Benioff Children's Hospital Oakland receives $17 million to launch innovative clinical trial

UCSF Benioff Children's Hospital (BCH) Oakland has received the largest research trial grant in its history to launch an innovative clinical trial that aims to cure sickle cell disease. The trial, which plans to enroll its first patient later this year, will use CRISPR-Cas9 gene-editing technology on patients' own blood stem cells to correct the mutated gene that causes the debilitating disorder.

BCH Oakland researchers have been awarded $17 million to support the four-year trial, in two grants of $8.4 million from the California Institute for Regenerative Medicine (CIRM) and $8.6 million from the NIH Cure Sickle Cell Initiative, supported by the National Heart, Lung, and Blood Institute (NHLBI).

This trial is the first to apply CRISPR-Cas9 technology in humans and is part of a UC research consortium led by UCSF with UCLA and UC Berkeley. Researchers are recruiting patients for treatment in Oakland and Los Angeles, beginning with up to six adults with sickle cell disease. If the technology is found to be safe and effective, the trial will expand to enroll three adolescents ages 12 to 17. Seven patients are expected to be treated in Oakland and two at UCLA.