The St. Jude Children’s Research Hospital and the World Federation of Hemophilia (WFH) recently announced that they have entered into a unique collaboration to establish gene therapy clinical trials for individuals with hemophilia B who live in low-to-middle income countries. Historically, patients in many developing countries have limited access to effective therapies.
“Presently, individuals in these countries have significant morbidity and mortality due to their hemophilia B,” Glenn Pierce, MD, PhD, WFH’s vice president, said in a press release. “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.”
One of the primary purposes of the partnership is to advance gene transfer therapies through strong relationships with local treatment programs and St. Jude’s investigators, who pioneered breakthrough research on gene therapy for hemophilia B. In recent years, researchers from St. Jude and the University College London (UCL) successfully used adeno-associated viruses (AAVs) to deliver the genes to liver cells to prompt FIX production in people with hemophilia B. AAVs, which are currently being evaluated in several gene therapy trials, deliver genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses.
Investigators for the first trial will enroll both adult and older-adolescent hemophilia B patients and will include the gene therapy approach developed by St. Jude and UCL. According to a St Jude press release, the clinical trial will enhance understanding of how advanced technologies, such as hemophilia B gene therapy, can move rapidly to countries with limited resources.
“We are delighted to work with our colleagues at WFH,” said Ellis Neufeld, MD, St. Jude’s 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.”
Source: Hemophilia News Today, May 10, 2019