Phase I Clinical Trials - A Changing Paradigm
Phase I clinical trials for cancer have traditionally focused on testing the safety of new drugs, enrolling only patients in advanced stages of disease who were counseled not to expect any personal benefit. As more targeted or "personalized" therapies emerge from the laboratory, the design of Phase I clinical trials is evolving to allow investigators to begin to look more closely at efficacy (how well a tumor is responding to therapy). In addition, investigators are testing not only new drugs but those that the FDA has approved for different types of cancer, which are now being studied in combination with previously approved breast cancer therapies.
A Personal Story
Suzanne Hebert has undergone many treatments, including surgeries and chemotherapies, to fight off her stage IV breast cancer. She was diagnosed with the advanced stage cancer at age 39, 7 1/2 years ago; it was only when she started treatment in a Phase I clinical trial at MD Anderson in July 2011 that she saw some respite from the disease.
Tell me why you decided to enroll in a Phase I clinical trial?
I had already been in a couple of Phase III trials but I had never considered a Phase I. I think there are a lot of preconceptions about clinical trials in general and about Phase I in particular. Like many patients, I just crossed it off my list. Then my doctor suggested that I go and see someone at the Clinical Center for Targeted Therapy, MD Anderson's Phase I Clinical Trials clinic. I ended up talking with Dr. Jennifer Wheler and she offered me a trial, which sounded great. She carried out some tests and decided that the drug I was going to be taking, Everolimus (Afinitor), might have a good chance of working on my cancer. So I agreed to enroll.
Were you surprised at some of what you learned from
Dr. Wheler about clinical trials?
Yes, I was. One of the first things I learned was that the drug
I would be getting was already FDA approved for kidney and brain cancer; it just wasn't approved for breast cancer. I had always thought that a Phase I trial would involve an unproven drug and that I would receive a dose escalation for safety reasons. I never considered that in Phase I, I would be getting a drug that was already approved. Knowing that I was not going to be one of the first to take the drug put my mind at ease. The other thing I learned was that I was eligible for solid tumor trials whereas I had always been researching breast cancer trials. This trial was open to people with varying types of cancer. So I would not have even have come across it if Dr. Wheler had not told me about it.