Sickle cell disease treatment nearing FDA approval

UCI Health expert says progressive condition impairs quality of life and longevity

IN THE NEWS: A potential cure for sickle cell disease in the form of a genetic therapy called exa-cel is nearing approval by the Food and Drug Administration.

UCI Health hematology expert Dr. Zahra Pakbaz, told Healthline that there is an urgent need for new treatments like the gene-editing therapy, which prevents red blood cells from forming the problematic “sickle,” the crescent shape that makes it hard for them to move through the blood vessels and leads to serious health conditions.

“This is a devastating disease. Pain crises are unexpected and recurrent, and in many cases, pain is chronic and does not go away. There is plenty of data showing sickle cell disease has impaired the quality of life and life expectancy of individuals with this condition, regardless of which country they live in.”

Pakbaz is a board-certified hematologist with UCI Health Blood Disorder Services and Hematology Oncology Services. who specializes in non-cancer blood disorders. Her clinical interests include thalassemia, sickle cell anemia, iron overload, clotting disorders and disorders of low or high counts of platelets and white or red blood cells. She is a member of the American Society of Hematology and the American Oncology Association of Southern California.

Pakbaz has published more than 60 research papers and presentations on sickle cell anemia, thalassemia and iron metabolism. Her findings have led to improved outcomes for sickle cell and thalassemia patients. She has received numerous awards for her contributions to sickle cell research and care.

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