The Cure Sickle Cell Initiative is a NHLBI-led collaborative research effort that will accelerate the development of genetic therapies to cure sickle cell disease. The Initiative will identify and support the most promising genetic therapies so they can be safely used in clinical research within five to 10 years. The Initiative aims to transform the lives of people who have sickle cell disease by creating a collaborative, patient-focused research environment. With new advancements in genetic therapy, the time is right to push toward cures that are more accessible to the approximately 100,000 Americans, and more than 20 million people worldwide, who have sickle cell disease.
Other goals of the NHLBI’s Cure Sickle Cell Initiative:
Read more about the Cure Sickle Cell Initiative in the Presidential Message on National Sickle Cell Disease Awareness Month.
Currently, bone marrow transplants can cure sickle cell disease, but they are most effective in children who have well-matched donors. The Cure Sickle Cell Initiative aims to develop curative strategies by initially focusing on genetic therapies that modify hematopoietic stem cells (HSCs), which make red and other blood cells. These modified HSCs can then be used in bone marrow transplants, making a cure available to more patients.
The following are possible ways to use genetic therapies to modify patients’ HSCs so that their red blood cells are no longer sickle-shaped:
These genetic therapy approaches are in the early stages of testing. The Initiative aims to bring these genetic therapies to the U.S. Food and Drug Administration (FDA) for review and approval so that patients can begin enrolling in clinical trials within five to 10 years.
The Cure Sickle Cell Initiative builds on the legacy of NHLBI-supported research that has contributed to improving clinical care for patients who have sickle cell disease. It will also complement the Institute’s broader sickle cell disease research investment, which includes basic, clinical, translational, and implementation science research.
The Initiative supports the following:
Through the years patients have been at the heart of NHLBI’s research progress. This Initiative will recruit individuals from the patient community to serve at all levels of governance—from the Executive Committee to every subcommittee. Similarly, as the Initiative starts new clinical trials, it will recruit patients who have sickle cell disease, as their participation will be critical to the Initiative’s success.
The Initiative is also supporting these activities:
The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates...