Dennis Slamon and Zev Wainberg from the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have been awarded a Disease Team Therapy Development award to begin clinical trials in human patients early in 2014.
In this clinical trial, Slamon and Wainberg will test a new drug that targets cancer stem cells. This drug was developed by research and development over the last decade on the cancer stem cell hypothesis. The cancer stem cell hypothesis predicts that proliferating stem cells are the main drivers of tumor growth and are also resistant to standard cancer treatments. This new drug, CFI-400945, has prevented cancer growth in an extensive series of laboratory animal tests.
An important extension of the cancer stem cell hypothesis is that cancer stem cells inhabit a particular niche that prevents anticancer drugs from reaching them. Alternatively, tumors become resistant to cancer drugs by a process called “cell fate decision,” in which some cancer stem cells are killed by chemotherapy, but other cells replace them and repopulate the tumor. This tumor repopulation is the main reason for cancer recurrence.
The new anticancer drug to be tested in this clinical trial targets the “polo-like kinase 4.” Inhibition of this enzyme effectively blocks cell fate decisions that cause cancer stem cell renewal and tumor cell growth. Thus inhibition of this enzyme effectively stops tumor growth.
This clinical trial will test this novel chemotherapeutic agent in patients to establish the safety of the drug. After these initial safety tests, the trial will quickly proceed to further clinical tests. “We are excited to continue to test this drug in humans for the first time,” said Wainberg. Slamon, Wainberg and others will also look for biological markers to determine how well their drug is working in each patient.
The US Food and Drug Administration approved the Investigational New Drug (IND) for this drug trial. Also, Health Canada, the Canadian government’s therapeutic regulatory agency, also approved this trial. These approvals are part of an international effort to bring leading-edge stem cell science to patients.
Beyond the Dish 