Patients who rely on recombinant, protein-based drugs must often endure frequent injections, often several times a week, or intravenous therapy. Researchers at Children’s Hospital Boston demonstrate the possibility that blood vessels, made from genetically engineered cells, could secrete the drug on demand directly into the bloodstream. In the November 17 issue of the journal Blood, they provide proof-of-concept, reversing anemia in mice with engineered vessels secreting erythropoietin (EPO).
The technology could potentially be used to deliver other proteins such as Factor VIII and Factor IX for patients with hemophilia, alpha interferon for hepatitis C and interferon beta for multiple sclerosis, says the study’s principal investigator, Juan Melero-Martin, PhD, of the Department of Cardiac Surgery at Children’s.