What?
Following a long tradition of advancing research in sickle cell disease, the National Heart, Lung, and Blood Institute (NHLBI) launched the Cure Sickle Cell Initiative in September 2018.
The Initiative is a collaborative, patient-focused research effort. It aims to transform the lives of individuals living with sickle cell by accelerating the development of genetic therapies to cure this devastating disease within five to 10 years.
The Initiative intends to work with patients, caregivers, clinicians, academic researchers and industry to identify and support the most promising genetic therapies.
Why?
While progress has been made to treat and prevent the complications of sickle cell disease and individuals living with the disease have an increased life expectancy, they still face significant health challenges and diminished quality of life.
With new advancements in genetic therapy approaches, the time is right to drive toward cures that are more accessible to the approximately 100,000 Americans, and more than 20 million people worldwide, who have sickle cell disease.
How?
The Cure Sickle Cell Initiative will actively engage the sickle cell disease community of patients, family members, caregivers, advocates, and researchers to work together on a path to cures. The Initiative will support enhanced clinical trial recruitment as well as increased capacity to safely manufacture cellular therapy products. The Initiative will also support research within academia and industry to identify the most promising cellular and genetic therapies.
The Cure Sickle Cell Initiative will build on the legacy of research that has greatly improved clinical care of individuals who have sickle cell disease. It will also complement broader sickle cell disease research efforts in basic, clinical, translational, and implementation science.
In the News
FDA grants CRISPR gene therapy fast track designation for sickle cell disease (04JAN2019)
Bipartisan bill aimed at fighting sickle cell disease signed into law by Trump (19DEC2018)
NIH Director's Blog (11DEC2018)
Gene therapies could transform the treatment of sickle cell disease (03Dec2018)
A Catalyst for Cures (09Nov2018)
ASH President: NIH Cure Sickle Cell Initiative “Bold and Visionary” (13Sept2018)