Stem Cell Therapy Summary

The Childrens Hospital Los Angeles Stem Cell Project is uniquely positioned in California to apply embryonic and adult stem cell technologies as therapies for many childhood and adult diseases.  The project goals align with the purpose of Proposition 71, the California Stem Cell Research and Cures Initiative, approved by California voters on November 2, 2004. 

As a result, the Saban Research Institute of Childrens Hospital Los Angeles has received an initial three-year, $2.4 million stem cell training grant from the California Institute for Regenerative Medicine (CIRM).  Childrens Hospital Los Angeles is the only pediatric institution in California awarded a stand-alone CIRM training grant. 

Investigators at The Saban Research Institute are working with human embryonic stem cells (hESC) and are focused on developing methods to culture and genetically manipulate hESC and to direct their differentiation to blood, pancreas cells, liver and lung cells.  In addition, investigators are studying adult stem cells from bone marrow, cord blood, lung, liver and mesenchymal tissue.  Other research is exploring amniotic fluid stem cells, which may have the potential to form many tissues of the body.

Childrens Hospital Los Angeles is in the forefront of gene therapy research.  As part of this research, investigators found that therapeutic effects can be achieved by administering cord blood-derived CD34+ cells that have been genetically engineered to encode a therapeutic agent gene.  This method has been applied to treating newborn infants suffering from ADA deficiency.  Critical to gene therapy is the development of safe vectors for delivering therapeutic genes to cells.  Childrens Hospital Los Angeles is a leader in retroviral and lentiviral vector development, and has proprietary modifications that increase the safety of these vectors.

The Gene, Immunology and Stem Cell Therapy Program at Childrens Hospital Los Angeles is led by Donald B. Kohn, MD, who has been an innovator in the field of gene therapy using stem cells for the past 15 years and is an expert in the design and development of gene delivery vectors.  Dr. Kohn led the group that performed the first clinical trial of gene therapy for newborn infants with severe combined immune deficiency ("bubble-baby disease").  His laboratory studies how gene transfer to stem cells can be used to influence how adult and embryonic stem cells grow, differentiate and generate organ-specific tissues, with a particular focus on pancreatic beta cells.  Dr. Kohn has been the recipient of the Elizabeth Glaser Scientist Award from the Pediatric AIDS Foundation (1995-2000) and a Distinguished Clinical Scientist Award from the Doris Duke Charitable Foundation (2000-2005).  He is past president and an advisory board member of the American Society of Gene Therapy.  Other lead investigators include: Gay Crooks, MD, (the director of the Childrens Hospital Los Angeles Stem Cell Project), who has been studying human stem cells in the bone marrow and umbilical cord blood for 14 years. In 2004, she was made a Stohlman Scholar of the Leukemia Lymphoma Society for her work with human hematopoietic (blood-forming) stem and progenitor cells; and Paula Cannon, PhD, who works on developing new gene delivery vectors for stem cell modification.