Lucia during her stay at Weill Cornell Medical Center and Memorial Sloan Kettering Cancer Center

Lucia during her stay at Weill Cornell Medical Center and Memorial Sloan Kettering Cancer Center



Cancer Treatments

The types of treatment that a child with cancer receives will depend on the type of cancer and how advanced it is. Common treatments include: surgery, chemotherapy, radiation therapy, immunotherapy, and stem cell transplant.  Most often, a combination of treatments are used.

If or when, the typical treatments are ineffective then a clinical trial may be the best or only choice left. 

Clinical Trials


Most treatments we use today are the results of past clinical trials.

Any time you or a loved one needs treatment for cancer, clinical trials are an option to think about. Trials are available for all stages of cancer. It is a myth that they are only for people who have advanced cancer that is not responding to treatment.


  • Clinical Trial: A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called clinical study.
  • Principal Investigator: Every trial has a person in charge, usually a doctor, who prepares a plan for the trial, called a protocol.
  • The Protocol: explains what will be done during the trial. It also contains information that helps the doctor decide if this treatment is right for you. Which drugs will be given, the tests required and who is eligible to participate depending on age, disease etc. 

Phases of Clinical Trials

Clinical trials to test new cancer treatments involve a series of steps, called phases. If a new treatment is successful in one phase, it will proceed to further testing in the next phase.


During the early phases (phases 1 and 2), researchers figure out whether a new treatment is safe, what its side effects are, and the best dose of the new treatment. They also make sure that the treatment has some benefit, such as slowing tumor growth.



  • To find a safe dose
  • To decide how the new treatment should be given (by mouth, in a vein, etc.)
  • To see how the new treatment affects the human body

   Number of people taking part: 15–30




  • To determine if the new treatment has an effect on a certain cancer
  • To see how the new treatment affects the body

   Number of people taking part: Less than 100




  • To compare the new treatment (or new use of a treatment) with the current standard treatment

   Number of people taking part: From 100 to several thousand


In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid.


There are also very early (phase 0) and later (phase 4) phase clinical trials. These trials are less common. Phase 0 trials are very small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness. They take place after a new treatment has been approved and is on the market.

Some researchers design trials that combine two phases (phase 1/2 or phase 2/3 trials) in a single protocol. In this combined design, there is a seamless transition between trial phases, which may allow research questions to be answered more quickly or with fewer patients.


A Word about Placebos

Though placebos are rarely used in cancer treatment clinical trials, they are used when there is no standard treatment. Or, they may be used in a clinical trial that compares standard treatment plus a placebo, with standard treatment plus a new treatment.


The placebo is designed to look like the medicine being tested, but it is not active. You should always will be told if the study uses a placebo.  The doctor most likely won't know either if your child is in the experimental drug group or the placebo group.

Finding Clinical Trials

-        The Children’s Oncology Group (COG) is the largest pediatric clinical trials group, with more than 200 participating hospitals throughout the world. To search the COG clinical trials, click here.


-        The National Cancer Institute (NCI) maintains a comprehensive list of cancer clinical trials that includes most trials sponsored by the NCI. It also contains many clinical trials sponsored by pharmaceutical companies, medical centers, and other groups from around the world. To search the NCI clinical trials, click here.


  • Treatments & Clinical Trials

    Pediatric Cancer Statistics

        Cancer Basics

        Types of Cancer

        Research News