Stem cell treatment and gene therapy could drastically improve the lives of more than 20 million people globally who have the genetic blood disorder sickle cell disease, says a leading expert from a top American paediatric hospital, Cleveland Clinic Children’s, marking Sickle Cell Disease Awareness Month in September.
Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. In a recent trial presented at the virtual American Society of Gene and Cell Therapy Annual Meeting in May, the results of a phase 1/phase 2 trial LentiGlobin gene therapy led to reductions in sickle cell disease-related complications and haemolysis.
New frontiers in sickle cell disease
According to Ravi Talati, a doctor of osteopathic medicine in the Department of Pediatric Hematology and Oncology at Cleveland Clinic Children’s: “Sickle cell disease is entering a new frontier where emerging treatments in stem cells and gene therapy can help people to significantly reduce comorbidities related to sickle cell disease and enable healthier lives for years. Newer treatments include transplanting stem cells from healthy bone marrow that can produce normal red blood cells, in addition to gene therapy, which can remove the abnormal gene or increase the amount of foetal haemoglobin created.”
More than 20 million people worldwide have sickle cell disease, according to the National Heart, Lung, and Blood Institute. In addition, the Sickle Cell Disease Foundation says that more than 250 million people globally have the sickle cell genetic mutation, putting them at risk of passing this mutation to their children. Sickle cell is prevalent in the Middle East, South Asia, Africa, Latin America, and the Mediterranean.
Treatment options
Fortunately, while clinical trials of newer therapies continue, sickle cell disease patients have a wide range of treatments that can help to reduce pain. For example, penicillin can reduce the incidence of infection, hydroxyurea can help to keep red blood cells in a spherical shape, and L-glutamine can help red blood cells to regain their flexibility.
Recently, the U.S. Food and Drug Administration approved two new drugs for sickle cell disease: crizanlizumab-tmca, a monthly infusion that can prevent or reduce pain, and voxeletor, which can help to prevent the red blood cells from turning into a sickle shape.