Three weeks after being diagnosed with Stage 2 breast cancer, 51-year-old Gail Hudson was given a treatment that caused her to break out in hives.
So doctors went out on a limb and gave her Abraxane, a drug that had only been used to treat more serious, Stage 4 patients.
“My cancer, as my doctor put it, melted away,” Hudson, a Minneapolis resident, said.
A novel, collaborative study beginning at the University of Minnesota in a few months will further explore the method of tailoring drugs to specific patients. Called I-SPY 2, the study will take place at more than 20 locations around the United States and Canada, including Mayo Clinic.
Researchers will take note of patients’ tumor characteristics and will look for biomarkers such as proteins or receptors, which can predict responses to certain treatments.
“We know that not all patients will respond to every single drug,” said Douglas Yee, breast cancer specialist and director of the Masonic Cancer Center. “We want to give more targeted therapy. We want to treat the abnormalities that cause that particular type of cancer. That’s the goal.”
Rather than test one drug at a time, the five-year, $26 million study will include 12 experimental drugs, discarding those that are shown not to work early on and tailoring those that do to the sub-groups in which they’re most effective.
The format is expected to cut drug approval time — typically about 15 years — in half, Tufia Haddad, the study’s lead investigator at the University, said, adding that she expects to know within a year whether a drug improves outcomes.
“Right now, the only way we can tell if a drug is going to work is just to give it to a woman,” said Haddad, an assistant professor in the Medical School. “Our goal is to get smarter in terms of selecting drugs that have the best chance of response for a woman’s particular tumor.”
If successful, the framework could be applied to research on other diseases, said Judy Boughey, the study’s lead investigator at Mayo Clinic.
A total of 800 Stage 3 breast cancer patients will participate, and each will receive standard chemotherapy in addition to the experimental drugs.
Rather than testing a drug on thousands of patients, researchers expect to know whether a drug works after only 20 to 80 patients, thereby dramatically reducing the cost, impact and duration of such trials, Haddad said.
Patients will be randomly divided according to the drug they’re given, with 20 percent receiving placebos.
Unlike in standard treatment, I-SPY 2 patients will receive chemotherapy and the experimental drugs for 12 weeks before they undergo surgery to remove their tumors, which will allow researchers to monitor the treatments’ effects on the tumors using MRIs.
Hudson, who participated in the study’s predecessor, I-SPY 1, said having multiple MRIs provided a feeling of comfort during her treatment.
“I felt like I was really being watched,” she said. “I felt like we could see how I was responding to the drugs they were giving me.”
The various trial locations will share their results through monthly Web conferences and a central database.
This trial is unique in that it doesn’t give credit to a single researcher, Boughey said. It also gives institutions access to state-of-the-art drugs they wouldn’t otherwise have.
“Women want the latest and the best drug rather than the one developed 10 to 15 years ago,” she said.
Unlike most trials, which are sponsored by a single pharmaceutical company, funding for I-SPY 2 comes largely from the Foundation for the National Institutes of Health.
Various nonprofit organizations have also contributed, as well as the participating drug makers.
It was difficult to convince some companies to participate, as the study doesn’t focus on a single drug, said Yee, chair of the drug selection committee.
“We’re free to tell them things that they don’t want to know,” he said, “and not all companies have embraced that idea.”
I-SPY 1 is the first phase of the ongoing series and has been carried out in a similar format. Its sites will close when I-SPY 2 sites open.
It currently costs between $700 million and $1 billion and takes nearly two decades to develop a cancer drug — a tragedy considering advances in the understanding of cancer biology, Anna Barker, deputy director of the National Cancer Institute, said at a press conference announcing the trial.
“We probably stand at a point where we can actually understand the underlying biology of these diseases,” she said. “If we can figure out what these biomarkers are and integrate them, we can figure out which drugs work and which don’t.”
The I-SPY 2 trial represents a “paradigm shift” in developing personalized cancer treatments, Barker said.
“It will set a standard for trials and processes to come,” she said. “It’s a new way of doing business.”