Georgia woman says new sickle cell treatment may be a 'gamechanger'
Drug aims to tackle the root cause of sickle cell disease
Sickles Cell disease can cause anemia, pain and land patients in the hospital. A new drug could offer some relief.
Atlanta - Children's Healthcare of Atlanta treats more than 2,000 kids and young adults each year with sickle cell disease.
That is where 24-year-old Tiffany George of Woodstock got early access to a breakthrough treatment for sickle cell.
George was born with the genetic blood disorder, but it took a few years for her to understand how it would affect her.
"I think I realized at a pretty young age, when I was hospitalized," George says. "Something that stands out to me specifically is, in third grade, when I was hospitalized over and over again in the same year, when I finally realized, 'Oh, this is a disease that is serious and that keeps me from doing things other kids can.'"
George would sometimes end up in the ER at Children's Healthcare of Atlanta having a pain crisis.
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The problem was her red blood cells, which carry oxygen to her organs and tissues.
Healthy red blood cells are typically round and flexible, like a doughnut.
In people with sickle cell disease, the cells can become crescent- or sickle-shaped, clumping together and blocking smaller blood vessels, which can trigger intense pain and other complications.
"Thankfully, my pain isn't too extreme," George says. "I've been very fortunate, where I will only have a couple of pain crises a year."
Sickle cell is especially common in children of color.
Of 100,000 US cases, 90% are in people of African descent.
Pediatric hematologist Dr. Clark Brown, director of sickle cell clinical research at the Aflac Cancer and Blood Disorders Center at Children's Healthcare of Atlanta, says the disorder affects hemoglobin, a protein in the red blood cells that transports oxygen to the body and carries carbon dioxide back to the lungs.
"People who are born with sickle cell will have a problem with their hemoglobin, in that it gets thick inside the cells and forms these rigged rods, which destroys the life of the cell," Dr. Brown explains. "And they experience a lot of problems, such as fatigue, pain, and also problems leading to damage to their internal organs."
At Children's, George joined a clinical study involving a breakthrough sickle cell treatment known as Oxbryta, or voxelotor.
Dr. Brown, who led the study at Children's, says Oxbryta zeroes in on the root cause of sickle cell: the sickling process, slowing down the breakdown of the red blood cells.
"The molecule binds the hemoglobin to make it more flexible so that the red blood cell isn't destroyed as quickly in the body," Brown says. "When we talk about sickle cell, they're anemic. On this drug, their anemia improved as well as their energy level and (there were) less complications."
Tiffany George initially was in the placebo group in the study.
Later, she was switched to Oxbryta, which involved taking 3 pills a day.
"Since taking the real one, I've definitely noticed a difference of just feeling more healthy altogether," George says.
George is no longer taking the medication but feels it was helpful in heading off pain crises.
Oxbryta is now FDA-approved for treating sickle cell disease in adults down to children age 4 and older.
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