The Current State

The majority of available treatments manage symptoms of the disease, rather than treating the underlying cause of sickling.

Although the molecular basis of SCD was established more than half a century ago, it has been challenging to translate this research into the development of novel targeted therapies. While new approaches in managing this disease have improved diagnosis and supportive care over the last few decades, people with SCD still have severe complications to overcome.

Our Progress

SCDC Stepping Forward

In 2017, the U.S. Food and Drug Administration approved L-glutamine powder (Endari), the first new drug the Agency approved for SCD in 20 years.

90%+ of people with SCD today live well into adulthood, which poses new issues and challenges.

Stem cell transplantation has shown success in curing some individuals with SCD, but it is not widely available.

There are currently only two treatments approved by the FDA

Our short-term goals:

  • Develop, support, and sustain hemoglobinopathy clinical trial networks as well as a better infrastructure to better enroll and study people with SCD and share data, including in community settings (need collaboration for sufficient enrollment).
  • Investigate combination therapies and new drug-delivery models.
  • Develop means to stratify people with SCD according to course of disease, response to therapy, and disease progression.
  • Optimize dosing and treatment response predictors for hydroxyurea.
  • Optimize SCD transfusion therapy to increase biological understanding and identify principles for accurate blood matching.

Our long-term goals:

  • Conduct better longitudinal studies to understand the determinants of prolonged survival and cause of death in people with SCD.
  • Continue to study and refine genome-editing therapies with curative potential.
  • Support development and expansion of effective state strategies ("carve-outs") to enhance public insurance coverage and reimbursement.
  • Continue to invest in molecular research on SCD pathogenesis.
  • Create a validated, SCD-specific functional assessment tool for pain.
  • Extend genotype studies to better understand the spectrum of sickle hemoglobinopathies.
  • Improve SCD diagnostic methods.
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SCDC TAKES A STAND

Join the many and take a stand along side the SCD Coalition, and learn how you can fight against sickle cell disease. Download our recent SCDC Snapshot to see our current priorities for access to care, research & clinical trials, and much more.

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Access To Care

Access To Care

Learn how the SCDC is working to address social determinants of health and promote high-quality, coordinated systems of care for people impacted by SCD.

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Global Issues

Learn how the SCDC is working to improve outcomes for individuals living with sickle cell around the world, particularly in low- and middle-resource settings.

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