Punam Malik, MD., a physician-scientist at the Cincinnati Children’s Cancer and Blood Diseases Institute and director of its Comprehensive Sickle Cell Center, presented promising data for a new gene therapy treatment for Sickle Cell Anemia, which has so far reversed disease symptoms in two adults.
Cincinnati Children’s developed the therapy, which, Malik calls “very promising.”
“One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms,” she says. “This includes near elimination of chronic pain and sickling events and improved anemia.”
“If sustained,” she says, “this therapy will provide a transportable, safe and feasible gene therapy for all SCA patients.”
To read more about the research, visit EuekAlert.