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 Cure Sickle Cell Initiative is a collaborative, patient-focused research effort designed to accelerate promising gene 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), CureSCi aims to advance the most promising gene therapies safely into clinical trials.

The NHLBI-led Cure Sickle Cell Initiative fosters a collaborative, patient-focused research environment that identifies and supports the most promising and innovative gene therapies to cure sickle cell disease. To that end, NHLBI engages patients, families, advocates, and researchers from academia and the pharmaceutical industry, to leverage resources. The Initiative’s support is in addition to NHLBI’s broader sickle cell disease research investment that aims to improve the lives of people living with sickle cell disease.

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.

The 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 point where a focused, collaborative and coordinated approach that includes academia and industry researchers, clinicians, patients, and advocacy groups will help us find sickle cell disease.

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

The U.S. Food and Drug Administration approval of Casgevy and Lyfgenia, the first cell-based gene therapies for the treatment of sickle cell disease, has the potential to provide a life-changing and disease-modifying outcome for individuals living with sickle cell disease. Both of these therapies, which use different ways to change the expression of an individual’s genes to make more fetal hemoglobin, show the importance of providing patients with options and not relying on one single curative option. NHLBI and CureSCi continue to advance gene therapy clinical trials for sickle cell disease and to ensure that they are safe, effective, and last over time.

CureSCi activities extend beyond the implementation of clinical trials, and leadership is working to address related areas that impact patients. For example, efforts are focused on safety and risks, addressing long-term follow up related to SCD gene therapies, best practices to evaluate patient readiness and resilience for gene therapy trials, mental health needs of the community, and cost effectiveness of gene therapies, among others.

A recent analysis of the cost effectiveness of gene therapy for SCD supported by CureSCi [*LK3 - Dharti: see note below ]  has found that gene therapies will likely be a cost-effective option compared to conventional treatments given during a patient’s lifetime.

NHLBI has partnered across the Federal government to address the cost and coverage of curative therapies, working closely with Centers for Medicare & Medicaid Service (CMS), for example, to increase the accessibility of gene therapies for Medicaid enrollees living with SCD. NHLBI is also working with CMS to identify and reduce barriers to uptake and to support readiness of patients and clinicians for gene therapies.

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 supporting the development of cell therapy resources, gene therapy clinical trials, comparator analyses of different management strategies, data repositories and resources, and patient and advocate engagement activities related to curative therapies. NHLBI has committed approximately $92 million so far to the Initiative’s research and engagement infrastructure.