Encouraging news that the technology can potentially cure a number of genetic disorders
Frangoul and other researchers caution, however, that the results involve just one patient who was only recently treated. It is far too soon to answer the most crucial questions: Will the modified-cell treatment continue to improve the patient's health? Will the treatment keep working? Will it help her live longer? Is it safe in the long term?
"We are hoping it is" a success, Frangoul says. But "it is still too early to celebrate."
NPR has exclusive access to chronicle the experience of Frangoul's patient, Victoria Gray of Forest, Miss., the first person with a genetic disease to be treated with CRISPR in the United States.
"So look at this," Frangoul said recently with a smile, as he showed Gray her latest blood test results. The testing indicated that the genetically modified cells had already started producing the crucial protein at levels doctors hope will alleviate her suffering.
"I am super-excited about your results today," Frangoul said.
In July, Gray was recovering from the medical procedure, which involved using an experimental technique called CRISPR to edit the genes of her own bone marrow cells.
While Gray knows it's still very early, she described how the treatment appears to be helping her. She has not suffered any of the painful attacks that torture sickle cell patients and has not needed to rush to the hospital for care since getting the modified cells this summer. She has not needed any blood transfusions either and has begun to reduce the pain medication she had been taking chronically.
"It's a miracle," says Gray, who says she has hope for the first time after a lifetime of struggling with excruciating pain and debilitating, life-threatening complications of the disease. Sickle cell disease is an inherited condition that is marked by defective oxygen-carrying red blood cells.