Tamara Mobley, 38 and married with 8 and 12 year old sons, has been battling multiple myeloma for five years now under the care of Dr. Sagar Lonial at the Winship Cancer Institute of Emory University. She went on a clinical trial at Winship in order to get the most advanced drug for treating this blood cancer. Because of that trial, the drug is now FDA-approved and is helping Tamara maintain her active life.
Clinical trials are responsible for most advances in medical treatment, but they can’t take place without volunteer participants like Tamara. Unfortunately, there are still many misconceptions about clinical trials that keep people from participating.
For instance, some believe joining a clinical trial is a last resort in the treatment process, which was not the case for Tamara and many other Winship patients. For Tamara, enrolling in a clinical trial was a good option once her standard cancer drugs stopped working.
In the video below, Fox 5 Atlanta talked to Tamara and Dr. Lonial about the decision to participate in a clinical trial.
February 27th, 2014 By R. Donald Harvey, PharmD, FCCP BCOP, director of the Winship Cancer Institute of Emory University’s Phase I Clinical Trials Unit
R. Donald Harvey, PharmD, FCCP BCOP, director of the Winship Cancer Institute of Emory University’s Phase I Clinical Trials section
Each of the agents we use to treat cancer had a beginning, a first step, in understanding how safe and effective they might be. As drugs are developed, we ask questions in different ways at each step, or phase, of testing. The National Cancer Institute reminds us that clinical trials are available for all patients at all points in their cancer journey, not just for patients with advanced cancer that is not responding to treatment.
When a drug is first given to patients, it enters testing in a phase I trial, where we ask questions such as:
What is the right dose?
How should it be given (e.g., by mouth, by vein, under the skin)?
What is the right schedule of treatment?
What side effects are there and how severe are they?
How often do we see side effects?
Where did the drug go in the patient? How well was it absorbed? How was it metabolized and/or eliminated? (Pharmacokinetics)
What did the drug do to the patient, both in blood and at the site of the cancer? (Pharmacodynamics)
Patients courageous enough to enter phase I trials are asked to do many time-consuming but important things during the trial. Frequently, patients are asked to spend 10-12 hours in our clinical trials unit and/or come in daily up to 14 times during the first treatment period, or cycle. During these visits, blood is drawn, tumor or bone marrow biopsies may be performed and safety tests are conducted, all in an effort to get a complete picture of drug effect, disposition and side effects.
Participation in phase I clinical trials:
To participate in a phase I trial, patients typically have cancer that has not been effectively treated with other therapies, and most trials require patients to be otherwise relatively healthy. Phase I trials usually enroll 10-40 patients, but may be larger or smaller depending on the questions being asked. Two types of phase I trials exist: those where the drug is being given for the first time, or first-in-human trials; and those where there is prior experience and the drug is given in combination with another drug or drugs (also called phase IB trials). In each, the investigational agent is given to small groups of patients, and doses are increased in each group. Both types are critical to the next step of development to define the dose, frequency, and understand what cancer types are most likely to benefit.
Phase I trials help to determine the future of drugs in cancer treatment. Right now, the large number of new agents in early testing indicates great potential in the transformation of therapy. People in good health may choose to participate in clinical trials simply to help researchers find better treatments. Participation in clinical trials is completely voluntary, but you should also speak with your physician before deciding to enroll.
R. Donald Harvey, PharmD, FCCP BCOP is director of the Winship Cancer Institute’s Phase I Clinical Trials section, and Associate Professor of Hematology and Medical Oncology at the Emory University School of Medicine. He is a Fellow of the American College of Clinical Pharmacy and a board certified oncology pharmacist. Widely published in peer-reviewed journals, Dr. Harvey’s research interests include the clinical application of pharmacokinetic, pharmacodynamic, and pharmacogenomic data to patient care.
Fox 5 News health reporter Beth Galvin features the first “patient” in a Brain Tumor trial that combined surgery with a new experimental agent. Petey, a dog, is a part of a research trial aimed at translating new brain cancer therapies to humans by assessing results in dogs with similar diseases.
Winship Cancer Institute of Emory University neurosurgeon, Costas Hadjipanayis, MD, PhD, developed the experimental agent in his Brain Tumor Nanotechology Laboratory. Petey’s tumor was partially removed in surgery at the University of Georgia (UGA) College of Veterinary Medicine, and the new, investigational drug was infused directly into the tumor area. Now, 15 months later, his tumor has shrunk and Petey is seizure-free and doing well. Watch the full story here:
What if there were a way for 32,000 of the 160,000 lives lost each year related to cigarette smoking to be saved? There may just be. Findings from a recent study show the risk of dying from lung cancer could be reduced by 20 percent by use of a low-dose helical computed tomography (CT) scan. In other words, this type of CT screening could save over 30,000 lives a year.
Lung cancer is the leading cause of cancer-related deaths, and as such, cancer research and treatment experts are constantly looking for ways to reduce the pervasive nature of the disease. The National Cancer Institute (NCI) launched the multi-center National Lung Screening Trial (NLST) in 2002. The trial compared two ways of detecting lung cancer using low dose helical (spiral) CT vs a standard chest X-Ray. Part of this research study was actually led at Emory by radiologist and researcher Dr. Kay Vydareny.
The trial aimed to determine the effects of low-dose helical CT scans vs chest X-Rays on lung cancer death rates in high-risk populations. Both chest X-rays and helical CT scans have been used as a means to find lung cancer early, but the effects of these screening techniques on lung cancer mortality rates had not been determined.
Over a 20-month period, more than 53,000 current or former heavy smokers ages 55 to 74 joined NLST at 33 study sites across the United States. In November 2010, the initial findings from NLST were released. The conclusion? Clinical trial participants who received low-dose helical CT scans had a 20 percent lower risk of dying from lung cancer than participants who received standard chest X-rays.
While the benefits of low-dose helical CT scans in the reduction of lung cancer deaths are obvious, not every diagnostic option comes completely risk free. The CT scans can occasionally detect suspicious abnormalities that do not turn out to be lung cancer – known as false positives. Many of these abnormalities are scars from smoking, areas of inflammation or other noncancerous conditions that may require additional testing to determine that they are not harmful. These tests have been known to cause undue anxiety for patients and may sometimes lead to biopsies or surgeries.
“It is certainly an individual’s choice whether they want to be screened for lung cancer with a CT scan if they have no symptoms. However, it is important to make certain that such individuals have complete information and be well-informed before having such a scan. If a patient has symptoms, such as a persistent cough, weight loss, persistent hoarseness or trouble breathing, he or she should see a physician as soon as possible. Often these symptoms are due to something other than lung cancer, but more tests should be performed to make certain,” says Vydareny.
What should a person at high-risk for lung cancer do? The answer that all physicians agree on is to stop smoking right now, the sooner the better. Or even better … don’t start smoking ever, further reducing the chances of getting cancer or suffering from a stroke or heart attack as well.
“All physicians hope that there will be a test that can screen for early lung cancer and that the dismal prognosis of those with lung cancer will improve. Perhaps it will be screening with CT scans. Perhaps it will be a blood test,” says Vydareny. “That day hopefully will come, but it has not yet arrived. But if you are or have been a heavy smoker, your first step should be to consult your primary care physician to discuss all possible screening options.”
Winship Cancer Institute of Emory University, Georgia’s only National Cancer Institute-designated cancer center, serves as the coordinating center for cancer research and care throughout Emory University and Emory Healthcare. Seeing more than 14,000 patients each year, Winship at Emory offers patients with access to progressive resources, technology and cancer treatment options. To benefit from these investigational advancements in cancer treatment, it is important to seek care from an academic medical center like Winship at Emory, first. Through discoveries made possible by a dedicated team of many of the nation’s best physicians and researchers, Winship at Emory works hard toward a future when science triumphs over cancer.
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