September is Sickle Cell Awareness Month, and this year it arrives with a milestone: the first gene therapy for the disease has reached young children.
According to the U.S. Food and Drug Administration, on July 1 it expanded approval of Casgevy — the first treatment built on the Nobel Prize-winning CRISPR gene-editing tool — to children as young as two, having previously cleared it only for patients 12 and older.
Sickle cell disease, an inherited disorder that most often affects Black Americans, bends red blood cells into a crescent shape that clogs blood vessels and triggers bouts of severe pain. Casgevy edits a patient’s own stem cells to switch back on a form of hemoglobin normally made only before birth, keeping the cells from sickling. The one-time treatment is demanding — it requires intensive chemotherapy to prepare the bone marrow — and costly, at around $2.2 million.
The change makes an estimated 5,500 more American children eligible, reaching them, doctors note, before years of organ damage set in.
Allyson Good curates senior living, health, and family content. She is formerly the Managing Editor of Active Life, a print newspaper published in 2016-2017 for seniors, choosing practical articles on health, money, safety, and everyday living for older readers. She is also an avid gardener and offers up Garden Hints and Tips on a regular basis.