The California Institute for Regenerative Medicine (CIRM) has awarded $6.3 million in grants to City of Hope researchers for research and development of stem cell-based therapies to treat HIV/AIDS, Parkinson’s disease and Canavan disease, an often-fatal neurological disease that affects infants.
|David L. DiGiusto (Photo by Markie Ramirez)|
David L. DiGiusto, Ph.D., research professor in the Department of Virology, is leading the development of a new gene therapy for HIV infection. Larry A. Couture, Ph.D., senior vice president of the Sylvia R. & Isador A. Deutch Center for Applied Technology Development, is collaborating with the Buck Institute for Age Research in Novato, Calif., to develop a treatment for Parkinson’s disease. Yanhong Shi, Ph.D., associate professor in the Department of Neurosciences, is leading collaborative work with the University of Bonn in Germany into Canavan disease, which has no standard treatment.
“We are pleased to have the innovative and promising translational research of our faculty at City of Hope advanced by the California Institute for Regenerative Medicine,” said Richard Jove, Ph.D., Morgan and Helen Chu Director’s Chair of Beckman Research Institute of City of Hope. “These grants are critically important to medical research and we are very fortunate to have funding in California for stem cell-based therapies.”
CIRM’s latest grants target translational research to support preclinical testing of potential treatments. Scientists must perform this rigorous laboratory research before an investigational therapy can reach patients through clinical trials. The National Institutes of Health reports that insufficient funding prevents most investigational therapies from making it through these preclinical testing stages, so CIRM grants can be critical to early stem cell research.
City of Hope pioneered research into stem cell-based treatment of HIV, beginning in the 1990s, using hematopoietic cell transplantation (HCT) to treat patients with HIV and AIDS-related lymphoma. Researchers now are investigating the use of blood stem cells engineered with short strands of ribonucleic acid (siRNA) that may provide new immune cells resistant to HIV infection.
“Our study builds on previous work at City of Hope that successfully demonstrated the ability of siRNA therapy to produce HIV-resistant cells in HCT transplant patients that prevent virus infection, replication and spread,” said DiGiusto. “With promising results from our trials of our first-generation therapy, we are looking to develop a second-generation therapy that will improve the production of HIV-resistant cells.”
|Larry A. Couture (Photo ©2007 Philip Channing)|
Couture is collaborating with the Buck Institute to develop an efficient process to manufacture the volume of specialized neurons needed for clinical testing in the treatment of Parkinson’s disease. Technologists will create the cells through City of Hope’s Center for Applied Technology Development.
“We are very excited about the opportunity to combine our expertise in translational science and biological manufacturing with the stem cell research facilities at Buck Institute,” said Couture.
Canavan disease is a rare neurodegenerative disorder that causes progressive damage to brain cells. It occurs in one in 6,400 Americans and currently has neither treatment nor cure. Infants born with the disease usually experience mental retardation, loss of motor skills and feeding difficulties, which often lead to an early death.
Shi’s research in collaboration with the University of Bonn focuses on turning a patient’s own skin cells back into stem cells that could potentially generate corrective neural cells to help treat the disease.
|Yanhong Shi (Photo by Darrin S. Joy)|
“There is a possibility that this treatment approach can be developed into the first effective therapy,” Shi said. “While we still have years of research and development ahead, what a wonderful day it would be if this research leads to a new treatment so that no parents will have to watch their child suffer.”
City of Hope has received more than $36.7 million from CIRM since the institute was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities and other vital research opportunities.