Clinical trials are essential for delivering new and better cancer treatments to patients. As a worldwide leader in cancer clinical trials, MD Anderson is driving the field of cancer medicine forward while also prioritizing the safety of our patients. Participating in a clinical trial can provide patients access to innovative therapies not otherwise available and provide physicians valuable information to help future patients. Funda Meric-Bernstam, M.D., chair of Investigational Cancer Therapeutics, describes the importance of clinical trials and what patients can expect. Request an appointment at MD Anderson by calling 1-877-632-6789 or online: https://my.mdanderson.org/requestappointment
As part of our mission to eliminate cancer, MD Anderson researchers conduct hundreds of clinical trials to test new treatments for both common and rare cancers. Look through our database to find studies for which you may be eligible.
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What are clinical trials?
Clinical trials are research studies in which patients may volunteer to take part. MD Anderson uses clinical trials to find better ways to prevent, diagnose and treat cancer. Doctors use treatment trials to learn more about how to fight cancer. This guide is for patients who may join a treatment trial.
Clinical trials are part of a long, careful process, which may take many years. First, doctors study a new treatment in the lab. Then they often study the treatment in animals. If a new treatment shows promise, doctors then test the treatment in people. Doctors do this in three to four steps, or phases. Your doctor may offer you a clinical trial as a treatment option.
How am I protected?
MD Anderson’s most important job is to protect patients. First, MD Anderson protects patients in clinical trials by following well-planned protocols.
A protocol:
- Explains the treatment plan
- Lists the medical tests patients will receive
- Gives the number of how many patients will take part in the clinical trial
- Lists eligibility criteria, which are guidelines to decide who may join the clinical trial
- Explains safety information
Second, MD Anderson protects patients by using a careful informed consent process.
Third, our Institutional Review Boards (IRBs) protect patients by reviewing protocols and monitoring trials. The IRBs are committees of doctors, nurses, chaplains, social workers, lawyers and patients. They make sure that trials follow federal laws and that patients are protected.
The U.S. Food and Drug Administration (FDA) audits the IRBs’ files. Also, FDA officials may visit MD Anderson at any time and review anything they choose related to clinical trials.
Did You Know?
In 2019, the FDA approved 35 new cancer drugs for public, clinical use. MD Anderson contributed to the development of 18 of those — more than half.
In the last decade, the FDA approved 166 cancer drugs for public, clinical use. MD Anderson contributed to the development of 94 of those — more than 56%.
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Phase I clinical trials are the foundation for how we develop new cancer drugs. Typically, they involve only several dozen patients and study a new medicine’s effect on a variety of cancer types.
David S. Hong, M.D., sat down with us to explain more about Phase I clinical trials and answer some of patients’ most frequently asked questions.
What is a Phase I clinical trial?
Phase I clinical trials are the first time human beings are being treated with an experimental drug. It starts with researchers figuring out a pathway to kill cancer. A drug company then develops a medication that mimics that process. The primary purpose of a Phase I is to figure out how much of the drug we can safely give patients and see a benefit.
But that’s not the only purpose. Phase I trials also help us figure out which cancers benefit from these drugs.
Who should consider participating in a Phase I clinical trial?
Phase I clinical trials aren’t for everyone. Most patients who choose Phase I clinical trials are running out of options. Typically, they have metastatic disease, haven’t had success with standard chemotherapy, have mild or no symptoms and don’t want to go to hospice. They’re willing to see if they can help others, and possibly themselves, by participating in a Phase I clinical trial.
What questions do you hear most from patients considering Phase I clinical trials?
“Is there a placebo?” Unlike Phase III clinical trials, there’s usually not a placebo in a Phase I trial. Everyone gets the drug, just at different dose levels.
There are also a lot of questions about schedules. Phase I clinical trials are very intensive and require multiple visits from the patient. Since it’s the first time this drug has been used in human beings, we’re very vigilant.
We also get a lot of questions about side effects. Typical side effects include nausea, vomiting and diarrhea. But side effects are different for each patient in each trial. We try to give patients an overview of the most common side effects, those that are most concerning and then all possible side effects. Most side effects are reversible and treatable. We either stop the drug or we give another medication to address the side effects.
How long will a patient be enrolled in a Phase I clinical trial?
Patients can usually stay on a Phase I clinical trial as long as they benefit. A traditional Phase I clinical trial lasts about two years (from the time we start enrolling to the time that we actually close out a study).
What happens if a patient’s condition doesn’t improve?
If a patient’s clinical condition declines, it may just be because of side effects and we can address those issues. Or, it’s because the tumors are growing. We re-image patients on a regular basis, and we do measurements on the patient following a preset criteria. Depending on the drug and the set criteria, we’ll take the patient off the study if a tumor grows a certain percentage. What happens next depends on how the patient is doing overall. Many patients go on to another Phase I study or back to their medical oncologist because he or she has one more therapy to try. Or, if they are out of options, they may decide to go to hospice.
What are some of the most promising Phase I clinical trials we have at MD Anderson right now?
There’s lots of exciting research happening. In a Phase I trial of the drug LOXO-101, we’ve seen tumors shrink in patients with cancers that are linked to the gene fusion of NTRK. We’ve also seen promising results in a Phase I trial of a combination of inhibitor drugs that target a cancer cell’s signaling. Lastly, we have many immunotherapy Phase I clinical trials, but one that’s exciting is the upcoming MAGE-A10 study on the use of engineered cells of the immune system (called CAR-T cell and T-cell receptor therapies).
What advice do you have for caregivers of patients enrolling in Phase I trials?
My number one piece of advice is don’t push your loved one to do a Phase I. It’s not always the best choice for the patient.
Two, be prepared to support this patient timewise. It’s an intensive and time-consuming process.
Three, ask questions. You need to be informed.
Anything else you want patients to know about Phase I clinical trials?
What we’re seeing in the Phase I environment is a lot of hope. Within the past decade, there’s been an explosion of new drugs. There are over a thousand new cancer drugs in the pipeline. We’re seeing a lot more activity than before; drugs that are actually working in lots of different tumors types than before. We’re also getting much more sophisticated in how we identify patients. I’m an optimist, and I wouldn’t be in this field if I wasn’t.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.
Clinical trials enable us to discover new and better cancer treatments. They offer patients access to cutting-edge treatments years before they’re available to the general public. And, in many cases, they extend participants’ lives or even eliminate their cancer.
Despite clinical trials’ benefits, far fewer adults ages 65 and older participate in them compared to younger patients. But with 50% of new cancer cases in the United States occurring in patients ages 65 and older, it’s important for older patients to consider clinical trials.
We spoke with Ishwaria Subbiah, M.D., about misconceptions about clinical trials and what older patients considering clinical trials should know. Here’s what she shared.
Why aren’t many older cancer patients enrolling in clinical trials?
Many people fear being guinea pigs. But with a clinical trial, you’re not a test subject. You’re a person going through cancer treatment. Everything we do is guided by you, your experience and how you’re feeling.
What should older cancer patients consider before enrolling in clinical trials?
There’s a lot of responsibility that goes with being on a clinical trial. You’ll have to be in very close contact with your care team, so they can quickly address any changes you experience. You’ll likely need to come into the clinic often – weekly, in some cases – and at times undergo blood draws, scans and biopsies. So consider these requirements with your care team to determine if the potential benefits outweigh the trouble.
What challenges do older cancer patients face when enrolling in clinical trials?
Over the course of your life, you’ve experienced events that have had a cumulative effect on your health and that have led to some weakening of your body. We don’t want the clinical trial treatment to cause more harm than good, so clinical trials have strict criteria for enrollment – specifically regarding organ function. The patient’s kidneys and liver need to be in great shape, and blood counts need to be excellent. We also consider a patient’s overall well-being, including independence and mobility. These are all reflections of how the body is doing overall.
When designing clinical trials, we’re starting to look at each criterion, such as age, and gauge if it’s truly necessary or if that symptom could be comfortably and safely managed while a patient is on the experimental drug. The hope is to open clinical trials to more cancer patients ages 65 and older who may be in good health but have weaker kidneys, diabetes or other signs of a long-lived life .
When are clinical trials not a good option for older cancer patients?
Your age shouldn’t keep you from considering a clinical trial. Approach a clinical trial as you do any other treatment decision, and consider the following questions:
- What’s the treatment?
- How does it work?
- How will I get it?
- How will it affect my everyday life?
- What are the side effects?
- What are the benefits?
When discussing side effects, ask if they will evolve over time. For example, if the fatigue will only last for a couple of weeks, that may be something you’re willing to accept for the long-term benefits.
By weighing the answers to these questions, you can determine if a treatment decision – clinical trial or not – aligns with your values and your desired quality of life.
What’s your advice for caregivers of older cancer patients?
Try to come with the patient to appointments, so that you’re as engaged in the processes as much as possible. If you’re not able to be there in person, try calling on speaker phone or ask the patient to record the conversation. This way, even if you’re not at the appointment, you can still be there for your loved one.
Keep in mind that cancer patients’ needs and experiences change over time. Lab tests show us of some of these changes, but there are others that only the patient can feel, so we rely on the patients to share these with us.
It’s common for patients to worry about being a burden, so many keep these changes to themselves. But the patients who do the best are those whose caregivers pick up on these changes even before the patient does. To help, we train family members on what questions to ask to identify a patient’s needs and help us address them as soon as possible. We encourage the caregiver to ask about pain, fatigue, nausea and breathing, as well as the patient’s mood, which can reflect overall health. A big part of pain expression is a person’s mood, and no amount of pain medication can make depression better. Start talking to the patient about how they’re doing emotionally sooner than later. That way, the patient doesn’t have to suffer in silence, and we can help get them the support they need.
What’s your advice for older cancer patients considering clinical trials?
Talk to your doctor early on about clinical trials and find out what options may be available for you. Even if you’re not eligible for a clinical trial right now, know that we’re making so many treatment advances and opening new clinical trials all the time, so there may be a clinical trial that opens up in the future that’s right for you. Whether it’s now or in the future, be open-minded about enrolling in a clinical trial because your participation can help us find better treatment for future patients – and potentially even improve your quality life and extend your life.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.
Clinical trials are how progress is made in medicine. “They’re really essential to drive our ability to deliver better cancer care,” says Funda Meric-Bernstam, M.D.
Enrollment in a clinical trial is entirely voluntary, but cancer patients can benefit from joining one while also helping future patients. The insights we gain from clinical trials today helps us improve treatment options that will benefit other patients.
Clinical trials are complex, though, and there are a lot of misconceptions surrounding them. Here's what you should know before you join a clinical trial – or decide it’s not for you.
Clinical trials are safe
“Safety is the most important thing,” says Ecaterina Ileana Dumbrava, M.D. Because patients on clinical trials are the first ones to receive experimental medications, there are many safeguards in place to ensure they’re not harmed.
In addition to the routine assessments cancer patients receive for standard treatment, clinical trial participants typically need additional clinical visits, lab work, imaging scans and biopsies.
Also, you’re not just communicating with your oncologist and your cancer care team. Patients on clinical trials also have continuous contact with a research team to ensure they’re doing well on the new drug.
“We’re talking to patients, often weekly, to see if they’re experiencing any side effects and just checking in to see if they have any questions,” Meric-Bernstam says. Your clinical trial team also provides guidance on next steps if the patient does experience any side effects. “There's a lot of help built in with clinical trials to make sure that patients are getting optimal care,” Meric-Bernstam adds.
But the focus on safety doesn’t start there – it happens well before patients are enrolled. “The FDA doesn’t come in just at the end of the trial to approve a drug; they approve the drug before it can be even be administered to patients in the trial,” Dumbrava says.
At MD Anderson, clinical trials are designed by experts and go through several rounds of approval to ensure they’re safe for patients. A proposed clinical trial receives its final approval from the Institutional Review Board (IRB), a committee made up of physicians, nurses, researchers, patients and lawyers.
Clinical trials occur in phases
There are four phases of clinical trials, and each has its own goal. When a new drug or a new drug combination is developed, it's tested in Phase I clinical trials. These usually have a small number of patients, about 15 to 50. “These trials are designed to determine the safety of the drug or drug combination, and what dose to use in following trials,” Meric-Bernstam says. “We’re also looking efficacy, and identifying which patients are the most likely to benefit from the drug, and, therefore, be enrolled in future studies."
Phase II clinical trials may focus on a specific cancer type and examine how it responds to the experimental drug or procedure. These trials may enroll patients with specific diseases or based on certain test results called biomarkers.
Phase III clinical trials test whether a new treatment is better than what’s being used as the standard treatment. At this point, the drug or procedure may go to the FDA for approval, but studies of the drug aren’t done. A fourth phase (Phase IV) reviews the new treatment’s long-term benefits and side effects.
Clinical trials don’t just examine new drugs
Although many clinical trials study new medicines, they help improve all aspects of cancer care. They can study new ways of dispensing treatment, explore a new dosage, test a new drug combination or examine a drug’s success in treating different cancer types.
They also focus on ways to prevent cancer or a recurrence as well as ways to reduce treatment side effects. “We want to save more lives, but we also want to maintain patients’ quality of life,” Dumbrava says.
Clinical trials don’t limit your access to other care
“No matter what the scenario, often the best treatment choice is a clinical trial,” Meric-Bernstam says. She notes that if there’s already an effective treatment approach, clinical trials offer the opportunity to build on that to make it more effective. They usually require patients to receive known effective treatment options before starting something that’s more investigational. If there aren’t clear, effective treatments, clinical trials offer access to something new that may work.
“Unfortunately, we don’t know if an experimental treatment will be effective, but that’s true for some standard treatments, too,” Meric-Bernstam says.
If your disease isn’t controlled on a clinical trial, your doctor will stop your participation and may offer another treatment option. In some cases, you may enroll in another clinical trial.
“By joining a trial, you’ll usually be getting a treatment that we think may be better than our current options,” Meric-Bernstam says.
Dumbrava agrees, adding, “At minimum, you’ll be receiving the best treatment available.”
And even if you personally don’t see clinical benefit from the experimental treatment, your participation still helps advance cancer research.
Clinical trials are for patients at all stages of cancer
Although all clinical trials have criteria for the participants, clinical trials are available to patients at all stages of cancer. The eligibility criteria, which is meant to ensure patients’ safety, may include the patient’s age, gender, cancer type and stage, previous treatments and overall health.
Your doctor is the best resource for knowing your treatment options. But don’t hesitate to ask if there are clinical trials that are available to you.
“It's also an opportunity to pay it forward and truly contribute to better outcomes for future patients,” Meric-Bernstram says.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.