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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.
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.
Clinical trials are at the core of MD Anderson’s mission to end cancer. They’re how we discover new ways to prevent, diagnose and treat cancer. Through volunteer patient participants, we can find new drugs and treatment procedures that may benefit current and future patients. They give patients access to cutting-edge treatments that are sometimes offered at discount through a sponsor.
To better understand clinical trials and to dispel some misconceptions, we spoke with Patrick Hwu, M.D., division head of Cancer Medicine. Here are five things he wants you to know about clinical trials.
Clinical trials occur in phases
There are four phases of clinical trials, and each has its own goal. Phase I clinical trials are the first time a new drug is tested in human beings. We’re trying to find the safest dosage and also which cancer types respond.
Phase II clinical trials focus on a specific cancer type and examine how it responds to the experimental drug or procedure.
Phase III clinical trials test whether a new treatment is better than the standard care. At this point, the drug or procedure goes to the Food and Drug Administration (FDA) for approval, but the trial isn’t done. A fourth phase (Phase IV trials) reviews the new treatment’s long-term benefits and side effects.
Although a treatment moves through the phases, a patient does not.
Clinical trials don’t just examine new drugs
Although many clinical trials study new medicines, some study new ways to dispense treatment, explore a new dosage or test a drug’s success in treating different cancer types.
Clinical trials don’t limit your access to other care
If a clinical trial isn’t successful in treating your cancer, your doctor may give you the option of stopping your participation and starting another course of treatment. In some cases, you may enroll in another clinical trial.
Clinical trials are safe
Patient safety is MD Anderson’s primary concern. Clinical trials are designed by experts and go through several rounds of approval. Before enrolling 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 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. By enrolling in a clinical trial, you help us advance cancer care by discovering more successful treatments that may extend or save your life. You’ll create a legacy of hope for future patients.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.
Patient Stories
April Schweigert
Mom's cancer, clinical trial sparks addressing BRCA mutation
Kelly Painter
Breast cancer survivor undergoes surgery with hypnosedation
Scot St. Martin
Throat cancer survivor shares why he chose a clinical trial
Michelle Hines
Breast cancer survivor participated in three clinical trials
Nicole Adams
Clinical trial gives recurrent glioblastoma survivor hope
Julia Dutton
Why a leukemia & melanoma survivor chose clinical trial
Don Kendall
Stem cell transplants success for multiple myeloma survivor
Jasmine Ross
College student takes on clinical trial for leukemia therapy
Debbie Ann Heckeroth
Clinical trial keeps stage IV thyroid cancer survivor stable
Chuck Caldwell
Throat cancer survivor chooses trial to 'help someone else'
Jessi Hooks
Survivor joins clinical trials after cancer returns
Sherry Zorzi
Clinical trial reveals early-stage lung cancer
Learn More About Clinical Trials
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.
Bringing patients new and better cancer treatments through clinical trials is what sets MD Anderson apart. It's more than a goal or a point of pride. It's a passion. And we offer more cancer trials than anyplace else.
Clinical trials are the key to developing new cancer treatment
options. Advances only reach patients by going through clinical
trials, which are the final step in a long process to find better ways
to prevent, diagnose and treat cancer.
"People's health is at stake," says Hagop Kantarjian, M.D., chair of Leukemia.
"So trials are conducted only after the procedure or medication
has passed many steps that provide confidence it's better than what's
available as standard of care. Trials are meticulously designed,
reviewed and monitored. Their importance and complexity require
expertise and supporting infrastructure, and MD Anderson has no equal
in these areas."
Saving lives through clinical trials
Kantarjian personally has conducted 345 clinical trials in his 34
years at MD Anderson. He says he's motivated and inspired by the
people he works with, as well as our patients.
"MD Anderson is the best place in the world because of
everyone's incredible will to do good by making a difference for so
many patients," Kantarjian says.
One of his clinical trials made a life-saving difference to Ken Geihsler.
In December 1999, things weren't looking good for Ken.
"Back then, the treatments for my cancer -- chronic myelogenous leukemia (CML) -- didn't
work very well," Ken says. "The only option left where I was
being treated was a stem cell transplant, and only 20% of people my
age survived. Then I read about this magic pill called Gleevec, but it
was only being tested in a few places in the country. MD Anderson was
one them."
Ken saw Kantarjian and joined the Gleevec clinical trial on a
Friday afternoon. He was able to get the pills that evening thanks to
extra efforts by Mary Beth Rios, research nurse manager in Leukemia.
"Gleevec saved my life," Ken says. "And when it
stopped working for me a few years later, new drugs available through
other clinical trials extended my life."
Clinical trials help surgeon do more by doing less
When you learn about the research of Kelly Hunt, M.D., professor in Surgical
Oncology, it may sound like she's working herself out of job. She's a
breast cancer surgeon whose many successes to date are related to
doing less (surgery, that is) for breast cancer patients.
"During my general surgery training just 20 years ago, many
women learned they had breast cancer by waking from their biopsy
surgery and finding their entire breast removed," recalls Hunt.
"I've dedicated much of my career to finding less invasive, yet
more effective procedures."
Some breast cancer patients avoid surgeries altogether thanks to
Hunt's work. She was part of a study that helped doctors and patients
make more informed decisions about removing the unaffected breast as a
preventive measure. Now many women feel confident about not having
this surgery.
Hunt led another clinical trial that focused on lymph nodes. It
showed some patients can safely avoid an additional surgery (complete
axillary lymph node dissection), as well as the potential serious side
effects of that surgery.
This trial changed the way many patients are treated by doctors
everywhere. But Hunt says there's more work to be done.
"We owe our patients more," she says. "Great
strides have been made, but much work remains, and it's rewarding to
be among so many here who are driven to help patients through clinical trials."
Hope for tomorrow
Margaret Cowart was diagnosed with the rare blood cancer
polycythemia vera in 2007. In 2013, the standard drug no longer kept
her disease in check. Thankfully, her doctor, Srdan Verstovsek, M.D., Ph.D., professor in
Leukemia, had another option.
"I'm so fortunate Dr. Verstovsek and his clinic team were
ready to move my treatment to a new option they were
investigating," says Margaret. "In the months I've been on
the trial, the drug seems to be working better and the side effects
are less."
Cowart says she's hopeful for the future and proud to participate
in the trial.
"I feel like I'm part of the process to make things better
for tomorrow," she concludes.