The California Institute for Regenerative Medicine (CIRM) awarded two scientists at UC San Diego $2 million each for their innovative research. The first award went to Alysson R. Muotri, PhD, professor in the UC San Diego School of Medicine departments of Pediatrics and Cellular and Molecular Medicine. The funding will support studies of new treatments for Zika. The second award went to Dan Kaufman, MD, PhD, professor of medicine in the Division of Regenerative Medicine. This funding will support the creation an “off-the-shelf immunotherapy” using NK cells to treat refractory or resistant tumors, such as ovarian cancer.
Zika has been a major concern within the American medical community for some time. Though the mosquitoes carrying the virus used to be confined to Central and South America, now they are being found within the borders of the United States. The virus is known to cause birth defects, including microcephaly. In adults, the virus can cause a variety of disorders of the immune system.
One of the CIRM award will be used to study anti-viral drugs developed for other infectious diseases as a possible treatments for Zika.
Though to date, they only have preliminary findings in cellular and animal models, the team reports promising results, suggesting that some FDA-approved drugs may work to fight the virus.
While researchers continue their efforts to develop a Zika vaccine to prevent infection, there is a need for treatment options for those already infected. In an article for the UC San Diego News Center, Dr. Muotri says, “There is urgent need to move as quickly as we can into clinical trials and, hopefully, find an effective treatment. This is especially true of infected mothers where a Zika infection during the first trimester of pregnancy appears to pose the greatest risk of congenital microcephaly.”
The goal of the second grant award is to create an immunotherapy using NK cells to treat refractory or resistant tumors.
The recipient of this CIRM award, Dr. Kaufman, has developed an efficient process to produce NK cells from induced human pluripotent stem cells (iPSC) at his laboratory.
“NK cells are part of the normal immune system and are known to kill certain tumors and virally-infected cells,” said Dr. Kaufman in the same UCSD News Center article. “Here, we are using the advantages of iPSCs to make NK cells with improved anti-cancer activity.”
He goes not to explain that, “Unlike current immunotherapies produced on a patient-specific basis, iPSC-derived immune cells can be targeted to tumors with high specificity, no off-target effects and without need for patient matching.” said Kaufman.
Another aspect of Kaufman’s research, is the optimization of iPSC-derived NK cells to kill leukemia cells. Kaufman and colleagues will conduct animal studies with the goal of translating findings to clinical therapies for cancers that would otherwise prove lethal.
CIRM was created in 2004 by California voters with $3 billion in funding support to accelerate stem cell research and treatments. Since that time, UC San Diego researchers have received at least 90 CIRM awards, totaling more than $169 million.