General FAQs

General

General
What is the Cure Sickle Cell Initiative?

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 goal is to move these potential cures into first-in-human clinical trials in the next five to ten years.

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How does the Cure Sickle Cell Initiative work?

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.

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What are some of the cures that the Cure Sickle Cell Initiative is targeting?

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.

There are several genetic therapy approaches in early stages of development.   The Initiative aims to bring the most promising ones to U.S. Food and Drug Administration (FDA) review and approval to begin enrolling patients in clinical trials within five to ten years.

The Initiative will also accelerate the development and production of new medicines to increase treatment options for patients around the world. This includes patients who live in areas where environmental conditions may require very stable medicines, and patients for whom genetic therapies may be too risky.

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Researcher FAQs

Researcher

Researcher
What is the Cure Sickle Cell Initiative?

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 goal is to move these potential cures into first-in-human clinical trials in the next five to ten years.

MORE
How does the Cure Sickle Cell Initiative work?

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.

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Why are you launching this effort now?

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 within the next five to ten years.

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.

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How is the Cure Sickle Cell Initiative being funded?

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)”. NHLBI expects to be able to fund promising new scientific opportunities over the next few years.

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Why are you using this mechanism to fund this effort?

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.

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Have other agencies used OTA?

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.

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How much funding is involved? Is there any new funding?

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. Already NHLBI has committed $7 million to jumpstart the initiative’s research and engagement infrastructure.

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What are some of the cures that the Cure Sickle Cell Initiative is targeting?

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.

There are several genetic therapy approaches in early stages of development.   The Initiative aims to bring the most promising ones to U.S. Food and Drug Administration (FDA) review and approval to begin enrolling patients in clinical trials within five to ten years.

The Initiative will also accelerate the development and production of new medicines to increase treatment options for patients around the world. This includes patients who live in areas where environmental conditions may require very stable medicines, and patients for whom genetic therapies may be too risky.

MORE
What has been the interest from industry in developing new sickle cell treatments? How many manufacturers are there to join in?

There are several pharmaceutical companies that have already expressed interest in working with us.

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