The UCSF Benioff Children’s Hospital Oakland recently received $17 million — in grants of $8.4 million and $8.6 million — to launch a four-year clinical trial looking to eradicate sickle cell mutation, using CRISPR-Cas9 gene-editing technology, a first-ever process applied in humans.
The grants are from the California Institute for Regenerative Medicine and the National Institutes of Health (NIH) Cure Sickle Cell Initiative, supported by the National Heart, Lung, and Blood Institute (NHLBI).
In a letter to colleagues, hospital president Matthew Cook wrote that the money is the “largest research trial grant ever awarded to UCSF Benioff Children’s Hospital Oakland” and continues a decades-long commitment to advancing new treatments and eliminating health disparities tied to this debilitating genetic blood disorder, which disproportionately impacts Black and Latino communities. On the recent Giving Tuesday, the hospital partnered with television station KTVU, raising $175,000 in donations.
The Cure Sickle Cell Initiative is a long-awaited dream for those living with sickle cell disease. In 2018, the NIH launched the initiative to help accelerate cures for sickle cell disease. The goal is to advance the development of the latest gene- and cell-based therapies safely into clinical trials for sickle cell disease within five to 10 years, fund research across the academia and private sectors to identify the most promising cellular and genetic therapies, and partner with federal agencies, academic institutions, pharmaceutical companies, professional societies, community advocacy organizations,
patient representatives, and foundations. The initiative also seeks to educate and
engage all stakeholders, especially patients.
Sickle cell disease is a genetic condition that affects the body’s red blood cells and occurs when a child receives a sickle cell gene from each parent discovered at birth during routine newborn screening tests. Healthy red blood cells are round and move through small blood vessels carrying oxygen to all parts of the body. However, in sickle cell disease, the blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle,” says the U.S. Department of Health and Human Services Office of Minority Health.
The result is that people living with sickle cell disease suffer mild to unimaginable severe pain that can last for a few hours to a few weeks and occurs suddenly. They also suffer other serious health problems such as bacterial infections, hands, and feet swelling, often along with a fever. Additional severe symptoms include eye diseases, acute chest syndrome, and stroke.
The only cure for the disease is a bone marrow or stem cell transplant. However, both options come with serious risks and require a close donor match, such as a sibling. CRISPR-Cas9 gene-editing technology will use patients’ own blood stem cells to correct the mutated gene that causes the debilitating disorder.
In a recent virtual sickle cell webinar, hosted by the California and Maryland chapters of the National Coalition of 100 Black Women, the call to action is to ask elected officials to support sickle cell legislation, donate and designate bone marrow to sickle cell research, access genetic counseling to know status and risk factors, and get tested for the sickle cell trait.
Earlier this year, U.S. Reps. Barbara Lee and Danny K. Davis, co-chairs of the Congressional Sickle Cell Disease Caucus, and U.S. Rep. Michael Burgess reintroduced bipartisan legislation to address sickle cell. At least 3 million people in the United States have the sickle cell trait, leading to sickle cell disease, including 1 in 12 African Americans and 1 in 100 Latinos.
We must build on the momentum of developing a safe, effective, and accessible cure for those suffering with sickle cell disease.
— The Vacaville author is a social issues advocate. E-mail: email@example.com
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