Happy Birthday, CIRM! 10 years, 10 new stem cell therapies

November 28, 2014 | by Nicole White

stem cell research City of Hope joined other leaders in stem cell research to celebrate 10 years since voters approved funding for the California Institute for Regenerative Medicine, or CIRM.

On its 10th birthday, the California Institute for Regenerative Medicine celebrated 10 stem cell therapies that have been approved for clinical trials, including an HIV/AIDS trial at City of Hope.

At the recent anniversary event at USC’s Broad Stem Cell Center, scientific leaders in the development of stem cell therapies for a range of diseases and conditions – such as AIDS, cancer, diabetes and blindness – reflected on the accomplishments of the last decade and thanked voters for their investment in research aimed at pushing the frontiers of medicine.

City of Hope has used its $55 million in CIRM funding to participate in every facet of stem cell research, including basic science, translational medicine, clinical trials and training the next generation of scientists in stem cell biology. John Zaia, M.D., the Aaron D. Miller and Edith Miller Chair in Gene Therapy and chair of the Department of Virology, said during his remarks at the event that CIRM also has encouraged academic institutions to partner with biotech companies to achieve results.

“CIRM has really taught us discipline in approaching these kinds of accomplishments,” Zaia said, using as an example City of Hope’s collaboration with Sangamo Biosciences.

In that collaboration, City of Hope is working with the company on research using zinc finger nuclease – which acts like a pair of molecular scissors to precisely edit genes – in a strategy to create a functional cure for HIV. This approach prevents the body from producing a key protein the virus requires to infect cells. It has been developed by investigators at City of Hope working with Sangamo Biosciences and Keck School of Medicine at USC.

Because of this collaboration, Zaia said, he already knows of 25 potential patients eligible for an upcoming clinical trial of the approach. The trial is one of the 10 CIRM has highlighted. In the study, patients' own blood-forming stem cells will be modified to produce immune cells that cannot be infected by the virus.

Also, a recent $8 million CIRM award will allow City of Hope to open its Alpha Clinic for Cell Therapy and Innovation. Already, City of Hope scientists are investigating two different means of altering stem cells to fight AIDS; one of those is the Sangamo collaboration.

The other approach wields a combination of stem cell and gene therapy using small ribonucleic – or RNA – molecules that block the genes HIV needs to infect immune cells, specifically T cells. Developed by John Rossi, Ph.D., Lidow Family Research Chair and chair of the Department of Molecular and Cellular Biology, this approach aims to spur to spur the immune systems to produce T cells resistant to HIV by infusing the patient with these altered stem cells.

“We think with the expertise we already have, we will know how to take these nascent therapies from our Alpha clinic and make them into sustainable methods of treatment for disease,” Zaia said.

City of Hope is also exploring the use of neural stem cells – which naturally home to cancer cells – as a delivery platform for chemotherapy drugs for brain cancer, another treatment that will be offered through the Alpha clinic.

Robert Klein, architect of the ballot initiative that led to CIRM’s creation, praised the scientists and physicians behind the more than 2,500 peer-reviewed articles published in the last decade and the 7 million voters who approved the initiative.

“The stem cell work being done in California benefits the whole world,” he said. “California, where the voters have vision; Where the future of medicine is changing through the will and genius of its people and its scientists, who are dedicating their lives to that end.”

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