National Institutes of Health launches initiative to accelerate therapies to cure sickle cell disease

The National Institutes of Health (NIH) today announced the launch of a new initiative to help speed the development of a cure for sickle cell disease, a group of inherited blood disorders affecting at least 100,000 people in the United States and 20 million worldwide. The Cure Sickle Cell Initiative will take advantage of the latest genetic discoveries and technological advances to move the most promising genetic-based curative therapies safely into clinical trials within five to 10 years.

“Our scientific investments have brought us to a point where we have many tools available to correct or compensate for the defective gene that causes sickle cell disease. We are now ready to use these tools to speed up our quest for a cure,” said Gary H. Gibbons, M.D., director of NIH’s National Heart, Lung, and Blood Institute (NHLBI), which is leading the effort.

Sickle cell disease results from a single genetic mutation that causes a person’s red blood cells to form an abnormal, sickle shape. These sickled cells can clog the blood vessels and deprive cells of oxygen. In turn, this lack of oxygen wreaks havoc on the body, damaging organs, causing severe pain, and potentially leading to premature death.

Decades of basic research on sickle cell disease have laid the groundwork for novel genetic approaches to cures, such as the genetic editing of bone marrow cells, which have shown great promise in animal models and in some small-scale human studies. In addition, the NHLBI Production Assistance for Cellular Therapies (PACT) program has been working with researchers to manufacture cellular therapeutic products, including genetically modified cells, that can be used safely in clinical trials with patients.

NIH spends approximately $100 million on sickle cell disease research each year. Through this initiative, NHLBI seeks to support the development of cell and genetic therapies resources, clinical trials, comparator analyses of different management strategies, data repositories and resources, and patient and advocate engagement activities related to curative therapies for this condition. Already in 2018, NHLBI committed an additional $7 million to jumpstart the initiative’s research and engagement infrastructure.

NHLBI has named hematologist Edward J. Benz Jr., M.D., President and CEO Emeritus of Dana-Farber Cancer Institute, as the Initiative’s executive director, and the Emmes Corporation, a contract research organization with specialized expertise in clinical trials, gene, and cell therapy development in preclinical studies, as its coordinating center.

The initiative and other research partners will establish a national data warehouse of genetic therapies for sickle cell disease and conduct comparative analyses of therapeutic approaches to assess both clinical and cost-effectiveness. They will also establish national networks to make it easier for patients and providers to learn and engage with the research, clinical trials, and other activities happening across the country.

“The engagement of patients will be a cornerstone of this effort,” said Benz. “Patients will work alongside researchers in developing and recruiting for clinical trials.”

Currently, the only cure for sickle cell disease is a bone marrow transplant, a procedure in which a sick patient receives bone marrow from a healthy, genetically-compatible sibling donor. However, transplants are too risky for many adults, and only about 18 percent of children with the sickle cell disease have a healthy, matched sibling donor.

The Cure Sickle Cell Initiative seeks to develop cures for a far broader group of individuals with the disease, and it is initially focusing on gene therapies that modify the patient’s own hematopoietic stem cells (HSCs), which make red and other blood cells. These modified HSCs can then be given back to the patient via a bone marrow transplant, making a cure available to more patients who lack a matched donor.

“This initiative is giving patients, families, and communities a reason for hope. I’m particularly pleased that we are able to make this announcement during Sickle Cell Awareness month when we are shining a spotlight on the toll of this devastating disease,” said ADM Brett Giroir, M.D. Assistant Secretary for Health at the U.S. Department of Health and Human Services (HHS).

This patient-focused Initiative builds on the legacy of NHLBI-supported research that has contributed to improving clinical care for patients who have sickle cell disease. It also complements the Institute’s broader sickle cell disease research investment, which includes basic, clinical, translational, and implementation science research.

Part of the National Institutes of Health, the National Heart, Lung, and Blood Institute (NHLBI) plans, conducts, and supports research related to the causes, prevention, diagnosis, and treatment of heart, blood vessel, lung, and blood diseases; and sleep disorders. The Institute also administers national health education campaigns on women and heart disease, healthy weight for children, and other topics. NHLBI press releases and other materials are available online at http://www.nhlbi.nih.gov.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

NIH...Turning Discovery Into Health

North Carolina Central University Awarded $16.3 Million by National Institute of Health

North Carolina Central University (NCCU) has been awarded $16.3 million by the National Institute on Minority Health and Health Disparities (NIMHD), part of the National Institutes of Health (NIH), for a new Research Center in Minority Institutions (RCMI) focusing on elevating the university’s health disparities research program. Led by Deepak Kumar, Ph.D., director of the Julius L. Chambers Biomedical/Biotechnology Research Institute and principal investigator for the grant, the funding will support health disparities research activities across campus at NCCU.

NCCU is one of the seven minority-serving institutions that were recipients of this award. The research funding is the largest annual grant amount received for a non-Title III grant by NCCU and the largest funding for a single principal investigator on the campus.

The new RCMI Center for Health Disparities Research (RCHDR) will conduct three innovative basic biomedical and behavioral research projects, along with health disparities research pilot projects, involving robust mentoring, development of core facilities and leveraging of resources and partnerships with community-based organizations and neighboring institutions in the Research Triangle area. The center will also promote a collaborative research environment conducive to career enhancement for postdoctoral trainees and NCCU faculty at all levels.

“This major research grant will allow North Carolina Central University to engage in transformative research that examines health disparities and identifies real-world solutions that strengthen health care for minority populations throughout our state,” said University of North Carolina President Margaret Spellings. “Moreover, the establishment of the new Research Center in Minority Institutions supports our shared goal of enhancing research opportunities, which will ultimately improve the quality of life of our citizens and generate economic growth.

Johnson O. Akinleye, Ph.D., chancellor of NCCU, said, “The significant research funding provided by the National Institutes of Health elevates North Carolina Central University’s noteworthy work investigating solutions to some of the nation’s toughest health disparities that persist as challenges to communities of color. Through this grant, I am thrilled that Dr. Kumar and his team are already fulfilling three of the six strategic priorities represented in ‘The Eagle Promise’initiative, which focus on expanding research, facilitating innovative strategic partnerships with the Research Triangle Park and building new infrastructure for the university.”

The mission of NCCU’s Research Center in Minority Institutions is to develop and strengthen the research infrastructure at NCCU for conducting cutting-edge health disparities research and to foster the next generation of minority biomedical researchers. It has three main objectives: 1) enhance the research capacity at NCCU within the areas of basic, behavioral and translational biomedical research; 2) diversify the biomedical research workforce and to prepare researchers who are successful extramurally funded health disparities investigators and 3) promote a collaborative environment for interdisciplinary research and establish sustainable relationships with neighboring research and community-based organizations to advance cutting-edge health disparities research at NCCU. The core of the research center will focus on: African-American men, stress, kidney and cardiometabolic disease; breast cancer disparities and metabolic stress; diet-induced obesity and metabolic syndrome; community engagement and pilot projects and mentoring.

“NCCU demonstrated its commitment to biomedical research by creating two biotechnology research institutes, BBRI and BRITE,” stated Kumar. “We are grateful to NIH/NIMHD for providing NCCU with this unprecedented opportunity to further enhance biomedical research by developing infrastructure, preparing the next generation of minority researchers and bringing faculty together in cutting-edge, interdisciplinary research projects to advance our mission of addressing health disparities. The grant will foster collaborations and leverage resources from research and community-based organizations from the Research Triangle area and in North Carolina. This is a team effort across campus that will impact five NCCU colleges and schools, along with BBRI and BRITE. I am excited about Chancellor Akinleye’s ‘The Eagle Promise’ initiative and am grateful for his support and vision.”

In a press release announcing the award, NIMHD Director, Dr. Eliseo Pérez-Stable said: “Institutions with historical commitment to diversity are essential to supporting scientific research and providing healthcare to underserved communities. These institutions are uniquely positioned to engage minority populations in research, and in the translation of research advances into culturally competent, measurable and sustained improvements in health outcomes.”