Oregon Health & Science University today announced the creation of the OHSU Center for Embryonic Cell and Gene Therapy, led by Shoukhrat Mitalipov, Ph.D., a world-renowned leader in embryonic stem cell and gene therapy research.
The center will allow Mitalipov and his team to accelerate their pioneering work, which over the last several years has opened up new routes that could lead to cures and treatments for Parkinson's disease, multiple sclerosis and a range of other conditions caused by diseased or injured cells in the human body.
"Our continuing work and discoveries can be revolutionary in how we cure and treat many diseases and injuries," said Mitalipov, who is a senior scientist at OHSU’s Oregon National Primate Research Center. "This new center will allow us to put together a comprehensive program where we can share our expertise, answer new questions and train the scientists needed to move this important work forward."
"The work that Dr. Mitalipov and his colleagues have accomplished in recent years has made them world leaders in cell and gene therapy," said Dan Dorsa, Ph.D., OHSU's senior vice president for research. "This center will give them a new foundation that will boost them to new levels of scientific discovery, and allow them to continue to lead for years to come."
OHSU’s announcement of the center comes after Mitalipov returned to Oregon last week from a two-day Food and Drug Administration hearing in Maryland that reviewed Mitalipov’s scientific work for consideration of human clinical trials in an area of gene therapy research where he is the U.S. scientific leader.
Mitalipov and his team have succeeded in preventing transmission of genetic defects in mitochondrial DNA in the cells of monkeys, in 2009, and in human cells in 2012. Thousands of babies are born every year in the U.S. with mutated mitochondrial DNA, which can cause devastating conditions and symptoms that include brain damage, muscle weakness, cardiac disease and damage to other organs. Most children with mitochondrial disease don't live past their teenage years.
Mitalipov said his discoveries mean that science is ready to begin to tackle the problem in humans.
"We are ready to move forward to clinical trials with this procedure," Mitalipov said. "We are ready to offer new hope to the thousands of families whose lives have been shaken by the tragic effects of these mutations."
Mitalipov and his team are also global leaders in embryonic stem cell research. Last year, the journal Cell published a Mitalipov paper that detailed how his team had reprogrammed human skin cells to become embryonic stem cells capable of transforming into any cell type in the body. Mitalipov's stem cell breakthrough was the culmination of six years of discoveries about the process in his lab.
Stem cell therapies offer the promise of replacing cells damaged through injury or illness — and could hold the key to treating Parkinson's disease, cardiac disease and spinal cord injuries, among other conditions. Mitalipov's procedure is among a very few alternatives to the controversial use of stem cells derived from fertilized human embryos. His lab is the only one in the world currently capable of producing these embryonic human stem cells.
While much of Mitalipov's work at OHSU has been supported by the National Institutes of Health, federal funding for human embryonic stem cell research is restricted. That means much of Mitalipov’s work is conducted under tightly regulated conditions that mandate a separate lab space for the embryonic stem cell research that is funded solely through private support. That’s why private philanthropy is so important for the continuation of his work.
“Only private investment will move this research forward,” said Dorsa. “Private funders need to step in where federal agencies cannot.”
The new Center for Embryonic Cell and Gene Therapy will allow for expansion of that private funding, and empower Mitalipov and his team to focus on making new discoveries and advances in stem cell and gene therapy.