Kids as young as two can now access a landmark gene therapy for sickle cell disease, after regulators expanded approval of the breakthrough CRISPR treatment.
The FDA gave the green light on July 1 for Casgevy, a one-time treatment that edits a patient’s own blood stem cells, to be used on children with the disease’s most severe form – as well as kids with transfusion-dependent beta thalassemia. Until now, the treatment was only approved for patients 12 and older.
Sickle cell disease is a brutal, inherited blood disorder, most common among Black Americans, that bends red blood cells into a rigid crescent shape. Those misshapen cells clog blood vessels, triggering pain crises so severe that patients often describe them as feeling like their bones are breaking from the inside, along with strokes, organ damage and a shortened life expectancy.
Casgevy works by editing a patient’s own stem cells outside the body, then infusing them back in to switch on production of healthy fetal hemoglobin – the kind babies naturally produce before birth. In the trials that led to approval, nearly all patients treated went a year or more without a single severe pain crisis.
Lowering the eligible age to 2 means families won’t have to watch their kids endure a decade of crises before qualifying for treatment.
The therapy still isn’t simple. It requires chemotherapy to clear space in the bone marrow before the edited cells go in, and it’s currently only available at a limited number of certified treatment centers. But for families of the youngest, sickest patients, the FDA’s decision means the treatment clock now starts years earlier.
