Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received a $4.6 million grant from the California Institute for Regenerative Medicine, known as CIRM.

The funds will support a phase 1 clinical trial of a treatment for advanced sarcomas and other cancers with a specific tumor marker called NY-ESO-1. The research, led by Dr. Theodore Scott Nowicki in collaboration with Dr. Antoni Ribas, will test a therapeutic approach that genetically engineers each patient’s own blood-forming stem cells to produce cancer-fighting immune cells called T cells.

Sarcomas account for 1% of all adult cancers and 15% of all pediatric cancers diagnosed in the U.S. Between 25% and 50% of sarcoma patients treated with conventional methods (surgery, radiation and chemotherapy) go on to develop metastatic disease, meaning the cancer spreads to other parts of the body, and people with metastatic sarcoma have very few treatment options — none of which has been proven to increase survival.  

“Immunotherapies hold promise in treating this cancer because they can empower the immune system to fight many different subtypes of the disease,” said Nowicki, a fellow physician in the UCLA David Geffen School of Medicine division of pediatric hematology/oncology.

Ribas is a professor in the UCLA David Geffen School of Medicine departments of medicine, surgery, and molecular and medical pharmacology, and a member of the UCLA Jonsson Comprehensive Cancer Center.    

The treatment platform being tested in the new study is already being deployed in a related trial focused on multiple myeloma, which launched in September 2017 and is led by Ribas, who is also director of the UCLA Parker Institute for Cancer Immunotherapy. That trial is funded by a $20 million grant from CIRM and support from the UCLA Broad Stem Cell Research Center.  CIRM has been supporting Ribas’ work since 2008, beginning with a $3.07 million New Faculty Award that enabled him to develop the technology to deliver T cell receptor genes into blood-forming stem cells.

The new grant was made at a CIRM meeting today at which two additional grants were made to projects involving UCLA faculty. Dr. Donald Kohn, a professor of pediatrics and microbiology, immunology and molecular genetics at the Geffen School of Medicine and a member of the Broad Stem Cell Research Center, is a collaborator on both grants. In one of those projects, Rocket Pharma Inc. will begin a clinical trial of a gene therapy for leukocyte adhesion deficiency, a rare genetic immune system deficiency. In the other, a UC San Francisco-led team is investigating a patient-specific stem cell treatment for sickle cell disease using the CRISPR-Cas9 genome editing tool. The team is seeking FDA approval to conduct a clinical trial.

Read more about the clinical trial being led by Nowicki and Ribas.