DUARTE>> Dozens of doctors, scientists, businesses and others gathered at City of Hope Thursday for the second California Institute for Regenerative Medicine (CIRM), Alpha Stem Cell Clinics symposium.

CIRM, the states stem cell agency, has developed a network of Alpha Stem Cell Clinics that focus on innovative stem cell-based therapies. The network of three clinics are located at City of Hope, University of California, San Diego, and UCLA/UC Irvine campuses.

The event served as a way for clinics to share their most recent advancements and successes in stem cell therapy clinical trials, and even hear directly from patients who benefited from some of the trials.

We want to review the trials, but we also want to see what other questions we should be asking, said Dr. John Zaia, the Aaron Miller and Edith Miller Chair in Gene Therapy, and director of the Center for Gene Therapy and principal investigator of City of Hopes Alpha Stem Cell Clinic. How will insurance companies charge or pay for these treatments? How do companies plan to develop these treatments? The symposium provides an opportunity to think about these other aspects.

There were also panel speakers who offered more of a motivational talk, such as Pat Furlong, founding president of Parent Project Muscular Dystrophy, discussing how to remove stakeholder barriers to stem cell therapy treatment.

Furlong had to become her and her sons own advocate when they were diagnosed with Duchenne Muscular Dystrophy at a young age. She found there was no standard of care for the disease and no studies or trials in progress to find a treatment, let alone a cure.

Families just didnt know the questions to ask, she said. At the time, few people cared about rare diseases.

After years of no real hope and losing her sons at 15 and 17 years old, with her and her groups persistence, Furlong said there are now 40 companies researching the disease and millions of dollars have gone into research specifically for Duchenne.

City of Hopes Dr. Behnam Badie, chief of neurosurgery and director of the Brain Tumor Program, and Christine Brown, Ph.D., Heritage Provider Network Professor in Immunotherapy and associate director of the T Cell Therapeutics Research Laboratory, discussed their recent successful treatment of a patient with recurrent multifocal glioblastoma using CAR-T cell therapy.

The case study for this unique type of immunotherapy on the most aggressive form of brain cancer was published in the Dec. 29 edition of the New England Journal of Medicine.

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In the Phase I clinical trial, the patient, who did not respond to other types of therapy including radiation and even developed tumors in his brain and spinal cord, was treated with his own genetically modified chimeric antigen receptor (CAR) T cells, injected directly into the tumor and through the ventricular system. The patient experienced remission over 8 months.

City of Hope is one of a few cancer centers in the nation offering studies in CAR-T cell therapy, and is the only cancer center investigating CAR-T cells targeting the specific receptors more common in a majority of glioblastomas.

Dr. Badie and Brown noted that working with CIRM has been instrumental in helping them along with their trial, and not just the funding.

You cant create a good trial without studying the product, said Brown. These are expensive trials. We have to treat these patients and understand what is going on.

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Symposium discusses latest and future of stem cell therapy - The Pasadena Star-News