All drugs that are studied in a pediatric research trial have been tested many times over in the lab and on adults before they can be tested in a pediatric patient. Most of the time, there are hundreds, if not thousands of adults who have been treated with the drug being tested in children. In many cases, the drug or drugs may even be approved by the US Food and Drug Administration for use in adults, or in a different type of cancer than your child has.
What might be "experimental" about the research study is:
This also means that we probably know quite a bit about the expected side effects of a given drug or combination of drugs. The known side effects may be similar or different in children compared to adults. In general, children tolerate most medications better than adults, but not always.
Similarly, children may have different side effects because they are growing and such side effects might not be noticed in fully grown adults in whom the drugs have been used before. In all research trials the primary concern is the safety of the patient. In a Phase I trial, the dose of the drug(s) is usually increased a little at a time. The drug dose will not be increased until the patients treated at a lower dose have shown they can tolerate the study drug without any serious side effects.
Safety is always the number one priority for all patients on a clinical research trial. At any time, the choice to stop participating in the study is yours and your child's. If the study physician is concerned about a patient's safety, they may take the patient off the study, even if the family or patient wants to continue to be treated. The first and most important goal is to ensure your child's safety.
We do not offer studies because they are "interesting" to us, or because we are being paid to give the drug to your child. Part of our promise to patients includes only offering those drugs that make sense for your child's type of cancer based on everything we know to date. We may not know in advance whether that drug will be effective or what side effects your child might experience – but we can promise that we would never offer treatment if we did not believe it might be beneficial to your child.
However if you are still concerned that your child will be treated as a "guinea pig", then a research trial may not be the best choice. We will always work to make sure that your child is getting the best available treatment – even if that means that there is a better treatment option at another hospital or medical center.
Before any research trial can begin, it has to be reviewed by many committees to determine its safety and fairness. Some of these committees are local, within the hospital or university, others are national review boards. After a research trial has begun to enroll patients, it is reviewed at least once a year formally. During the therapy, patients are assessed to determine that they are not experiencing any serious side effects.
The doctors and nurses who are participating in the trial often talk by phone and exchange information with each other to better understand what is happening with patients in the study. Usually these conversations take place at least once a week, sometimes every day. The reason for this is to make sure that everyone participating in the research understands what side effects are being seen in all the patients. This helps us make sure your child is being treated as safely as possible.
The medical team also talks about the results they are seeing in the patients being treated. If there is any new information about safety or responses in patients, you will be told about them.
Some people who take part in a Phase I or II research trial believe there will be therapeutic benefit from the drug being tested. This is known as a "therapeutic misconception". Phase I and Phase II trials are not considered "therapeutic" trials. This means that your child may not benefit from the study drug at all. Phase I trials are conducted to determine the safe dose and to learn about the side effects of a drug. Phase II trials help to determine if the drug if effective against a certain type of cancer. While we design these studies to make sense based on the biology of your child's cancer, and are hopeful that the drug might help your child, there is no promise that your child will benefit at all. We will try to offer your child a treatment that we think may offer some benefit, based on what we know about the drug, and about your child's cancer, but there is no guarantee that it will help.
The newer drugs that are being developed often try to take advantage of something about a cancer cell that is abnormal. If that abnormality is shut down, but it is only present on cancer cells, then the new medicine may work on the cancer cells, but not the healthy normal cells. This means that a lot of the usual side effects will not occur, or will not be as bad as with many traditional chemotherapy drugs.
Many medicines, especially many of the newer cancer drugs, are only available in a pill or liquid form, and are as effective or more effective than the IV form. Just because your child is getting an oral medicine doesn't mean it might not be effective.
With the more traditional chemotherapy medicines, this is usually true. With many of the newer medicines, a higher dose may not be any better. If the medicine shuts off an important path in a cancer cell, that is all that matters. If more of the drug is given, it may not shut down that path any better. Sometimes, higher doses only mean that there are more side effects but not necessarily any better chance of benefit.