A new drug combination, tetrahydrouridine (THU) and decitabine has shown great promise in the treatment of sickle cell disease (SCD). Results from a phase 1/2 clinical trial suggest this novel oral therapy was well tolerated and produced the desired effect on red blood cells by increasing fetal hemoglobin (HbF) levels twofold.
Yogen Saunthararajah, MD, a hematology expert and researcher at Cleveland Clinic has said that “We’ve known the molecular basis for this disease for decades, and now we finally have a rational, molecular-targeted therapy that addresses not just one or two symptoms of the disease, but the root cause of the disease,“This therapy can in principle address all the symptoms, all the complications sickle cell disease, so it’s very exciting.”
Dr. Saunthararajah is co-author on the study which was presented at the 2016 American Society of Hematology Annual Meeting in San Diego. Despite longstanding knowledge about this devastating disease, effective treatments for SCD have remained elusive. Patients still contend with clinically significant complications and risk of early death despite the standard of care, hydroxyurea. The new regimen is a non-cytotoxic, normal stem-cell-sparing, rationally targeted approach to disease modification.