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Winthrop University Hospital

Understand Childhood Cancers and Blood Disorders

Clinical Trials / Treatment Protocols

If your son or daughter was just diagnosed with cancer, we understand how worried, upset, helpless, and confused you must feel. We want you to know that all childhood cancers are treatable and our entire staff is committed to achieving the ideal goal - a cure. Nothing else matters to us more than that. During the next few days you will hear a great deal of information about your child's illness, about the treatment, and its side effects. Many words, terms, and ideas will be presented to you in a very short time span. Even if you are an expert in medicine, it is important to explain everything to you as clearly as possible. We believe that when you have a better understanding of all the new words and terms, you will become our partner in that all-important task of curing your child. We embrace the notion that "knowledge is power" and are willing to explain everything to you clearly and as often as you need. Please know that there is no such thing as a "stupid question" and there is no such thing as "bothering" anyone with questions.

One confusing term that you may be approached about would be the enrollment of your child in a clinical trial (or treatment protocol). Among the many concerns of a parent of a child with a newly diagnosed malignancy is that no parent wants his or her child to be a "subject in an experiment" or even literally become a "guinea pig". The purpose of this section is to clarify exactly what is meant by the words "clinical trial" and "treatment protocol" so that your questions and worries can be addressed openly and in an understandable fashion.

Cancer clinical trials (also called cancer treatment studies, research studies, or treatment protocols) test new treatments in children with cancer. Clinical trials test many types of treatments such as new drugs, new approaches to surgery or radiation therapy, stem cell transplants, new combinations of treatments, or novel methods such as biological therapy. The goal of this research is to find better ways to treat children with cancer. As part of this process, we will discuss the treatment plan with you in great detail, including the potential side effects. All of this information and more will be contained in the consent form.

Childhood cancers showed some of the greatest improvements in cancer survival during the past 20-30 years. The current five-year survival rate of over 75 percent (for all children with all cancers) confirms the substantial progress made since the early 1960's, when childhood cancers were nearly always fatal.

Fortunately, cancer is a rare disease in children, being diagnosed yearly at a rate of about one in 7,000 children between birth and 14 years of age in the United States. Some cancers have very impressive overall survival rates (e.g., Wilm's tumor, a tumor of the kidney, with a prognosis well over 90 percent) but not all cancers have such excellent prognosis. The limited number of children diagnosed with cancer annually (12,000-14,000 cases yearly) makes it very difficult to come up with the best treatment option for each cancer type. In order to prove that one treatment approach is superior to the alternative, one must convincingly demonstrate that fact statistically. To that end, one must treat a large number of patients in order to show that the difference in outcomes between the two treatments is not coincidental but is indeed true. Pediatric oncologists (pediatricians who specialize in treating children with cancer) realized that in order to find the best treatment choice for a given cancer, they had to unite and share their experiences and information as no one center sees enough patients to draw strong conclusions. Children's Oncology Group (COG) is a national organization with more than 240 participating children’s cancer centers, that collects information about all children who are diagnosed with cancer in the US, as well as other countries (notably, Canada, England, France, Germany and others). The information that is collected encompasses not only treatment results, but also biological data collected from each and every tumor. That way, all COG members not only share their results in treating children with cancer but also contribute an invaluable resource to the scientists who are working hard to find ways to kill such tumors. While it is possible to create cancer in animals and obtain some information that way, there is nothing more effective than working with real tumors in humans. The years of collaborative experience gained by our national organization has led directly to the successful treatment of many current cancer patients.

Cancers behave differently because they have different biological properties. Therefore, it is no surprise that treatments differ according to the specific cancer diagnosis, with therapy based on how well children in the past fared on different treatments. The previously acquired experience or knowledge is the basis for the design of our clinical trials. They are not designed randomly and they only try to refine and improve on what has worked previously. In certain trials we compare two (or sometimes more) similar treatment strategies (also called "study arms") that differ slightly from each other. The purpose of such trials is either to identify a superior treatment (that would result in better cure rates) or seek out the treatment resulting in fewer side effects. If we knew ahead of time which study design (or "arm") was superior or resulted in lessened side effects, there would be no need to even conduct that trial. However, though much progress has already been made, there are still many open questions that need to be resolved. In some cases, we are still not satisfied with the cure rates and are therefore trying to achieve better survival rates. In other cancers where we already have good success rates, we are trying to minimize the side effects.

There are different types of clinical trials, called Phase I, Phase II, and Phase III trials. Each is one of the final stages of a long and careful cancer research process. Before any new drug is introduced, it must undergo a lengthy and rigorous process of development and testing, first in the laboratory and then with animals. Most drugs are also first tested in adults before they are brought into clinical trials involving children.
  • Phase I: These are the earliest human trials in the life of a new drug or treatment. They are usually small trials, recruiting few patients. The trials may be open to children with any type of cancer. Patients enrolled into phase I trials are usually those with advanced cancer that failed all known treatments. The specific aim of a phase I trial is to find out the safe dose of the new drug, and its side effects. The most typical phase I trial is designed such that each patient who is enrolled receives a slightly higher dose of the drug. All side effects are recorded, and the process of increasing the dose continues until the doctors find the maximal dose that can be tolerated by a child. Naturally, doctors who conduct such trials hope to witness a good response and shrinkage of the tumor. Some patients may benefit from such a trial, but unfortunately, many will not.
  • Phase II: These trials arise from promising or successful phase I trials and may be open to patients who share the same diagnosis or those having different types of cancer. These trials tend to recruit more patients than phase I trials. The specific aims of this study are to find out:
    • Which cancer types the new treatment is best suited for
    • More about the proper dosing of the particular drug
    • More about ways to handle the side effects of the new drug
    • If the drug is promising enough to proceed to a phase III trial
    If the results of a phase II trial show that the new drug or treatment may be as good, or perhaps even better, than currently used treatments, it advances to a phase III trial.
  • Phase III: These trials compare the new treatment (the one that just made its way through phases I and II) directly against the best currently used treatment (also known as "standard treatment") for a certain cancer. The comparison is usually direct or "head to head" in order to prove which treatment is superior. Even if the survival rates do not differ, doctors can still learn which of the treatments results in fewer side effects. Phase III trials may compare treatments in various ways, such as:
    • New treatment against the current or standard treatment
    • Different doses, ways, or schedules in which to give an established or standard treatment
    • Compare treatments that are fixed against treatments that are flexible and dependent on certain measurements or tests of response done during the treatment
  • Phase III trials are generally larger because one must enroll many patients in order to demonstrate that a certain approach is superior to another in a statistically significant manner. These studies, which typically last a few years, are necessary to prove beyond any doubt that one treatment is clearly superior to another, and that differences in outcomes are real and not purely accidental or as a result of sheer luck. Most children on studies are enrolled into phase III trials and have the assurance of either receiving a previously established treatment or is treated according to a new or slightly different method that has successfully passed phases I and II trials.

What are the benefits to your child's enrollment into a clinical trial?

  1. Clinical trials provide top rate cancer care.
  2. The new trial is based on previous experience.
  3. Side effects have been identified and their treatment is better known.
  4. If the new strategy indeed proves to be superior to the standard treatment, your child may be one of the first to benefit from that success. Even if the new treatment only works as well as the older treatment, your child could still benefit from lessened side effects.
  5. Participation in a clinical trial gives your physician easy access to the national committee to discuss any problems your child is having. Likewise, as a participant, one can easily continue treatment at another COG center if one relocates.
  6. Many new clinical trials or protocols now offer highly sophisticated laboratory tests that may affect treatment. Those specialized tests are almost always available only to children enrolled in a particular trial.
  7. While health insurance companies and health managed organizations (HMO's) are often reluctant to cover the cost of novel drugs, the new medication will still be provided to your child if he or she is assigned to the study arm containing the new medication, at no extra cost to you.
  8. You are helping other children in the future who may be diagnosed with the same type of cancer as our own child.
One of the key concepts in having your child enrolled in a clinical trial is the process of randomization. This means that the research study divides all the patients into two (or more) groups randomly so that both groups consist of approximately equal makeup. One group receives the new treatment whereas the other receives the standard (or older) treatment. Since we do not know which group will fare better, there is no way for us to determine which child should be assigned into which group ahead of time. Furthermore, by randomizing patients into treatment groups we avoid a bias. Sometimes doctors can be biased (even subconsciously) either for or against a certain type of treatment. If assignment into treatment groups is left to the treating doctors themselves, there would be the possibility of biased, misleading conclusions. These trials take several years and it is extremely important that we let the truth emerge on its own so as not to risk the mistreatment of future patients.

What are the disadvantages to your child's enrollment into a clinical trial?

  1. New treatments may not always work better than the old treatments.
  2. Even if the new treatment proves superior to the old treatment, it may still not work for your child.
  3. If your child is assigned to the standard group and if the new treatment proves superior, your child may not benefit from such results. (Your child may still do very well since the older treatment has been the standard of care that has been in use up until the newer treatment went through phases I and II.)
Before deciding to have your child enrolled in a clinical trial you will have ample opportunity to discuss important issues with the doctors. We encourage you to read the details included in the consent forms. It is also wise to write down a list of questions, terms, or issues you wish to clarify with the doctors when you seem them the next time. Examples of such may include:
  • The short- and long-term risks, side effects, and benefits related to the trial.
  • The effect of the trial on your child's daily life and school attendance or performance.
  • Your financial responsibility for any treatment, tests, or other charges.
  • Your wish to seek a second opinion regarding the diagnosis and/or treatment choices.
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