A team at NewYork-Presbyterian and Columbia University Irving Medical Center is pioneering a potential new treatment that may offer hope for patients with sickle cell disease (SCD), a devastating inherited blood disorder that causes anemia, infection, debilitating pain, and organ damage.
More than 100,000 Americans and millions more across the globe have this life-threatening disease, which causes red blood cells to change shape and malfunction, clogging blood vessels and preventing oxygen from moving throughout the body.
SCD is more common among people of African descent. Experts believe the genetic trait evolved because it protects people from malaria. In the United States, SCD occurs in about 1 of every 365 black or African-American births, according to the Centers for Disease Control and Prevention.
“Patients who are born with SCD, depending on the severity, have a reduced life expectancy, and so far the only way of curing this disease is through a bone marrow or stem cell transplant,” says Dr. Markus Mapara, director of the Bone Marrow Transplantation and Cell Therapy Program at NewYork-Presbyterian/Columbia University Irving Medical Center. “Unfortunately, the chances of having a matched sibling donor, which provides the best outcomes, are only 25%. So it’s important for us to find other ways to cure this disease.”
Today, one new approach is showing promise. Dr. Mapara is leading a groundbreaking clinical trial at NewYork-Presbyterian/Columbia University Irving Medical Center, which is part of a larger multicenter trial, that is using gene therapy to try to cure SCD patients. Health Matters spoke with Dr. Mapara to learn about the experimental trial that has shown encouraging results for patients.
“Advances in gene therapy are unlocking potential new treatments for this disease, and I am excited that they may offer hope and a new option for those suffering from SCD,” says Dr. Mapara.