Scientists in California have received approval from the U.S. Food and Drug Administration for a trial that could be life-changing for about 100,000 Americans.
Sickle cell disease is a blood disorder that causes debilitating pain and can shorten life span by 30 years. It primarily affects in people of African descent.
Doctors from UCSF, UC Berkeley and UCLA are conducting the first in-human clinical trial that uses CRISPR technology in patients with the genetic disorder.
Dr. Mark Walters is a professor of pediatrics at UCSF and principal investigator of the clinical trial and gene-editing project. He told KCBS Radio's "As Prescribed" on Thursday the process begins with the collection of blood stem cells from a person with severe sickle cell disease.
"These are the mother or seed cells that give rise to all the blood cells in the circulation," he explained. "So, we take these outside body, we introduce our CRISPR gene editing reagents, and, like a needle in the haystack, they find that one change in the DNA code of the blood stem cell that corrects the sickle mutation."
Those new cells are returned to the circulation to make room for healthy new cells. Because the change is made at the blood stem cell level, the benefit should be lifelong after the one-time treatment.
The therapy could be a game-changer.
Currently, medications used to treat the symptoms of sickle cell disease do not cure the disorder. In certain cases, a bone marrow transplant could provide a cure but this requires a healthy donor who has the same transplant type, usually a sibling of the patient.
"It turns out very few of our patients have a suitable donor, so a cure is not readily available," said Dr. Walters. "This would advance the field by allowing persons to act as their own donor. So, theoretically, everyone who wanted to get the treatment could, if in fact it were safe, effective, affordable, and widely-accessible."
Developing a cure that is affordable and widely-available is a big part of the challenge, he added.
"How do we ensure that people have access to it where they live - and not just in the United States and other rich countries in the world - but where the disease is really common in less wealthy countries around the world."
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