A unique public-private partnership made up of research institutes, regulatory bodies, and drug companies, has launched a new clinical trial model to help screen promising new drugs being developed for women with high risk, fast-growing breast cancers—women for whom an improvement over standard treatment could dramatically change the odds of survival.

 

Led by the Foundation for the National Institutes of Health, the U.S. Food and Drug Administration, and the Biomarkers Consortium, the I-SPY 2 trial will employ a groundbreaking clinical trial model that uses genetic or biological markers from individual patients’ tumors to screen new treatments to identify which ones are most effective in specific types of patients. An adaptive trial design will also enable researchers to use early data from one set of patients to guide decisions about which treatments might be more useful for patients later in the trial, and eliminate ineffective treatments more quickly.

 

“I-SPY 2 promises to leverage convergence of progress on a number of research fronts to speed the evaluation of promising new breast cancer drugs using molecular cancer biomarkers to identify those agents that are effective in specific subpopulations of breast cancer patients,” says Anna Barker, deputy directory of the National Cancer Institute, and co-chair of The Biomarkers Consortium Cancer Steering Committee. “This will allow us to finally design advanced, smaller and less expensive phase 3 trials that test the right drugs in the right patients.”

 

The large-scale trial will involve nearly 20 major cancer research centers across the country. Study results will be made broadly available to the entire cancer research and development community. I-SPY 2 is expected to cost approximately $26 million over five years. Funding will come from a variety of sources, and Safeway, one of the largest food and drug retailers in North America, has contributed significant seed funding.

 

The hope is that the I-SPY 2 model will significantly reduce the cost of drug development by allowing the activity of drugs to be assessed much earlier in the research process, potentially enabling drugs to be developed and approved using fewer patients, less time and far fewer resources.

 

“Developing individualized medicines needs a solution bigger than any one group can generate,” said Janet Woodcock, director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration.  

 

The I-SPY 2 trial will focus on treatment in the neo-adjuvant therapy setting, in which chemotherapy is given to patients to reduce tumor size before surgery. All patients will receive the current standard of care and most participants will receive one investigational drug. A distinctive feature of the trial is that it will screen multiple drugs from multiple companies—up to 12 different cancer drugs over the course of the trial. In order to do this, the Foundation for the National Institutes of Health received a master Investigational New Drug approval from the FDA. That allows the I-SPY 2 TRIAL team to graduate, drop, and add drugs seamlessly throughout the course of the trial without having to stop the trial to write a new protocol. Researchers have already selected five new investigational agents currently in development by three major pharmaceutical companies for testing as part of the first phase of the trial: Abbott's veliarib, Amgen' conatumumab, and Pfizer's figitumumab and neratinib, all of which are being donated by the drug companies.

 

“I-SPY 2 will provide a path to personalized medicine,” said Laura Esserman, principal investigator and breast cancer surgeon and researcher at the University of California, San Francisco. “The collaborative power behind this trial is truly transformational for breast cancer patients and for cancer research as a whole. We have set up a system where everyone can learn faster and, together, we can dramatically reduce the amount of time and the cost to bring those drugs to market that can make a difference in whether women live or die.”

 

Breast cancer advocates, including former patients, helped design the trial, create brochures, a website, and DVD to inform patients about the trial. The Foundation for the National Institutes of Health is managing the trial as part of The Biomarkers Consortium, a public-private biomedical research partnership that endeavors to develop and qualify biomarkers to speed the development of medicines and therapies for detection, prevention, diagnosis, and treatment of disease and improve patient care.