CLINICAL TRIALS IN WALDENSTROM’S MACROGLOBULINEMIA
The search for the cure in Waldenstrom’s Macroglobulinemia (WM) will never be successful without the participation of WM patients in clinical trials. Rare diseases, also called “orphan” diseases like WM, are at a disadvantage when it comes to a search for a cure simply because of the low numbers of patients with WM. In a country such as the United States, with over 300 million in population, there exists an approximate 6,000 people with WM, and approximately 1,500 are newly-diagnosed every year. This contrasts markedly with breast cancer, where 50,000 individuals will die of the disease in a single year!
The IWMF has increasingly supported the promotion of clinical trials as it recognizes that a cure, or major advances in the treatment of the disease, is impossible without clinical trials, and that the development of a cure or major advances in treatment may actually be hampered by the low levels of participation by adults afflicted with non-Hodgkin’s lymphomas and cancers in general. It is a rather startling fact that over 80% of pediatric patients participate in clinical trials, whereas only 5% of adults do. The present and past leaders of the National Cancer Institute at the National Institute of Health, and past Surgeon Generals have frequently repeated the assertion that if we could double the number of adults participating in clinical trials, we could conceivably see cures in cancers in 5-10 years. 90% of pediatric blood cancers are now curable. We seek the same results in adult blood cancers!
This webpage will introduce you to basic information about clinical trials and provide links to information on clinical trials as well as the listing of specific clinical trials in Waldenstrom’s macroglobulinemia.
COMMON QUESTIONS ABOUT CLINICAL TRIALS
What are clinical trials?
Clinical trials are cancer treatment research studies that test new treatments in people with cancer. The goal of a cancer clinical trial is to develop better treatments and ultimately a cure. Trials may include new drugs, new surgical or radiation therapy techniques, new combinations of treatments, and revolutionary methods such as gene therapy.
Why are clinical trials necessary?
Clinical trials contribute to knowledge and progress against cancer. Most of today’s most effective cancer treatments are the direct result of clinical trials. Important scientific questions may be answered by clinical trials and may also suggest future research directions. Cancer survival is directly linked to progress from clinical trials.
Why should I enter a clinical trial?
Patients receive up-to-date care from cancer experts, and receive either a new promising treatment or the best available standard treatment for their cancer. Many patients now choose to receive their first treatment in a clinical trial because of the increasing evidence that clinical trials (certainly in WM) provide better overall results.
How are clinical trials carried out?
Each clinical trial has a protocol, a detailed action plan that explains how it works. Each trial enrolls a certain number of volunteer patients who may be alike in key ways –type of cancer, stage, history of prior treatments, current health status, etc. – these are called eligibility criteria.
What are the different “phases” of clinical trials?
There are three principal phases of clinical trials that attempt to answer different questions about a new treatment.
Phase I trials are the first step in testing a new treatment in humans. The goals of phase I trials are: how much of a new drug can be given safely, how often the drug needs to be given, and what the side effects of the new drug are. These trials usually include only a limited number of patients and are often carried out at large academic medical centers. Phase I trials in WM are quite rare.
Phase II trials attempt to determine whether the new treatment works for a specific type of cancer. The goals of phase II trials are: continue to gather information about the safety of the drug, and test whether or not the drug works against a specific type of cancer.
Phase III trials compare the treatment outcomes of patients taking the new treatment with results of people taking standard treatment. Is the new treatment an improvement over the standard treatment for a specific type of cancer? Phase III trials usually have the largest number of trial participants.
What are Randomized Controlled Trials?
In these Phase III trials the participating patients who receive the treatment under study are assigned by random. This is necessary to ensure that the outcomes are determined only by the treatment under study and not by other factors that could influence treatment assignment.
MYTHS AND FACTS*
Myth: Cancer patients avoid clinical trials because they are too risky.
Fact: In a survey of 5,900 cancer patients**, most said that they would have enrolled in a clinical trial if they were aware of it as a treatment option. In clinical trials, patients are watched closely by their doctors and other members of the health care team.
Myth: You need to be near a big hospital to take part in a clinical trial.
Fact: Many cancer clinical trials take place at local hospitals. Some also take place at local cancer clinics and doctors’ offices.
Myth: Cancer clinical trials patients are given “sugar pills”.
Fact: Patients who join clinical trials are given the best treatment available or the chance to receive a new treatment being considered. “Sugar pills” (also called placebos) are rarely used in cancer clinical trials and are never used in place of treatment.
Myth: Health insurance will not cover the costs of a clinical trial.
Fact: Many insurers (in the U.S.) cover the costs of treatment on cancer clinical trials. So do many states. Check with your doctor or insurance plan to see if you are covered.
Myth: Medicare does not cover the patient care costs of clinical trials.
Fact: Medicare has been covering these costs since June of 2000.
Myth: Patients in clinical trials are treated like “guinea pigs.”
Fact: Of the 5,900 cancer patients surveyed**, 97% of those you participated in a clinical trial said that they were treated with dignity and respect. In addition, 93% said that they had an overall positive experience.
*From Coalition of Cancer treatment Groups at www.CancerTrialsHelp.org
**Harris Interactive Survey 2000
WHO SHOULD TAKE PART IN A CLINICAL TRIAL?
This question can only be answered in an individual fashion. Consult those who are close to you, and discuss it with your health care team. Educate yourself about clinical trials by visiting the websites listed below, and remember that patients who participate in clinical trials are increasingly benefiting from better outcomes than patients on standard therapy.
GENERAL INTERNET CLINICAL TRIAL RESOURCES
American Cancer Society (ACS): www.cancer.org
Centers for Disease Control and Prevention (CDC): www.cdc.gov
Coalition of Cancer Cooperative Groups: www.CancerTrialsHelp.org
Education Network to Advance Cancer Clinical Trials (ENACCT): www.enacct.org
National Institutes of Health (NIH): www.nih.gov, www.clinicaltrials.gov, http://cancernet.nih.gov, http://cancertrials.nci.nih.gov
National Cancer Institute (NCI): www.cancer.gov
National Coalition for Cancer Survivorship: www.canceradvocacy.org
SPECIFIC INTERNET CLINICAL TRIALS RESOURCES
Dana-Farber/Harvard Cancer Center: www.dfhcc.org/clinical_trials/index.shtml
U.S. National Library of Medicine’s List of Clinical Trials: http://clinicaltrials.gov
TELEPHONE NUMBERS
American Cancer Society (ACS): 1-800-422-6237
Dana-Farber/Harvard Cancer Center: 1-866-790-4500
National Cancer Institute (NCI): 1-800-332-8615
National Cancer Institute’s Clinical Studies Support Center (CSSC): 1-888-624-1937
National Coalition for Cancer Survivorship: 1-877-622-7937
VIDEO PRESENTATION
A copy of the VHS tape from an October 2002 PBS educational program titled “Test of Hope; Cancer Clinical Trials” by the Coalition of National Cancer Cooperative Groups is available upon request from the IWMF office at info@iwmf.com
OTHER SOURCES OF INFORMATION
The IWMF will provide to you upon request a copy of the NIH-National Cancer Institute’s booklet “Taking Part in Clinical Trials – What Cancer Patients Need to Know."
You can also download from the NIH website a very helpful publication titled: “How to find a Cancer Treatment Trial” at www.cancer.gov/clinicaltrials/findtrials
Dana-Farber/Harvard Cancer Center offers a patient-friendly internet program on clinical trials at www.dfhcc.harvard.edu/clinical_trials/index.shtml
LIST OF CLINICAL TRIALS IN WALDENSTROM’S MACROGLOBULINEMIA
In order to access the NIH-National Cancer Institute Comprehensive list of current WM clinical trials, go to the NCI’s clinical trials webpage at www.cancer.gov/clinicaltrials and select Waldenstrom’s macroglobulinemia from the Type of Cancer tab, select All (no other choices) from the Stage/Subtype of Cancer tab, and, finally, select treatment in Type of Trial. You may wish to enter a Zip Code or a distance from the Location of Trial tab, although this is not necessary. Hit Search.
Guy Sherwood
