... NOT ALL GOOD NEWS, THOUGH

THE YEAR 2017 started with no gene therapies sold in the US and only a couple elsewhere. Then the Food and Drug Administration (FDA) approved the first CAR-T cell therapies, which alter a patient’s own blood cells to turn them into specialised cancer killers. They’re only for certain types of leukaemia and lymphoma now, but more are in the works for other blood cancers.

Last week, the FDA approved Luxturna, the first gene therapy for an inherited disease, a form of blindness. People with it can’t make a protein needed by the retina, tissue at the back of the eye that converts light into signals to the brain, enabling sight. The therapy injects a modified virus containing a corrective gene into the retina so the cells can make the protein.

Children who received the treatment told what it was like to gain vision.

“Oh yikes, colours. Colours are super fun,” said 13-year-old Caroline Carper of Little Rock, Arkansas. “And the sunshine is blinding.”

Gene therapies also showed some promise against a variety of diseases including haemophilia, a blood clotting problem; “bubble boy” disease, where a flawed immune system leaves patients vulnerable to fatal infections, and sickle cell disease, a serious and painful blood disorder common among black people.

It’s not all good news, though. The therapies don’t work for everyone. They’re shockingly expensive. And no one knows how long some results will last, though scientists say the aim is a one-time repair that gets at the root cause.