New hope for those living with Sickle Cell Disorder thanks to experimental gene treatment
A Sickle Cell Disease patient discusses how a new gene therapy is giving her hope for the future. A new gene cell therapy has been approved by the U.S. Food and Drug Administration to treat Sickle Cell Disorder patients. Victoria Goray, a patient of the disease, is seeing results and hopes to inspire others with the disease. The Centers for Disease Control and Prevention reports that over 90% of patients with the disorder are African American. The condition causes severe pain, drops oxygen levels, and causes trouble breathing. Goray is undergoing a unique treatment to cure these symptoms. The FDA approved in 2023 the first cell-based gene therapies that target the flawed DNA in blood stem cells. However, the treatment isn't without risk.
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Published : 10 months ago by Jorge Estevez in Health
ATLANTA — There’s new hope for those living with Sickle Cell Disorder thanks to a unique new treatment approved by the U.S. government.
The treatment is a gene cell therapy recently approved by the U.S. Food and Drug Administration.
For the first time, we’re seeing results.
For Victoria Goray, the new treatment helps her and makes her want to inspire others living with the disease.
The U.S. Centers for Disease Control and Prevention, based in Atlanta, says more than 90% of patients born with the blood illness are African American.
Now, for patients that can fight off the disease into adulthood, there may be a sanctuary of better health.
Sickle Cell Disease can cause a number of health complications, according to the CDC.
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“In my case, it induces severe pain and this takes away your ability to do regular things like hold cups or walk,” Goray said.
She’s had to overcome some long odds. According to the National Heart, Lung and Blood Institute, one in ever 365 African American babies are born with Sickle Cell Disease.
The condition causes red blood cells, which carry life-giving oxygen through the body, the be misshapen, virtually useless, and bringing the person with those misshapen cells immense pain.
“It’s severe pain, it drops your oxygen levels, you have trouble breathing,” Goray said.
Now, Goray is undergoing a unique treatment to cure those symptoms.
In 2023, the FDA approved the first cell-based gene therapies that target the flawed DNA in blood stem cells. Then they go back into the pa tient, but the treatment isn’t without risk.
“July 2, I received what I like to call my super cells,” Goray said. “I received one transfusion while waiting, because I didn’t have an immune system.”
For patients like Goray, there’s still care needed after treatment, whether with experimental therapy or for other care management procedures.
That’s where the Sickle Cell Sanctuary comes in.
Tabatha McGee, the Executive Director, hopes to bless sickle cell patients with the care they need.
“They have not been taken care of by urgent care, they have felt not heard, not seen,” McGee said. “Inside of the sanctuary, we have holistic services, mental health counseling and therapies, nutrition and exercise counseling.”
Goray hopes to take the blessing of her super cells and turn it into a super blessing for others.
“I wanna be that light that I needed when I was going through,” Goray said.