Historic grants to UCSF Benioff Children’s Hospital Oakland may end sickle cell disease, hospital officials said.
The hospital received $17 million in grants to launch a four-year study to use recently developed technology to alter the gene that causes sickle cell disease and eradicate the illness.
The disease is especially prevalent among Black people, and it is known for the pain it causes people and for early death. Blacks with the disease die roughly 30 years earlier than those without.
“Finding a cure means finding a future,” said 26-year-old Black patient Brooklyn Haynes.
A cure also means “finding a path that we can walk on that’s pain free,” Haynes said.
Researchers plan to use the recently developed CRISPR-Cas9 gene-editing technology on the blood stem cells of patients to correct the mutated gene that causes sickle cell disease.
Researchers are planning to enroll the first patient by mid-2022. Recruitment has started in Oakland and Los Angeles.
“The goal is to develop a cure that is not just safe and effective, but one that is affordable.”
Jennifer Doudna, 2020 Nobel laureate
First, up to six adults with the disease will be treated and if the treatment is safe and effective, three children 12 to 17 years old will be enrolled.
Researchers expect to treat seven patients in Oakland and two at the University of California at Los Angeles.
The study was approved earlier this year by the U.S. Food and Drug Administration, according to officials with the University of California at Berkeley.
It is the first clinical trial to apply CRISPR-Cas9 in humans to remove and replace the gene that causes sickle cell disease, likely curing the patient including the associated pain and early death, UC Berkeley officials said.
Sickle cell disease has no cure presently. Drugs and treatments manage the pain.
“The goal is to develop a cure that is not just safe and effective, but one that is affordable,” said Jennifer Doudna, a professor at UC Berkeley who shared the 2020 Nobel Prize in chemistry for her work on the CRISPR-Cas9 gene-editing tool.
Researchers don’t have an estimate for what a cure will cost patients or their families because the research is just beginning.
“There have been a lot of promising developments in recent years with CRISPR-based therapies for sickle cell and other genetic diseases, but it’s essential that we have studies like this that can help us create truly accessible cures,” Doudna added.