“On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition,” reports the Times of India:
For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s months-long medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments. For a lucky few, like Kendric, the treatment could make possible lives they have longed for.
A solemn and shy adolescent, he had learned that ordinary activities — riding a bike, going outside on a cold day, playing soccer — could bring on episodes of searing pain. “Sickle cell always steals my dreams and interrupts all the things I want to do,” he said. Now, he feels as if he has a chance for a normal life.
Near the end of last year, the FDA gave two companies authorization to sell gene therapy to people with sickle cell disease — a genetic disorder of red blood cells that causes debilitating pain and other medical problems. People are born with the disease when they inherit the mutated gene for the condition from each parent.
The treatment helped patients in clinical trials, but Kendric is the first commercial patient for Bluebird Bio, a Somerville, Mass., company.
In other news, gene therapy is now restoring vision for some people with inherited blindness. A British toddler has had her hearing restored in a pioneering gene therapy trial: “Opal Sandy was born unable to hear anything due to auditory neuropathy, a condition that disrupts nerve impulses traveling from the inner ear to the brain and can be caused by a faulty gene. But after receiving an infusion containing a working copy of the gene during groundbreaking surgery that took just 16 minutes, the 18-month-old can hear almost perfectly and enjoys playing with toy drums.”
A new gene therapy blocks the painful hereditary condition angiodema. A virus is being used to cure deafness in new gene therapy. Researchers also discovered that a plant virus could be used to save crops from root-eating pests. A genetically-modified chicken lays eggs that are non-allergenic, which could make it possible for people with egg allergies to eat eggs.
