Stem Cell Scientists Awarded $28.7 Million to Pursue Clinical and Basic Biology Research

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$20 Million Grant to Develop New Melanoma Treatment and Test it in Clinical Trials

A team of scientists led by Dr. Antoni Ribas, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and a professor of medicine, were awarded a $20 million grant from the state’s stem cell agency (CIRM) to take leading-edge stem cell science from the laboratory and translate it into a new treatment for metastatic melanoma, a deadly form of skin cancer.

The four-year grant is part of CIRM’s Disease Team Initiative, which seeks to explore new ways to integrate and organize the highest quality basic, translational and clinical research with the aim of developing new therapies and diagnostic tools. As part of the approval process, disease teams must submit an investigational new drug application, or IND, to the U.S. Food & Drug Administration within four years in an effort to fast-track stem cell-related drug development.

Dr. Ribas and his team propose to develop an IND and launch a Phase I clinical trial to genetically engineer a patient's immune response to specifically attack the melanoma starting from their blood stem cells. The specialized immune cells use a protein called the T-Cell Receptor (TCR) to seek out and recognize the melanoma. Evaluation of immune system reconstitution, effectiveness and immune response during treatment will be monitored using Positron Emission Tomography scans.

$8.7 Million Awarded to Stem Cell Researchers in Clinical & Basic Biology Grants from the State Stem Cell Agency

Dr. Stanley Nelson, a scientist with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, was awarded a $6 million grant from CIRM to develop a combination therapy for Duchenne Muscular Dystrophy (DMD), a potentially fatal disease that currently has no effective treatment.

Two other scientists with the Broad Center, Drs. Owen Witte and Kathrin Plath were awarded more than $2.7 million dollars in two grants to investigate basic mechanisms underlying stem cell biology and differentiation.

Nelson’s Early Translational award and the two Basic Biology IV grants –totaling $8.7 million –continue efforts by CIRM to support research designed to take stem cell science from the laboratory to the clinic. The studies supported by these awards will form the foundation for translational and clinical advances, enabling realization of the potential of human stem cells for therapies and as tools for biomedical innovation.

To date, UCLA and its stem cell scientists have received 57 grants from CIRM totaling more than $182.7 million.