When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters was with a 24-year-old patient whose sickle cell disease left them with almost weekly pain episodes.
- By HMS COMMUNICATIONS
"The encounter made me wonder, couldn't we do more for these patients?" said Sankaran, who is now the HMS Jan Ellen Paradise, MD Professor of Pediatrics at Boston Children's Hospital.
As a budding hematologist, Sankaran knew all too well that people with sickle cell disease - marked by malformed, sickle-shaped red blood cells that can aggregate and block small vessels - experience excruciating pain crises, tissue and organ damage, and shortened life expectancy.
He also understood that the only treatment available at the time was hydroxyurea, which reduces sickling but isn't effective in all patients and can cause side effects. The only chance at a cure was to undergo a bone marrow transplant, available to only a small percentage of patients because it carries significant risks and requires a well-matched donor.
Sankaran's rotations through the hematology clinic made him want to change the story of the disease, both at the bedside as a soon-to-be physician and by joining the laboratory of HMS alumnus Stuart H. Orkin, the HMS David G. Nathan Distinguished Professor of Pediatrics at Boston Children's and Dana-Farber Cancer Institute.
In 2008, Orkin, Sankaran, and colleagues achieved their vision by identifying a new therapeutic target for sickle cell disease.
