Innovative Fanconi Anemia Research

In partnership with our adult hospital and Stanford University, we are conducting FA research of all kinds. In partnership with our Stanford Center for Definitive and Curative Medicine (CDCM), we are also performing cell and gene therapy research, from the initial discovery through clinical trials, with the hope to cure the incurable. We’ve sped up the research-to-treatment process, empowering our clinicians to innovate faster than ever before, and we offer several novel treatments for FA that arise from our research.

Fanconi anemia clinical trials

Please contact us to learn if your child may be a good fit for one of the clinical trials at Stanford, including:

• Phase I/II gene therapy for Fanconi anemia subtype A: preventative stem cell treatment to maintain the blood and immune system
• ALDH activator FP-045: drug that may help maintain stem cell function
• Antibody-based conditioning using JSP191: method of preparing your child’s body before a stem cell transplant instead of radiation/chemotherapy to make transplants safer
• TCR alpha/beta T-cell/CD19 B-cell depleted haploidentical stem cell transplant with standard conditioning: method of adjusting donor’s cells to make them less likely to attack your child’s body yet enabling the immune system to recover more quickly
• TCR alpha/beta T-cell/CD19 B-cell depleted hematopoietic cell transplant with antibody-based conditioning using JSP191: a combination of the two methods above