The Cure Sickle Cell Initiative is a collaborative research effort to accelerate the development of genetic therapies to cure sickle cell disease. Working closely with researchers in academia and industry, clinicians, patients, and advocates, the initiative will identify and support the most promising genetic therapies for scalable cures. 

The NHLBI-led Cure Sickle Cell Initiative fosters a collaborative, patient-focused research environment that will identify and support the most promising and innovative genetic therapies to cure sickle cell disease. To that end, NHLBI will engage patients, families, advocates, and researchers from academia and the pharmaceutical industry, to leverage resources and fill in gaps that cannot be covered by traditional funding strategies. The Initiative’s support is in addition to NHLBI’s broader sickle cell disease research investment that aims to improve the lives of patients.

Currently, bone marrow transplants are the only cure for sickle cell disease. In this procedure a sick patient receives bone marrow from healthy, genetically compatible sibling donors. However, only about 18 percent of children with sickle cell disease have a healthy, matched sibling donor.

The Cure Sickle Cell Initiative aims to develop cures for all patients who have sickle cell disease and is 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.

Visit Scientific Activities & Updates to see the list of projects that the Cure Sickle Cell Initiative has funded so far.

The Cure Sickle Cell Initiative is a collaborative, patient-focused research effort designed to accelerate promising genetic therapies to cure sickle cell disease (SCD). The Initiative allows us to rethink the traditional ways that we advance research and brings together the SCD community—including patients, advocates, caregivers, researchers, providers, industry, and others.  Launched in September 2018 by the National Heart, Lung, and Blood Institute (NHLBI), the Initiative aims to transform the lives of individuals living with SCD by moving the most promising therapies safely into clinical trials.

The NHLBI-led Cure Sickle Cell Initiative fosters a collaborative, patient-focused research environment that will identify and support the most promising and innovative genetic therapies to cure sickle cell disease. To that end, NHLBI will engage patients, families, advocates, and researchers from academia and the pharmaceutical industry, to leverage resources and fill in gaps that cannot be covered by traditional funding strategies. The Initiative’s support is in addition to NHLBI’s broader sickle cell disease research investment that aims to improve the lives of patients.

This initiative builds on the foundation of advancements in basic science that has been consistently supported by NHLBI and others. That foundational work has brought us to this moment.

The science is now at a point where a focused, collaborative and coordinated approach that includes academia and industry researchers, clinicians, patients, and advocacy groups will help us find a cure.

With the growing number of tools available to correct the genetic mutation associated with sickle cell disease, researchers now have an opportunity to develop genetic based curative therapies for a far broader group of patients.

NHLBI’s Cure Sickle Cell Initiative is being supported by a variety of funding mechanisms. We plan to leverage existing NIH investments from our intramural and extramural portfolios. NIH will also help facilitate public-private partnerships to help bring new tools and treatments to market.  In addition, NHLBI plans to utilize something referred to as an “Other Transaction Authority (OTA)”.

It allows NHLBI the flexibility it needs to form unique partnerships with a diverse set of stakeholders to accelerate discovery and translation. It provides an opportunity to reach out and work with non-traditional partners, less familiar with working with government. It also allows for a nimbler framework to follow the science and technology and adapt or redirect strategies as needed.  

Basically, the OTA allows NHLBI the ability to build the infrastructure and expertise needed to accelerate treatments aimed at a cure for sickle cell disease.

Yes, Congress has authorized 11 federal agencies, including HHS and NIH, to use this mechanism. NASA first used this authority in 1958. Most agencies use it for research and development purposes. NIH has used it as far back as 2004 to help stimulate the Nanomedicine Program. More recently, NIH used OTA to help support the launch of the All of Us Program.

Currently, bone marrow transplants are the only cure for sickle cell disease. In this procedure a sick patient receives bone marrow from healthy, genetically compatible sibling donors. However, only about 18 percent of children with sickle cell disease have a healthy, matched sibling donor.

The Cure Sickle Cell Initiative aims to develop cures for all patients who have sickle cell disease and is 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.

Visit Scientific Activities & Updates to see the list of projects that the Cure Sickle Cell Initiative has funded so far.

NIH spends approximately $100 million on sickle cell disease research each year. Through this Initiative, NHLBI is anticipating supporting development of cell and genetic therapies resources, clinical trials of genetic therapies, comparator analyses of different management strategies, data repositories and resources, and patient and advocate engagement activities related to curative therapies for Sickle Cell. At launch NHLBI committed $7 million to jumpstart the Initiative’s research and engagement infrastructure and the Institute continues to invest in the same range.