Scientists bring stem cell science directly to patients

Published on December 13, 2013 at 1:51 AM · No Comments

Scientists from UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research are bringing stem cell science funded by the California Institute of Regenerative Medicine (CIRM), the state stem cell agency, directly to patients in two exciting new clinical trials scheduled to begin in early 2014. The recipients of the Disease Team Therapy Development III awards were Dr. Dennis Slamon and Dr. Zev Wainberg, whose phase I clinical trial will test a new drug that targets cancer stem cells and has been approved to begin enrolling patients in the US and Canada, and Dr. Donald Kohn, whose first-in-human trial is on stem cell gene therapy for sickle cell disease (SCD).

The announcement of the new awards came on December 12, 2013 at the meeting of the CIRM Independent Citizen's Oversight Committee (ICOC) at the Luxe Hotel in Los Angeles. Dr. Owen Witte, Director of the UCLA Broad Stem Cell Research Center, highlighted that the "The CIRM support demonstrates that our multidisciplinary Center is at the forefront of translating basic scientific research to new drug and cellular therapies that will revolutionize medicine."

Targeting solid tumor stem cells
The Disease Team III grant to Dr. Dennis Slamon and Dr. Zev Wainberg and their US-Canadian collaborative team will support the first in human clinical trial scheduled to open in early 2014. The project builds on Dr. Slamon's previous work partially funded by CIRM to develop a drug that targets tumor initiating cells with UCLA's Dr. Zev Wainberg, assistant professor of hematology/oncology and Dr. Tak Mak, director, Campbell Family Institute of the University Health Network in Toronto, Canada. Dr. Slamon, renowned for his research that led to the development of Herceptin, the first FDA-approved targeted therapy for breast cancer, is the director of clinical and translational research at the UCLA Jonsson Comprehensive Cancer Center, and professor, chief and executive vice chair for research in the division of hematology/oncology.

With investigational new drug approval from the Food and Drug Administration (FDA) and Health Canada, the Canadian government's therapeutic regulatory agency, this trial is an international effort to bring leading-edge stem cell science to patients.

"We are delighted to receive this CIRM grant that will drive our translational research from the laboratory to the clinic," Slamon said, "and allow us to test our targeted drug in a phase I clinical trial."

The trial is based on the evidence built over the last decade for what has become known as the cancer stem cell hypothesis. According to this hypothesis, cancer stem cells are the main drivers of tumor growth and are also resistant to standard cancer treatments. One view is that cancer stem cells inhabit a "niche" that prevents cancer drugs from reaching them. Another view is that tumors can become resistant to therapy by a process called cell fate decision, by which some tumor cells are killed by therapy and others become cancer stem cells. These cancer stem cells are believed to be capable of self-renewal and repopulation of tumor cells, resulting in the recurrence of cancer.

The target of the new drug is an enzyme in cancer stem cells and tumor cells called Polo-like kinase 4, which was selected because blocking it negatively affects cell fate decisions associated with cancer stem cell renewal and tumor cell growth, thus stopping tumor growth.

This potential anti-cancer drug is now ready to be tested in humans for the first time. "Our goal is to test this novel agent in patients in order to establish safety and then to proceed quickly to rapid clinical development. We are excited to continue this academic collaboration with our Canadian colleagues to test this drug in humans for the first time," said Wainberg. Drs. Slamon, Wainberg, Mak and colleagues will also look for biological indications, called biomarkers, that researchers can use to tell if and how the drug is working.

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