Medical Research and Clinical Trials:
Unique Opportunities for Giving and Hope
Anne Coscarelli, Ph.D.
Medical research is an area of contemporary life that creates both controversy and hope. It has been the subject of horror and science fiction films and often has sparked political demonstrations. For many people facing life-threatening illnesses, however, medical research holds the glimmer of hope for cure or remission when all other standard treatments have been exhausted. Medical research is the one proven means we have to advance our understanding of disease and develop breakthroughs in prevention and treatment. I wonder how many people who read our newsletter have had the opportunity to participate in medical research, and how many have made the choice to participate? My guess is that since the UCLA Medical Center is a research and teaching hospital, these opportunities have been many, and more people in our community have taken part in them than one might expect.
This issue of our newsletter focuses on giving, and I want to discuss the ways in which participation in medical research and clinical trials is a unique and valuable way of giving. Participating gives to people in future generations the potential to live with different circumstances and options than those with which you and your family have lived. It gives hope, not just for future, but also for the present. I have known patients with cancer who felt that there was no hope, but who made the decision to participate in clinical trials that not only extended their life but also enriched its quality. Their participation also made a contribution to further knowledge that ultimately will benefit many in the future.
Research is the most important way we have to advance our understanding of cancer - its causes, the ways in which it affects individuals, and its treatment. Research comes in many different forms from completing questionnaires and participating in interviews, to trying new medications, combining medications and altering doses, to new procedures and other interventions such as genetic manipulation. Research can be conducted in many ways. For example, sometimes reviewing literature that has already been written and evaluating the implications of this body of work can advance science and lead to new questions. Studying populations of people and comparing them to others can lead to understandings of potential relationships of behaviors and health outcomes. For example, research showed that there were significant differences between the rates of certain cancers in the United States and those in Japan. This finding led to questions and studies of what happened to Japanese people who immigrated to the United States. In one generation, the rates for breast and prostate cancer increased substantially. Scientists then began to focus on the aspects of their lives that had changed, particularly in their diets, which led to new hypotheses and studies into probable risk factors for these two types of cancer.
Most medical research takes place in highly specific laboratory studies where small pieces of information are learned and, over time, are combined with other findings that lead to new understandings. This is called basic research and often is likened to the story of the blindfolded person who was allowed to touch just the individual and small parts of an elephant at one time. Only when he was allowed to roam his hands over the elephant's entire body did he have any realization of what was in front of him. Basic research depends upon many individual efforts contributing to form a greater whole.
Basic research eventually leads to bedside research: laboratory findings are translated into studies, called clinical trials, in which humans participate and benefit from. In the initial course of such an investigation, the studies are small in scope and may investigate issues such as safety and appropriate dose of medications. These studies grow over time into larger scale studies in which old treatments are tested against new treatments in large samples of individuals. Many times, these studies are randomized and double- blinded, meaning that individuals do not choose which treatment they receive, and neither the scientists nor the research participants know which treatment they will receive. Recently, a few such studies have had significant impact on our understanding of the ways in which cancer can be treated and possibly prevented. The Herceptin clinical trials led at UCLA by Dr. Dennis Slamon developed a new treatment for women with metastatic breast cancer. The recent tamoxifen trials for women at risk for breast cancer also have created new treatment and prevention opportunities.
Not all studies evaluate cancer treatment. Issues regarding quality of life and compliance to treatment regimens also must be studied. Psychologists and other behavioral scientists play important roles in advancing our understanding of these issues.
UCLA's Jonsson Comprehensive Cancer Center, of which we are an integral part, is highly committed to research: "the end of cancer begins with research" is its slogan. Being committed to research includes having a constituency of patients and family members who also are dedicated to furthering investigations and are willing to invest time and sometimes take the risk of trying something new. Recently one of our researchers at UCLA, Dr. Patricia Ganz, began a voluntary registry of families at high risk for cancer. Many breakthroughs in the future may involve understanding genetic links to cancer. By bringing together those people who have certain types of cancer within their families and who are willing to help advance our knowledge, this registry will help our researchers to quickly identify and call upon these individuals to participate in new studies as they are designed.
If you are considering participating in research, please keep in mind several steps that are part of the process:
Information gathering
Both you and the researchers must gather information to determine whether you meet eligibility criteria and whether the project is something in which you want to participate. It is important to ask questions.
Informed consent
Special boards are required to review all studies prior to their inception to determine the risks and benefits of the research and to make certain that they meet the appropriate guidelines. Indi-viduals interested in participating in any study should receive a written explanation of the study and what is involved as an Informed Consent form to be signed.
Data collection
Data often are collected at the beginning and at various points throughout the study. If a randomized component is involved, it usually occurs fairly early in the data collection phase. Randomization is a process by which a person is assigned to be a part of a particular section of the study, which is then referred to as an arm of the study. As the study progresses, it is likely that evaluations will take place and more data collection will occur.
Follow-up
Some studies have an end point that coincides with an event or time period; other studies are ongoing with less definite end points. Most studies involve some follow-up, which can be one of the most important aspects. It is important, therefore, that individuals continue to participate until the researchers indicate that their involvement is no longer needed. Dropping out of a study can affect the researchers' ability to draw accurate conclusions and can invalidate a study.
Data analysis
After all the data are collected, a process of analysis, which often involves complicated statistics, is conducted to obtain results and from which conclusions are drawn. In the best research, the "outcome" raises new questions, which lead to new studies. Sometimes, the results can lead to changes in the way that a disease is standardly treated. In most instances, studies are carried out at multiple sites because each study will contribute different information and will serve to corroborate findings. Treatment strategies are more likely to change when more than one study has a similar finding.
Revelation of results
Once the conclusions have been drawn, research studies usually are presented at scientific meetings so that discussions can take place with other researchers doing similar work. The studies and their findings also are published in professional journals. In a few instances, the media report on these studies, making the results more widely known. While it is beneficial to inform the general public about the research, the information can get distorted especially when complicated findings are simplified or the potential implications are over-exaggerated. Most research studies are not reported to the general public by the media. Your physician can play an important role in helping you to determine whether or not a study that has received media attention has been accurately portrayed and whether or not it has any relevance to you and your situation.
To find out more about available research studies, you must ask questions. Talk to your doctors, nurses, and mental health professionals and pick up fliers that are in their waiting rooms. Do not rely upon just one source of information. Call the Cancer Information Service (1- 800 - 4 - CANCER) and review information on the UCLA Jonsson Comprehensive Cancer Center's web site at www.cancer.mednet.ucla.edu/.
It should be noted that not everyone can or should participate in research studies. There may not be a study available that addresses your particular situation. Some people have complicating factors that would make it unwise for them to follow the specific protocols of a study. If you find that for whatever reason you can't take part in a study, please don't feel that you are not doing your part. What matters is that you were open to the idea of participation, that you were willing to give of your time and experience, and that you recognized that supporting research is a way of investing in hope for the future.
Anne Coscarelli, Ph.D.
Wallis Annenberg Director’s Initiative in Psychosocial Oncology
© Anne Coscarelli, Ph.D. All rights reserved.
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