Research and Clinical Trials
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.
- 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.