UCSF Benioff Children's Hospital Oakland has received its largest-ever research trial grant for a study that will use gene-editing technology in patients with sickle cell disease.
The four-year study will be the first to use CRISPR-Cas9 technology in humans.
UCSF Professor of Pediatrics, Dr. Mark Walters, is the principal investigator of the clinical trial and gene-editing project.
"What we're going to do is remove a small portion of the blood system, change its programming to no longer make sickle red blood cells but healthy red bloods cells, and then return them to the circulation where they'll begin to grow and build new blood cells," Dr. Walters told KCBS Radio's "As Prescribed" on Thursday.
He said the first phases will focus on safety and seek to find out whether cells will behave normally once they are back in the body's circulation.
Working together with researchers at UCLA and UC Berkeley, the UCSF researchers will enroll nine patients for the trial.
"We will enroll, initially, adults who are having a lot of difficulty related to the sickle cell disease, who we believe would die of the disorder if not treated in a way such as this one," Walters added.
If successful, the team hopes to make the treatment accessible and affordable to the people who need it. Patients could effectively become their own donors instead of having to rely on blood transfusions and bone marrow transplants from a suitable match.
Access to care remains a problem for many who suffer from sickle cell disease, which primarily affects African-Americans.
"A hallmark of the disease is pain and it’s treated with opioid medications," Walters said. "So there has been over the years in emergency departments this assumption that somebody coming in seeking pain medications is faking it or has an addiction."
Walters explained the $17 million is a welcome public investment on novel therapies for the disease.
"Finally an opportunity to tackle the core of the problem, which is why don't we just correct the mutation that makes all these changes in the red blood cells in the stem cells from which they grow?"
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