At the age of 45, Dr. Lakiea Bailey, who serves as the executive director of the nonprofit patient advocacy group known as the Sickle Cell Consortium, has often considered herself the eldest person she knows living with sickle cell anemia. Diagnosed with sickle cell disease at the tender age of three, her life has been marked by numerous challenges, including heart issues, hip replacements, and enduring chronic pain.
Dr. Bailey recently addressed the independent advisory committee of the US Food and Drug Administration, sharing her optimism about a groundbreaking therapy currently undergoing evaluation. She believes that this therapy offers the sickle cell community something they have long yearned for – hope.
During her address to the FDA committee on Tuesday, Dr. Bailey expressed her belief in the emergence of hope, offering a potential transformation in the lives of those who have endured excruciating pain for far too long.
The independent committee is helping the FDA think through how it should evaluate a treatment called exa-cel that could potentially cure people of sickle cell disease, a painful and deadly disease with no universally successful treatment. This was an ongoing discussion. There was no vote or decision about the therapy, but the discussion likely moves the US one step closer to approving a groundbreaking new treatment that uses gene editing. the executive director
If approved, exa-cel, made by Boston-based Vertex Pharmaceuticals and the Swiss company CRISPR Therapeutics, would be the first FDA-approved treatment that uses genetic modification called CRISPR.
CRISPR, or clustered regularly interspaced short palindromic repeats, is a technology researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function and develop.