Using Physical Chemistry and a High-Throughput Drug Screen to Find a Drug to Treat Sickle Cell Disease - On Demand

Presenter: Dr. Emily Dunkelberger, Staff Scientist, National Institutes of Health

Sickle cell disease is considered an orphan disease in the United States, but it affects millions of people worldwide, primarily in sub-Saharan Africa and India. The molecular basis for the disease is a point mutation on the gene that codes for the beta‐strands of hemoglobin. In the 1990s, the first and only anti‐sickling drug, hydroxyurea, was approved by the FDA.
 

Hydroxyurea induces the production of fetal hemoglobin, which dilutes sickle hemogoblin and slows or prevents hemoglobin aggregation upon deoxygenation. However, hydroxyurea only works for a portion of those affected by sickle cell disease and can come with many severe side effects. At NIH, scientists have found that about 10% of sickle cell patients can be cured by stem cell transplantation, but an inexpensive drug that can be given orally and distributed worldwide is needed.

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