Grants to develop treatments for AIDS, cancer and sickle cell disease were awarded by the California Institute for Regenerative Medicine to three UCLA stem cell research teams on Oct. 28.
The grants totaled $49.2 million and contain a four-year time line to go from preliminary research to human clinical testing.
The grants are aimed at creating less evasive and more effective treatments for some of the world’s most life threatening diseases.
The price tag is high, but for good reason, according to Dr. Judith Gasson, co-director of the UCLA Broad Center for Stem Cell Research. These research grants fund the entire path from early research to clinical trials in a tight time line.
The CIRM is the California state stem cell agency created by Proposition 71 in 2004. The agency was allocated $3 billion over 10 years to fund state stem cell and other biomedical research. UCLA has received about $122 million from the agency since 2005.
UCLA scores high in stem cell research funding compared to other universities, Gasson said, ranking second in overall funding from CIRM after Stanford University.
“The people of California want to see progress in real time,” she said. “They want to see treatments for life threatening diseases as quickly and safely as they can be developed.”
Dr. Donald Kohn, director of the UCLA Human Gene Medicine program and professor of microbiology, immunology, molecular genetics and pediatrics, was awarded one of the grants. He received $9.2 million to develop a treatment to sickle cell disease.
Sickle cell disease is an illness where patients have a mutated hemoglobin gene, causing red blood cells to have an issue with getting through blood vessels.
The standard practice to treat the disease is by conducting a bone marrow transplant. Transplants are evasive, according to Kohn. He said he was developing a treatment that involved fixing the stem cells in the hemoglobin gene, with no transplants necessary.
Typical research funding is usually in hundreds of thousands of dollars, Kohn said. This grant is much higher because the money would go to salaries of the researchers under him, as well as preliminary stem cell studies and development of a treatment for clinical trials.
Kohn emphasized a series of strict deadlines the research teams had to follow. He said that if the team doesn’t meet the four-year deadline for submission of the treatment to the Food and Drug Administration, the money could be cut.
“Even though we have this four-year award, (the institute) can stop it at any time,” he said. “They do very scrupulous oversight of all of the grants they’ve given out ““ much more than any other agency I’ve dealt with.”
Only four of the 14 research grants involve embryonic stem cells ““ the rest of the research grants are focused on adult stem cells.
Adult stem cells are specialized cells found in everyone, in contrast to embryonic stem cells, which that have not yet transformed into a specialized tissue.
Research on embryonic stem cells has historically garnered controversy because the cells are found in infant embryos.
Kohn said the use of embryonic stem cells in research was incredibly promising. Researchers will theoretically be able to turn the embryonic stem cells into any type of cell in the entire body, but he noted that the research just isn’t ready for clinical trials yet.
“The adult stem cells have one job,” Kohn said. “They are a lawyer but they can’t be a doctor, or they are a reporter but they can’t be a physicist. The embryonic ones can be any of the above when we learn how to do it.”
Dr. Dennis Slamon received a nearly $20 million disease team grant to discover new treatments for cancer. Slamon, the director of Clinical/Translational Research at UCLA’s Jonsson Comprehensive Cancer Center, said that his research is focused on getting to the root of cancer cells.
His theory is that cancer stem cells are driving the growth of the tumor. His grant will fund research to develop drugs treating those cells. The overall goal would be to stop the growth of the cancer.
The ultimate goal is getting new and innovative medicines into the clinic, Kohn said. He said these grants are a step in the right direction.