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ASH President: NIH Cure Sickle Cell Initiative “Bold and Visionary”

(WASHINGTON, September 13, 2018) —The National Institutes of Health today announced the launch of the Cure Sickle Cell Initiative, a collaborative research effort to accelerate the study of curative gene therapies for sickle cell disease (SCD).

The following statement is from American Society of Hematology (ASH) President Alexis A. Thompson, MD, MPH, of the Ann and Robert H. Lurie Children’s Hospital of Chicago:

“SCD is a chronic and debilitating genetic blood disease for which there are few treatments and curative options. The only cure available outside of clinical trial is a bone marrow transplant, a procedure that replaces the blood-forming stem cells in the bone marrow with healthy cells, but this therapy is not available to everyone. Gene therapy allows us to correct mutations in the patient’s own blood stem cells that are then infused back into the patient. While gene therapy for SCD is still experimental, it is extraordinary that to date more than a dozen clinical trial participants have been cured. These techniques have been highlighted by ASH in its Agenda for Hematology Research as one of the most promising avenues of study in hematology. Gene therapies must not only be efficacious, but they must also be durable, safe, cost-effective, and widely available.

“The Cure Sickle Cell Initiative is a bold and visionary plan to bring potential cures to the broader community of individuals living with SCD. The fact that the National Institutes of Health has taken on this important challenge sends a powerful message to individuals with SCD and their health care providers that moving promising science from the laboratory bench to the clinic is a real priority for the United States government. 

“ASH enthusiastically supports the Cure Sickle Cell Initiative and is committed to being a productive partner to the National Heart, Lung, and Blood Institute (NHLBI), which is leading the effort. The Society has also made an unprecedented commitment to conquering SCD on all fronts – from investment in research to improving access to care in the United States and in developing countries. ASH has launched an SCD research data registry and will launch a clinical trials network, which is expected to be complementary to the Cure Sickle Cell Initiative by accelerating the further exploration of these therapies once they are ready for late stage clinical trials. Next month, ASH will co-host a clinical endpoints workshop with the U.S. Food and Drug Administration that aims to bring uniformity and standards to existing SCD clinical trials. On other fronts, ASH is developing clinical practice guidelines and expanding health care professional education and training on SCD. The Society also launched the Sickle Cell Disease Coalition, a group of organizations including NHLBI, which was formed to amplify the voice of SCD and will continue to support this initiative.

“SCD remains a serious public health issue, but efforts from ASH, NIH, and other partners are making unprecedented progress toward improving the state of care for a population that has been underserved. The future for individuals with SCD is bright.”

The American Society of Hematology (ASH) (www.hematology.org) is the world’s largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 50 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. ASH publishes Blood (www.bloodjournal.org), the most cited peer-reviewed publication in the field, which is available weekly in print and online. In 2016, ASH launched Blood Advances (www.bloodadvances.org), an online, peer-reviewed open-access journal.

The FDA-ASH Sickle Cell Disease Clinical Endpoints Workshop is entirely funded by charitable gifts to the ASH Foundation, a division of the American Society of Hematology.

Amanda Szabo, American Society of Hematology
[email protected]; 202-552-4914