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United to Conquer Sickle Cell Disease

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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.

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 to treat SCD.

Stepping Forward

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.

    • More goals

Progress

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.

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.
  • Improve understanding of vascular system changes and mechanisms of organ damage in SCD.
  • 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.

Why it matters

There is no widely available cure for SCD and few effective treatments.
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