LOS ANGELES (May 30, 2012) Patricia Dickson, M.D., principal investigator at The Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed), is co-principal investigator of a project that was just awarded a $5.5 million grant from the California Institute for Regenerative Medicine (CIRM). The goal of the project is to develop a stem cell based therapy for the treatment of mucopolysaccharidosis I (MPS I), a fatal pediatric lysosomal storage disease that causes neurodegeneration as well as defects in other major organ systems. Dr. Dickson is working with lead investigator Philip H. Schwartz, Ph.D., senior scientist at the CHOC Children's Research Institute and managing director of the facility's National Human Neural Stem Cell Resource.
For nearly a decade, Dr. Dickson's research at LA BioMed has focused on enzyme replacement therapy for mucopolysaccharidosis, a group of metabolic disorders caused by the absence or malfunctioning of enzymes needed to break down molecules - called glycosaminoglycans - which help build bone, cartilage, connective tissue and other essential parts of the body. As part of this study, she and her colleagues will begin with proof-of-principle experiments for MPS I.
"Dr. Dickson has been at the forefront of mucopolysaccharidosis research for many years, working tirelessly to help develop therapies for MPS I," said David I. Meyer, Ph.D., president and CEO of LA BioMed. "We congratulate her on her continued success, and for her role in this project which could be an important breakthrough for children suffering from neurodegenerative disorders."
"The unique aspect of this research is that it uses a single donor for the transplantation of stem cells into the body and the brain, which allows the best treatment for both physical and neurological disease and avoids rejection of neural stem cell grafts by the host immune system," said Dr. Dickson. "Pediatric neurodegenerative diseases are generally neglected in stem cell research, but stand the greatest chance of success. The high probability of success, along with the need to alleviate suffering in children, is why we believe the first applications of stem cell therapies should be for these kids."
Dr. Schwartz, Dr. Dickson and project collaborators are working to address two critical issues in the development of a therapeutic candidate based on stem cells: that early intervention is not only required but is indeed possible in this patient population, and that the concept of immune tolerance is also required, where the immune system is trained to attack only real threats in the body but not the body's own cells or tissues.
To date, there is still a significant unmet medical need to better impact and prevent the neurodegenerative processes in this disease. If successful in MPS I, this technique can be expanded to help treat other neurodegenerative disorders due to lysosomal storage.
|Contact: Diana Soltesz|
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed)