Pros and cons of clinical trials for children

Watch Stuart E. Siegel, MD's video on Pros and cons of clinical trials for children...
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Pros and cons of clinical trials for children


Clinical trials are a very important element of the treatment of childhood cancer. First of all, all the progress we have made in the treatment of childhood cancer, which is considerable, have come from clinical trials. Testing new treatments and new approaches to treating children with cancer. But there are situations where a humanized mouse model is used instead. In these cases, a mouse has been genetically modified to have a human version of the gene involved in the disease. That's how we've learned. How to get where we are now. But, obviously we are not done. Because, we need to find treatments for the kids we can't cure. And, we need to find less toxic treatments for the ones that we can effectively treat. So, clinical trials are critical. And participation in critical trials is very important for the future of pediatric oncology. For the individual patient, the reason I think the clinical trial is important is: 1. Clinical trials are very specific in outlining the therapy and exactly how it's to be given; in what sequence; in what dose; when you need to make modifications; how you modify it. That's the basis of the results that we have from the past that we use now to treat the patients. So, in another words, you're getting the very, very best exact treatment out of a clinical trial. The other reason that could be important for the trial is that a new treatment maybe turn out to be better then the old treatment. So, a clinical trial usually involves comparing the best available treatment from the last studies we did to something a little bit different that may be better. It may not turn out to be better. Rarely, rarely does it turn to be worst. So, you have an opportunity, at least, to be part of a study that you might get a treatment that is going to be better. On top of contributing to the future knowledge of the next patient or the next year patient that come in and getting treated perhaps with the better treatment. So, the other thing is that the clinical trials are done at the Centers that have to pay us very rigorous standards of how to treat. And, by both local, national, even federal guidelines. So, you know you are being treated at a place that has the absolutely best treatment programs and has all the necessary tests and ability to do this. I think clinical trials are important. However, I will say that it's very important for a patient, a family to understand the clinical trial. If they do feel, for some reason, uncomfortable about the trial that they do not want to be part of, which may use one treatment or the other, based on some random assignment. That they really want the best available current therapy, then they have the right to choose that. And, that's okay to. But, just to put forward, what the advantages of a clinical trials are. For other patients and potentially possibly for the child themselves. It's up to the family to decide which way they want to go. And, I would say that the majority of the patients that we offer clinical trials to and their families, they accept them. But, there is a significant minority who decide to use the best available therapy. Which we obviously provide to them.

Watch Stuart E. Siegel, MD's video on Pros and cons of clinical trials for children...


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Stuart E. Siegel, MD

Director, Children’s Center for Cancer & Blood Diseases, Children's Hospital Los Angeles

Before recently shifting his focus to international medicine, Stuart Siegel, MD, was Chief of the Division of Hematology/Oncology for 35 years and the founding director of the the Children’s Center for Cancer & Blood Diseases at Children’s Hospital Los Angeles, and Professor and Head of the Division of Hematology-Oncology Department of Pediatrics, Keck School of Medicine of University of Southern California. He remains a leader in supportive care and research in pediatric oncology, with a special focus on neuroblastoma, non-Hodgkin lymphoma, Ewing Sarcoma, acute lymphoblastic leukemia and brain tumors. From developing the first pediatric protective environment in 1971 for children undergoing intensive chemotherapy, to pioneering current efforts to develop academic and clinical care programs nationally and locally for adolescents and young adults with cancer, Dr. Siegel’s contributions have revolutionized the field of pediatric oncology. Dr. Siegel has been honored for his work by the American Cancer Society, Children Foundation, the Cancer Foundation, the Chase Foundation, Padres Contra El Cancer, the Israel Cancer Research Fund and Ronald McDonald House Charities, where he is a member of the National Board, and has consistently been listed among the nation’s top doctors in such publications asAmerica’s Top Doctors and Best Doctors in America. He is a father of one son, Joshua; grandfather of David and Elijah; and lives in Pacific Palisades with his wife of seven years.

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