The purpose of a clinical trial is to find a better way to prevent, diagnose or treat a disease. Clinical trials are part of an ongoing, careful research process. It is through this continual process that new treatments have improved cure rates and decreased toxicity for patients with many forms of cancer.
Types of Clinical Trials
If you care team asks you to participate in a clinical trial, that means they have decided that the trials offers the best treatment for your child's condition. Some trials test whether a new treatment is safe to give to patients (Phase I). Others seek to determine how effective a new treatment will be (Phase II). Finally, the largest trials make direct comparisons between two similar treatment options, to determine which option is better (Phase III). All three types of trials provide critical information to help us improve cancer care. Many standard treatments today are based on the results of previous clinical trials.
Why participate in a clinical trial?
Participating in clinical trials is voluntary. You can choose to stop participating in a clinical trial at any time. Your doctors and nurses want to give you the very best chance for cure. If your treatment is no longer the best option for you, both you and your doctor will discuss other options. If you decline to participate on a clinical trial, your doctor and medical team will still take the very best care of you. You will be offered the standard-of-care therapy, which is the best known treatment at that time.
The Children's Cancer Hospital at MD Anderson, through its Pediatric New Agents Program, is at the forefront of developing innovative treatments for a wide range of pediatric cancers and moving them from the lab to the clinic for maximum benefit to young patients.
You can read more about clinical trials on our main clinical trials site.
MD Anderson has close to 2,000 doctors. Several hundred never treat
patients, yet they are crucial to our mission.
"Our mission is to end cancer, not just provide excellent care," says Helen Piwnica-Worms, Ph.D., vice provost of Science. "We don't yet know enough, so our faculty must include a robust community of researchers who apply their scientific expertise to answer important biological questions."
According to Piwnica-Worms, delivering on our commitment to finding answers sets us apart.
"Discovery is what distinguishes breakthrough institutions," Piwnica- Worms says.
Birthplace of new cancer treatments
Basic research, also referred to as laboratory research and discovery science, is a part of MD Anderson's DNA. Ending cancer requires investments in this type of research, as well as clinical, translational and population sciences research. Many of today's treatments exist because of yesterday's basic research. For example, many patients with advanced pancreatic cancer rely on the drug gemcitabine, which is available to them thanks in large part to basic research conducted by William Plunkett, Ph.D., professor in Experimental Therapeutics.
Plunkett's discoveries of the metabolism, mechanism of action and clinical pharmacology of the drug led to the rationale for fixed-dose-rate infusion. His work complemented trials conducted by our clinical doctors.
As a result, in 1996, gemcitabine was the first drug for pancreatic cancer approved by the Food and Drug Administration (FDA).
Had the science stopped there, only pancreatic cancer patients would
have benefited. Thanks to continued research, the drug has since been
FDA-approved to treat forms of lung, breast and ovarian cancers.
Ongoing research has led to its testing as a treatment in even more
One discovery builds on another
Leukemia patients around the world also have benefited from Plunkett's discoveries, many of which were made available to patients through collaborations with Michael Keating, M.D., professor in Leukemia, and co-leader of the Chronic Lymphocytic Leukemia (CLL) Moon Shot. For those with acute myeloid leukemia, he demonstrated that lowering the dose of the drug cytarabine was just as effective and less toxic to patients.
Plunkett's discoveries of how the drug fludarabine worked in the body led to testing it for patients with CLL. The drug prompted a more effective response and survival benefit over what was considered the best treatment at that time.
But the research didn't stop there. Further laboratory work led to combining fludarabine with the drug cyclophosphamide, which proved to be even more beneficial to patients. The combination became the new standard of care worldwide.
"It's very rewarding when your laboratory work leads to benefits for patients," notes Piwnica-Worms, who speaks from personal experience. Her pioneering discoveries in cell cycle checkpoints have been translated into new therapeutic opportunities for cancer patients, particularly for those with triple-negative breast cancer, ovarian cancer, and head and neck cancers.
"We must continue to support discovery science," Piwnica-Worms states. "When I was in training, the field focused on the cancer cell itself, but through continued basic research, we now recognize that it's critical to think of cancer as an organ and to study the cancer cell in the context of its surrounding microenvironment."
We can do things now -- like sequencing whole genomes -- that weren't even ideas 30 years ago, Piwnica-Worms says.
"With the pace of science today, we can't even fathom what we'll be working on 30 years from now. We must continue to position ourselves to capitalize on new technologies and opportunities as they arise."
A longer version of this story originally appeared in Messenger, MD Anderson's bimonthly publication for employees.