Clinical trials at Massey
"Advancements made in clinical trials have improved survival of cancer," explains medical oncologist Andrew Poklepovic, M.D.
Every day VCU Massey Comprehensive Cancer Center is discovering new and better ways to treat and prevent cancer. One way in which Massey conducts research is through clinical trials. Clinical trials are research studies that evaluate new medical treatments and devices and increase scientific understanding of a wide variety of diseases.
These trials are important because of the advances in cancer medicine that come from new ideas and approaches developed through research. A vast majority of treatments that are standard today were first shown to be effective in clinical trials.
As one of only two National Cancer Institute (NCI)-designated Comprehensive Cancer Centers in Virginia, Massey is partially funded by the NCI to conduct research in the labs and in clinical trials to find better ways to prevent, diagnose and treat cancer.
“To maintain our NCI designation, we must show that we make new discoveries in regard to cancer and also translate that basic research into clinical applications,” said Robert Winn, M.D., director of VCU Massey Comprehensive Cancer Center. “This presents a huge advantage to patients throughout our region. For patients whose cancers do not have an effective treatment, this makes protocols available to them years before they become standard practice.”
Patients who choose to enroll in clinical trials at Massey receive some significant benefits for participating. First, these patients will gain access to promising drugs, medical devices or treatment approaches before the general public. Patients will have a more active role in their own health care and will receive expert medical care. Physicians, nurses and other health care professionals closely monitor patients’ health and side effects during the trial. Some patients will also get free or subsidized health care for the duration of the trial.
“We believe every patient should have the opportunity to participate in clinical trials because they might be able to benefit from a therapy that is more effective than current standard-of-care protocols,” said Winn.
The clinical trial team at Massey typically consists of doctors and nurses as well as social workers and other health care professionals. Many clinical trial participants also continue to work with their primary health care provider.
“Clinical trials are not just for patients who have ‘run out’ of options,” said Andrew Poklepovic, M.D., medical director of the Massey Clinical Trials Office. “With the recent major breakthroughs in cancer, often a clinical trial is appropriate and an opportunity for a patient with a new diagnosis of cancer.”
One area of current cancer research is in combination therapy. There are several new combinations of drugs that are being used to treat several types of cancers. For example, a clinical trial may test the combination of two specific drugs to determine if it is an effective treatment for breast cancer.
Another area of research interest has been in image-guided radiation therapy trials for the treatment of prostate cancer. Because it is often difficult to establish the ideal amount of radiation patients should receive, research was conducted to determine if image-guided radiation technology provides a better outcome.
The areas of research that Massey’s clinical trials span are numerous and varied — current trials are evaluating the latest treatments for many different cancer types, from brain and breast to prostate and leukemia. At any given time, Massey offers more than 100 clinical studies to adult and pediatric cancer patients, representing one of the largest selections of cancer clinical research in Virginia.
“Choosing to participate in a clinical trial is an important personal decision,” said Harry Bear, M.D., Ph.D., associate director for clinical research at Massey. “Many people participate in clinical trials because it’s an opportunity to help researchers find new ways to fight disease. Imagine being a part of discovering tomorrow’s treatments today.”
A clinical trial is a research study involving patients that is used to find better ways to diagnose, prevent or treat diseases using new drugs or new devices. Trials are designed to answer specific questions about new and established therapies. They help doctors tell if a new treatment works and is safe. Millions of patients have taken part in this kind of research, and nearly all of today’s cancer drugs were tested this way.
Some common reasons for conducting clinical studies include:
- Evaluating one or more interventions (for example, drugs, medical devices, approaches to surgery or radiation therapy) for treating a disease, syndrome, or condition
- Finding ways to prevent the initial development or recurrence of a disease or condition. These can include medicines, vaccines, or lifestyle changes, among other approaches
- Evaluating one or more interventions aimed at identifying or diagnosing a particular disease or condition
- Examining methods for identifying a condition or the risk factors for that condition
- Exploring and measuring ways to improve the comfort and quality of life through supportive care for people with a chronic illnessThe National Cancer Institute designated and partially funded VCU Massey Comprehensive Cancer Center to conduct research that addresses the nation’s cancer problems. Clinical trials are an essential tool in solving these problems because they evaluate the latest therapies and bring to patients novel laboratory research findings.
VCU Massey has one of the largest offerings of clinical trials in Virginia. Evaluating the latest cancer treatments for adult and pediatric patients, Massey conducts more than 200 trials on more than 20 types of cancers from brain and breast, to leukemia, prostate, lung and more. View the video below to learn more (¿Qué es un estudio de investigación clínica?)
Clinical trials are offered to patients as an additional treatment option to consider. In health care, offering clinical trials is standard and is not exclusive to a certain type of patient or condition. In some instances, a clinical trial is the only treatment option.
There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies:
- In a clinical trial, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants' behavior, such as diet. Clinical trials may compare a new medical approach to a standard one that is already available, to a placebo that contains no active ingredients, or to no intervention. Some clinical trials compare interventions that are already available to each other. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants.
- In an observational trial, investigators assess health outcomes in groups of participants according to a research plan or protocol. Participants may receive interventions (which can include medical products such as drugs or devices) or procedures as part of their routine medical care, but participants are not assigned to specific interventions by the investigator (as in a clinical trial)
Most clinical research progresses in an orderly series of steps or phases.
- Phase 0/Exploratory Phase: involves dosing a very small and limited number of subjects (patients) with a very small and limited range of doses for a specific period of time
- Phase I/Early Phase: tests an experimental treatment on a small group of often healthy people (20 to 80) to judge its safety and side effects and to find the correct drug dosage
- Phase II: uses more people (100 to 300). While the emphasis in Phase I is on safety, the emphasis in Phase II is on effectiveness. This phase aims to obtain preliminary data on whether the drug works in people who have a certain disease or condition. These trials also continue to study safety, including short-term side effects. This phase can last several years.
- Phase III: gathers more information about safety and effectiveness, studying different populations and different dosages, using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people. If the FDA agrees that the trial results are positive, it will approve the experimental drug or device.
- Phase IV: takes place after the FDA approves use or drug/device. A device or drug's effectiveness and safety are monitored in large, diverse populations. Sometimes, the side effects of a drug may not become clear until more people have taken it over a longer period of time.
It varies based on the patient, however here are some reasons as to why they may decide to participate:
- Other treatment options did not work
- There is no other treatment option
- They want help researchers learn more about certain health problems
- They want to be able to help future patients based on the outcomes of the clinical trial
- It is completely voluntary and a patient can stop participation at any time throughout the process
Usually, the clinical team will present clinical trials options to patients during any clinic visit when discussing treatment options based on the study’s eligibility criteria:
- Patients can also look for clinical trials via https://clinicaltrials.gov
- Patients can be engaged through open communication and education
- Patients should never be coerced into participating in a clinical trial
After patients consent to participate in the clinical trial, they will complete screening procedures to determine eligibility. A physician or advanced practice provider that is trained on the clinical trial determine a patient’s eligibility to participate based on the set parameters in the clinical trial protocol
Clinical trial protocols are the action plans or guidelines that must be followed during the course of any study. Guidelines may cover:
- The design of the study – what it will study and how
- Who may participate – criteria for participants who enter the study
- What medications and medical tests will be used with the participants
- What information is being researched
Several research centers may be involved in the study, and each uses the same protocols to ensure that information from all centers can be combined and compared.
Large cancer centers, university hospitals, local medical centers or physician offices may offer clinical trials. There may be only one or two locations involved in a particular study, or hundreds around the country.
Many of the clinical trials at VCU Massey Comprehensive Cancer Center are offered as part of a collaborative effort among multiple cancer centers and research facilities organized into national or regional groups. Working in unison, members of these groups offer the same studies and follow the same protocols for each trial. The protocol is a study’s game plan that each researcher follows to the letter in order to ensure the consistency of the data collected and the integrity of the results drawn from the data.
With strength in numbers, cooperative groups help expand the number of patients on studies and can collect more data to evaluate the effectiveness of the trial. Also, enrolling participants in multiple locations means a trial can reach its enrollment goals sooner, accelerating the research.
Currently, VCU Massey participates in or has access to trials from numerous consortia:
Patients on clinical trials are monitored very closely by their doctors and care team. In fact, many patients have said they appreciated the extra attention they received while participating in a clinical trial.
In addition, trials are generally overseen by an independent data and safety monitoring board as well as by any cooperative group study teams through which the trials are offered. Patient data — without individual identifiers — is reported and shared among the cooperative group members.
As part of the monitoring and data-sharing process, clinical researchers can identify any irregularities in symptoms. If a problem arises, the trial can be altered or stopped, and a patient may opt out at any time for any reason. Just like all medical treatments, cancer clinical trials are not risk-free. However, the safety of patients is of utmost importance to the researchers. There are several procedures in place as part of the protocols to protect the safety of the people who participate. Three groups that oversee the safety procedures include:
- The organization that sponsors the study (for example, the National Cancer Institute).
- The Institutional Review Board (IRB) that oversees clinical research in the health care institution. The IRB includes physicians, other health care providers, consumers and sometimes members of the clergy, who do not have any personal interest in the results of the study. As neutral reviewers, they ensure that the study is conducted fairly and that participants are not likely to be harmed.
- The Data Safety and Monitoring Committee, an objective committee that looks at the test results, monitors the safety of the participants and decides whether the study should go forward as originally planned.
Sugar pills (placebos) are seldom used in cancer clinical trials and are never used if a known, effective treatment is available. Patients in clinical trials always get the best treatment available. If a new drug is being tested as part of a trial, one group of patients might receive the current best standard treatment plus the new drug, while another group may receive the current best standard treatment plus a placebo. Either way, everyone is getting the best treatment available. Comparing the results of the two groups enables researchers to determine if the new drug is more effective than the current standard treatment.
VCU Massey Comprehensive Cancer Center would never let the cancer go untreated. The only time a placebo would be used alone is if there is currently no standard treatment or if a new cancer prevention measure is being tested for people without cancer.
No, clinical trials will list the potential side effects that patients may get. It is not broken down by race and just provided as a percentage based on outcomes from other clinical trials studying the same drug
The cost to a patient on a clinical trial will vary dependent on various factors:
- If it is standard of care, it will be billed to patient’s insurance
- If solely being done for research purposes, it is usually covered by trial funding
- Some studies may compensate patients for their time or travel. The informed consent form will explain any costs or payments if applicable
Many insurers cover the normal costs of treatment for cancer clinical trials and many states, including Virginia, have mandatory coverage for certain types of trials. Research nurses at VCU Massey Comprehensive Cancer Center will gladly contact a patient’s insurance company on his/her behalf.
The cost to VCU Massey is considerable given the infrastructure expenses and labor-intensity of clinical trials, but offering them is a critical component of our research mission. Clinical trials are labor-intensive because we must collect and manage a wealth of data and have more frequent contact with these patients. For example, we have over 600 breast cancer clinical trial participants who are now cancer-free but we follow up with them at least once a year to continue to report their data and to monitor for any long-term effects or trends.
The only difference is that a clinical trial patient will have additional individuals as part of their care team and may be receiving an intervention/treatment that is not currently approved/available to patients not participating on the clinical trial as well as potentially adding additional visits to collect items per the study requirements
Several cancer prevention and control studies are also available for people without cancer. Many participants are those who have a strong family health history of cancer and want to explore ways that may reduce their chances of developing cancer.