Last week, a woman named Victoria Gray became the first person in the U.S. to have her cells edited with CRISPR. The 41-year-old patient was suffering from sickle cell anemia.
The condition, caused by a genetic mutation that messes with the shape of red blood cells, causes havoc on patients, and to make things even worse, the options for treatment are very limited and ineffective. The only current treatment for sickle cell anemia patients is a donor transplant that works for just 10% of patients, but all that is about to change.
A first in the U.S.
It was clear that an alternative, much more effective solution was desperately needed. After much consideration, doctors believed that editing cells extracted from a patient's own bone marrow could restore effective red blood cell creation, and this is exactly the operation they attempted on Gray.
The doctors used CRISPR to tweak Gray's bone marrow DNA to turn on a specific protein that would allow proper red blood cell generation. The operation makes Gray the first person in the U.S. to undergo a CRISPR editing procedure and the second globally.
A treatment from the 1940s
The treatment comes from observations made back in the 1940s. “In 1941 a pediatrician named Jane Watson noticed that babies with sickle cell didn’t have symptoms until 6 months to 1 year of age,” Vivien Sheehan, a hematologist at Baylor University told Popular Science.
The pediatrician also discovered that these infants produced fetal hemoglobin for much longer periods than healthy babies. Following Watson's observations, the research since then has indicated that increasing fetal hemoglobin could provide an effective treatment for the disease.
Now, CRISPR may just make that treatment viable. But before we get too excited, it should be noted that the strategy comes with several risks.
In order for the edited cells to be inserted back into the patient’s bone marrow, other stem cells need to be deactivated. Otherwise, there is the chance the unedited stem cells may continue to produce sickled red blood cells very fast, outpacing the edited cells' production of healthy cells.
Now researchers say they need to follow Gray's progress for at least 15 years to rule out any other potential dangers of the procedure. Still, for those 90% suffering with sickle cell anemia that don't respond well to current treatment, the procedure, if successful, would offer the much-needed lifeline they've been hoping for.