Cmaj Podcasts

Transformative therapies for sickle cell disease are redefining what is possible for patients, offering the potential for cure alongside substantial risks. In CMAJ, the article Transformative therapies for sickle cell disease outlines how stem cell transplant and emerging gene therapies are changing the trajectory of a condition long defined by recurrent crises, shortened life expectancy, and inequities in care.Dr. Kareem Jamani, a haematologist and clinical associate professor at the University of Calgary, explains how stem cell transplant replaces a patient’s blood-forming system to eliminate sickling haemoglobin, offering what can reasonably be considered a cure. Outcomes are generally favourable, particularly with matched sibling donors, but risks remain, including graft-versus-host disease, infertility, rejection, and mortality that can reach 5–7% with less well-matched donors. He also outlines the role of gene therapy, which modifies a patient’s own stem cells to increase fetal haemoglobin production, r