Thanks to funding from Proposition 71, California’s $3 billion investment in stem cell research, and the state funding entity it created, the California Institute for Regenerative Medicine (CIRM), there are now 36 human clinical trials in various stages of progress, including a trial for sickle cell disease.
Condition: Sickle Cell Disease
Accepting Applicants: Yes
Status: Phase 1 trial in progress
Description: Clinical trial of stem cell gene therapy for Sickle Cell Disease
CIRM Funding Brief: “The stem cell therapy approach to be developed by this Disease Team will be used to treat patients with SCD by transplanting them with their own bone marrow adult hematopoietic stem cells that are genetically corrected by adding a hemoglobin gene that blocks sickling of the red blood cells. This approach has the potential to permanently cure this debilitating and common illness with significantly less toxicity than with a bone marrow transplant from another person.” Read more at CIRM
Current Sponsor: UCLA
Principal Investigator: Donald Kohn, MD
Institution: UCLA
Phase 1/2 Filed: May 2014
Phase 1/2 Approved: June 2014
Cell Therapy Type: Gene therapy + blood-forming stem cells
NCT No. / Link: NCT02247843
Completion Date: Projected April 2018
Last Updated: December 20, 2016
As this clinical trial moves forward, we will update this page. Check back here for future developments.
As scientists and patient advocates build on the progress that Proposition 71 has enabled, we must keep the momentum going, understanding that there is still much work to be done. We must remember that human trials will celebrate successes; but, barriers will surface, along with complications and challenges, so patience and understanding of the scientific discovery process is essential. Even the setbacks will provide critical knowledge that will bring us closer to curing or mitigating devastating diseases and injuries. Only with continued support for biomedical research can we increase our understanding of the therapeutic potential of stem cells, and translate that understanding into meaningful treatments that help give patients their lives back.