New hope for hemophilia treatment


Researchers have created a fusion protein that could help space the infusions that hemophiliacs need.

New therapy to treat the most common form of hemophilia has shown promise for all 16 men in a clinical trial. This is what researchers report this September 10 in The New England Journal of Medicine.

Hemophilia is an inherited disease linked to the X chromosome which can take many forms but in all cases results primarily in a disruption of the blood clotting process so that minimal trauma can lead to prolonged bleeding, for example example. Hemophilia A affects about 1 in 6,000 men, while hemophilia B, which is rarer, affects 1 in 30,000 men. according to the French Association of hemophiliacs.

Lighten the burden on patients

Currently, treatments which consist in preventing bleeding require patients to inject the coagulation factor absent from their body by infusion, several times a week. “Thanks to what specialists consider a feat of bioengineered, the researchers succeeded in creating a protein fusion which should make it possible to lengthen the interval between treatments, to reduce it to a few days or even a week ”, reports the site US News.

This new therapy has not yet been the subject of a large-scale clinical trial, but the prolonged duration of the effect of coagulation factors exalts many researchers. “Frequent infusions can be a huge burden on patients”, points out to Science Barbara Konkle, a hematologist at the Bloodworks Northwest Research Institute and first author of the new study. “She and others on the ground have focused their efforts on ways to reduce this burden”, explains the scientific journal.

In an editorial accompanying the release of the study, Pier Mannucci, hematologist at the Polyclinic in Milan, assures us that this study “Potentially marks a clinically significant breakthrough, notwithstanding some questions that remain open”. The sentence III a clinical trial involving a large number of patients should answer these questions. If this final phase confirms the promises of the new therapy, the Bloodworks Northwest research institute hopes to submit its data to the US drug regulatory authority (FDA) with the aim of obtaining a marketing authorization by 2022.

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