At 16, She’s a Pioneer in the Fight to Cure Sickle Cell Disease

Changing Lives

At 16, She’s a Pioneer in the Fight to Cure Sickle Cell Disease 

Gina Kolata
Source: The New York Times 

 For more than a half century, scientists have known the cause of sickle cell disease: A single mutation in a gene turns red blood cells into rigid crescent or sickle shapes instead of soft discs. These misshapen cells get stuck in veins and arteries, blocking the flow of blood that carries life-giving oxygen to the body and causing the disease’s horrifying hallmark: episodes of agony that begin in babyhood.  

Millions of people globally, a vast majority of them Africans, suffer from sickle cell disease. Researchers have worked for decades on improving treatment and finding a cure, but experts say the effort has been hindered by chronic underfunding, in part because most of the estimated 100,000 people in the United States who have the disease are African-American, often poor or of modest means.  

 Like many others affected by sickle cell, the Obando family faced a double whammy: not one but two children with the disease, Helen and her older sister, Haylee. They lived with one hope for a cure, a dangerous and sometimes fatal bone marrow transplant usually reserved for those with a healthy sibling as a match. But then they heard about a potential breakthrough: a complex procedure to flip a genetic switch so the body produces healthy blood. 

Read full story here.

Helen Obando, 16, is the youngest person ever to get a gene therapy that scientists hope will cure the disease, which afflicts 100,000 Americans.”