Blood Disorders and Blood Safety
Research Making a Difference
Research Making a Difference
WHY IT'S IMPORTANT
NHLBI has major research responsibilities in blood diseases. One is to ensure the adequacy and safety of the Nation's blood supply. A second is to support efforts to treat and cure blood disorders, such as scientific advances in stem cell biology and new gene and cell-based therapies to repair and regenerate human tissues and organs. By funding and supporting research on the causes, prevention, and treatment of blood disorders, we help researchers make new discoveries, which lead to better medical care for the millions of people affected by blood disorders around the world.
- The NHLBI-led Cure Sickle Cell Initiative is a patient-focused research effort to develop genetic therapies for use in clinical research within 5 to 10 years.
- NHLBI-sponsored research has helped and continues to help Americans with sickle cell disease live longer and well in into adulthood.
- NHLBI-supported scientists developed life-saving treatments for patients with hemophilia and are now working on gene therapy approaches to cure the disease.
- NHLBI-funded research helped develop therapies that increased the survival rates for patients who have aplastic anemia.
OPPORTUNITIES & CHALLENGES
In 2016, NHLBI released its Strategic Vision, which will guide the Institute’s research activities for the coming decade. Many of the objectives, compelling questions, and critical challenges identified in the plan focus on blood biology, blood disorders, and blood safety. Training the next generation of blood scientists is also a high priority for NHLBI.
- Blood Disorders and Blood Safety
Researchers are working across scientific disciplines to advance blood disorders research. NHLBI is assisting in this effort by enabling early translational research to accelerate the development of new clinical interventions. Currently, we are supporting research resource programs that provide regulatory, pharmacology, toxicology, and manufacturing services to NHLBI investigators who are doing blood science research. The hope is for patients with blood disorders to live long and full lives, and to improve blood safety measures both in the United States and around the world.
Advancing the Research
NHLBI is advancing blood disorders and blood safety research in many ways. Learn more about some of our key efforts related to sickle cell disease, anemia, blood transfusions, and more.
We Fund Research
The research we fund today will help improve our future health. Our Division of Blood Diseases and Resources is a leader in research on the causes, prevention, and treatment of blood diseases other than cancers of blood cells. NHLBI’s Division of Cardiovascular Sciences also supports some blood science research.
The Promise of Precision Medicine
Through NHLBI’s Trans-Omics for Precision Medicine (TOPMed) program, researchers will use data from studies focused on heart, lung, blood, and sleep disorders to better predict, prevent, diagnose, and treat blood disorders based on a patient’s unique genes, environment, and molecular signatures. Learn more about NHLBI precision medicine activities.
Accelerating Cures for All Patients Who Have Sickle Cell Disease
The Cure Sickle Cell Initiative is a NHLBI-led collaborative research effort to develop genetic therapies for patients who have sickle cell disease. The goal is to have these genetic therapies ready to safely use in clinical research within five to 10 years. The Initiative is patient-focused, and it will bring together researchers, industry, patients, providers, advocacy groups, and others as it supports research, education, and community engagement activities.
Improving Care for Adolescents and Adults with Sickle Cell Disease
While most U.S. children with sickle cell disease survive to adulthood, the transition from pediatric to adult care is often challenging. By funding the Sickle Cell Disease Implementation Consortium, we are working to understand current barriers to care, test interventions to overcome those barriers, and develop a new sickle cell disease registry.
Funding Global Health Research Efforts
More than 75 percent of infants with sickle cell disease are born in sub-Saharan Africa. By funding the Sickle Cell Disease in Sub-Saharan Africa Collaborative Consortium and associated Data Coordinating Center, we are building the regional capabilities to research sickle cell disease and monitor patients in Africa. These efforts may ultimately improve treatment and care for Americans who have sickle cell disease.
Improving Blood Safety and Availability
The NHLBI started the Recipient Epidemiology and Donor Evaluation Study (REDS) program in 1989 to protect the Nation's blood supply from threats, improve the benefits of transfusions, and reduce the risks of transfusions. REDS is the largest multi-center research program of its kind in the United States, and after 30 years, it is entering a new, important phase that aims to evaluate and improve the safety and effectiveness of transfusion therapies with attention not only to adults but also to understudied populations, including newborns and children.
Addressing Issues in Blood and Marrow Transplantation
The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) was established to conduct large multi-institutional clinical trials to understand the best possible treatment approaches in blood and marrow transplantation. In the United States, nearly 21,000 patients receive blood or marrow transplants annually, mainly for rare blood disorders.
Supporting Safe Manufacturing of Cell-based Therapies
The NHLBI’s Production Assistance for Cellular Therapies (PACT) supports translational research on cellular and genetic therapies by increasing the capacity to manufacture cell products that follow current Good Manufacturing Practices (cGMP) regulations. PACT offers education and resources through its five Cell Processing Facilities and a Coordinating Center that support activities such as safety testing, product shipment, and design of clinical testing protocols.
Studying Underlying Factors in Severe Trauma Cases
The Trans-Agency Research Consortium for Trauma-Induced Coagulopathy (TACTIC) research program was established in collaboration with the Department of Defense to study the disruptions in the normal process of blood clotting that occurs in individuals who experience severe trauma. Trauma, particularly severe trauma, can disturb the body’s mechanisms for stopping hemorrhage or lead to the overproduction of blood clots, both of which can cause major or even fatal complications.
Establishing Publicly Available Resources for Scientists
Myelodysplastic syndromes (MDS), a collection of rare disorders that for unknown reasons leads to defects in blood stem cells, affects at least 10,000 adults each year in the United States. The National MDS Natural History Study is a joint NHLBI and National Cancer Institute study that aims to create a resource of high quality biospecimens linked to clinical data that scientists can use to conduct research to improve diagnosis and treatment of MDS.
Providing Access to NHLBI Biologic Specimens and Data
The Biologic Specimen and Data Repository Information Coordinating Center (BioLINCC) centralizes and integrates biospecimens and clinical data that were once stored in separate repositories. Researchers can find and request available resources on BioLINCC's secure website, which maximizes the value of these resources and advances heart, lung, blood, and sleep research.
Supporting scientists performing NHLBI-funded gene therapy research
Research on genetic therapies is part of our broader commitment to advancing scientific discovery aimed at developing safe and effective treatments for heart, lung, and blood disorders and diseases caused by faulty genes. Our National Gene Vector Biorepository and Gene Therapy Resource Program help investigators transform early-stage research into genetic therapies. These programs equip scientists with the tools, resources, safety testing services, and animal models they need to advance genetic therapy research from the laboratory into clinical trials.
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