A new collaboration between St. Jude Children’s Research Hospital and the World Federation of Hemophilia (WFH) is aimed at helping blood disorder patients in countries where they may not receive regular treatment.
The partnership’s centerpiece will be a program to conduct a gene therapy clinical trial in low- and middle-income nations for treatment of severe cases of hemophilia B. The ultimate hope is that the collaboration will advance promising treatments by facilitating relations between local treatment programs and St. Jude researchers.
“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.”
A genetic disorder caused by missing or defective factor IX (a blood clotting protein), hemophilia B is roughly four times rarer than type A. In the most severe cases, bleeding symptoms may occur after a minor injury or without a known cause.
Currently, the disease can be treated with replacement therapy, which calls for an infusion of the missing clotting factor. However, the therapy is pricey and unavailable in some countries. According to the WFH, 75% of bleeding disorder patients receive inadequate treatment, if at all.
Sponsored by St. Jude, the planned Phase 2 trial will include adult and older-adolescent hemophilia B patients, and will use a new gene therapy produced jointly by St. Jude and University College London. The method uses a re-engineered virus (adeno-associated virus) as a vector made on St. Jude’s campus to transport the F9 gene — which provides instructions for creating factor IX — into liver cells.
It is hoped that the planned study will enhance understanding of how quickly such emerging technologies can be used in countries with limited resources. In addition, the initiative will show whether gene therapy is a viable option in areas where factor IX replacement therapy is limited.
”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.”
For more than 50 years, the WFH has aimed to improve and sustain care for those with inherited bleeding disorders through training, advocating, and education.
St. Jude is the sole National Cancer Institute-designated comprehensive cancer center devoted exclusively to children.