What is the goal of the Cure Sickle Cell Initiative?

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:

  • Engage academic researchers, private sector researchers, and patients and their caregivers to develop strategies for cures.
  • Determine the safest, most effective, and most readily and widely adoptable genetic therapies.
  • Move newly developed genetic therapies, including gene-editing approaches, into clinical research.
AT A GLANCE
  • Sickle cell disease affects about 100,000 Americans and more than 20 million people worldwide.
  • The NHLBI-led Initiative will accelerate the development of genetic therapies and other cures for use in clinical research within five to 10 years.
  • The Initiative builds on NHLBI’s broader sickle cell disease research investment that aims to improve the lives of patients.
  • Patients are the most important part of NHLBI research; clinical trial participation will be critical to the Initiative’s success.
  • The Initiative is building partnerships, as well as supporting educational and community engagement activities.

What cures are being explored in the Cure Sickle Cell Disease Initiative?

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:

  • Introduce the normal hemoglobin gene into the HSCs.
  • Correct the mutation in the abnormal hemoglobin gene and transfer that corrected gene into the HSCs.
  • Modify the HSCs so they stop making abnormal hemoglobin and start making other hemoglobin.

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.

How is the Cure Sickle Cell Initiative contributing to scientific discoveries?

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:

  • Enhanced clinical trial recruitment and establishment of transplant standards to quickly and safety move clinical studies forward
  • Increased capacity to safely manufacture cellular therapy products through the NHLBI Production Assistance for Cellular Therapies (PACT) program, which includes cell processing facilities to produce genetically modified cells so they can be safely used in patients

How does the Cure Sickle Cell Initiative work?

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:

  • Funding research within academia and the private sector to identify the most promising cellular and genetic therapies. Resources come from the collaboration of professional societies, academic institutions, patient and advocacy groups, pharmaceutical companies, federal agencies, and biotechnology companies.
  • Partnering with federal agencies, academic institutions, pharmaceutical companies, professional societies, community advocacy organizations, patient representatives, and foundations.
  • Educating and engaging communities to inform patients, providers, and stakeholders about its work, ensuring trust and collaboration, while also motivating patients and caretakers to participate in and support clinical trials