CEO of the WFH. “Our hope is to contribute to a solution of limited clotting factor replacement therapy in resource-constrained countries.”
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