6 months ago
23
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.
For the estimated 20,000 people with sickle cell in the US who qualify for the treatment, the start of Kendric's monthslong journey may offer hope.
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 authorisation 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. Another other company, Vertex Therapeutics of Boston, declined to say if it had started treatment for any patients with its approved CRISPR gene-editing-based remedy.
Kendric began his treatment at Children's National Hospital in Washington. Wednesday's treatment was only the first step. Doctors removed his bone marrow stem cells, which Bluebird will then genetically modify in a specialised lab for his treatment. That will take months. But before it begins, Bluebird needs hundreds of millions of stem cells from Kendric, and if the first collection - taking six to eight hours - is not sufficient, the company will try once or twice more. If it still doesn't have enough, Kendric will have to spend another month in preparation for another stem cell extraction. nyt
