Medically Reviewed On: July 11, 2008

HILDY DILLON, MPH: The purpose of cancer clinical trials is to improve current treatments for cancer or to improve the quality of life. For patients with CML, there are many opportunities today for people that are not responding. There might not be a trial for every person, depending upon their history, but it's a time of great hope because there are many new drugs that are being developed that show a lot of promise for people that have not responded to the standard therapy.

ANNOUNCER: The value of clinical trials in CML is well illustrated by the experiences of a patient named Betty. Betty was diagnosed with CML in Fall of 2002. Her blood counts were normalized with hydroxyurea. She then tried interferon, but had an immediate problem with side effects. Then she went on Gleevec. Betty seemed fine for a year. Then blood tests showed her CML was developing resistance to the drug.

BETTY: I went in, and the doctor, he looked at my blood count and he said, "That can't be right." Then he went and looked it up again and it was, and my platelet count was two million.

ANNOUNCER: Hydroxyurea again lowered her blood counts. But her disease was no longer being controlled by Gleevec.

NEIL SHAH, MD, PhD: Betty clearly lost her hematologic response, and as such, I think she is at substantial risk for subsequent disease progression. Although Gleevec remains a wonderful drug for the majority of patients who are taking it and doing well, for those patients whose disease no longer responds, there are really very few medical options available.

ANNOUNCER: Betty says her doctor at the time provided very little information about next steps.

BETTY: The only thing he said was that it just meant that the Gleevec was not working the way it should. I was frustrated, because he just didn't seem to have any more information, or he just said, "Well, we'll hope by the time that the Gleevec completely stops working there will be something else out there." And that's about all he said. So I thought, "Well, if there's something else out there, why don't I find out about it?" And I thought if they were doing it, it would be at UCLA.

ANNOUNCER: Back in 2002, Gleevec was a relatively new drug, and it was Betty who convinced her doctor to try it. Now Betty had to take the lead again.

NEIL SHAH, MD, PhD: She is actually quite a remarkable person. She herself found out about clinical trials for her disease, for disease that had become resistant to Gleevec. So not only did she first propose the idea of Gleevec when she was diagnosed, but when her disease was no longer responding adequately, she did her own homework and found out about our clinical trial and essentially referred herself for participation in our clinical trial.

BETTY: People were really surprised that I didn't get referred by a doctor. They say, "Okay, your doctor didn't refer you there?" "No. I just found out about it by myself." And I've told people that you can't wait for the doctor to decide to tell you, "Okay, go here or go there." You have to research it yourself.

ANNOUNCER: Betty lives an hour away from the University of California at Los Angeles Medical Center, and she called up, inquiring about clinical trials. She spoke with the coordinator of a trial for one of two drugs being evaluated for effectiveness in cases of resistance to Gleevec.

BETTY: I was really happy to find out that there was a trial going on, and that I would probably be eligible for it, and given what my doctor had said, that that would be the next best thing. I thought, "Oh, this is great. It's going to come along at the right time."

ANNOUNCER: It was a Phase II trial, testing high doses of Gleevec against a new drug called dasatinib now also called Sprycel, for patients whose disease developed resistance to Gleevec on the standard dose. Betty was randomly assigned to the new drug, and the initial results were quite good.

NEIL SHAH, MD, PhD: So she had an immediate, complete hematologic response, and we were able to remove her hydroxyurea, and that was the first sign that we were actually controlling her disease. And she subsequently has had bone marrow biopsies which have shown a complete cytogenetic remission, which is really very exciting, that we can take patients who otherwise have very few options to control their disease and get it back into a remission state that's pretty much as good as anything Gleevec could accomplish. So she's now in what we would call a complete cytogenetic remission.

BETTY: I called everybody and told them and called my husband, and I was crying. I said, you know, "Guess what?" So I was really, and still am surprised to this day that it's doing so good.

ANNOUNCER: The drug was not without complications. Betty has experienced shortness or breath and weakness. About 10 percent of patients develop some buildup of fluid around the lung.

NEIL SHAH, MD, PhD: Recently she has developed a little bit of fatigue, which we're in the process of trying to figure out. But my suspicion is it may be related to some of this fluid collection around the lung, and we're going to be investigating that a little bit further and doing any necessary procedures to hopefully not only treat that but prevent it from recurring.

ANNOUNCER: Betty's story illustrates how patients may sometimes do best when they take the initiative when it comes to clinical trials.

HILDY DILLON, MPH: Patients in general are becoming a lot more proactive in finding out about clinical trials on their own, better understanding what a clinical trial is and how they may or may not be eligible for specific programs. And they are proactively calling investigators at academic centers or at cancer centers and asking can they be part of a study.

ANNOUNCER: But how's a patient to know where to start?

KIMBERLY FREDERICK, LCSW: There are many resources out there available for patients who are considering doing a clinical trial or who are receiving standard treatment right now, but just want to know, is there anything else out there for me? Just in case, or to be prepared down the road. And if someone has access to a computer, they can access a host of different websites, government websites that provide a listing of hundreds of clinical trials that are available.

So, for example, for someone who has CML, they can go on to the National Institute of Health's website or the National Cancer Institute's website and actually type in "chronic myelogenous leukemia" and it will pull up all the trials that are going on around the United States and around the world for chronic myelogenous leukemia.

ANNOUNCER: Many patients need help with the process. A good first step is the Leukemia and Lymphoma Society's Information Resource Center, available through the internet, or by phone.

KIMBERLY FREDERICK: The Information Resource Center-we like to call it the IRC-has a team of master-level health professionals who provide information to patients and family members.

HILDY DILLON: What is very difficult for patients is to search on their own when they're doing this for the first time and come up with a list of trials that might actually be appropriate for them. So what we do at the Leukemia and Lymphoma Society is we ask them a number of questions that will help them to narrow down their search and find out which trials might be most appropriate for them.

ANNOUNCER: Because of a clinical trial, Betty's doing well. Her long-term prospects remain uncertain, but the trial drug has brought about a remission of disease.

NEIL SHAH, MD, PhD: I don't go so far as to presume that she is cured. I don't go so far as to presume that she will never need any additional therapy. But clearly, I think we're setting the clock back a bit, and hopefully for a period of years, and maybe more than ten years, by having this second-generation drug available for her.