Why Clinical Trials Matter
An Interview with Sal Bertolone, MD

  • Sal Bertolone, MD
  • Chief of Operations
  • Pediatric Oncologist
  • Kosair Children's Hospital

Each year, approximately 75% of children diagnosed with cancer are enrolled in clinical trials as part of their treatment. Recently, CureSearch talked with Sal Bertolone, MD, Chief of Clinical Operations and pediatric oncologist at Kosair Children’s Hospital in Louisville, KY about the importance of such trials.

Clinical trials are the main way children with cancer are treated, why is that?
The success we’ve had in treating and curing children with cancer, and beginning to reduce toxic side effects of treatment, is attributable to clinical trials. These trials enable us to take proven medications and study them further to find better individualize patient treatment and cures. That’s an important thing to understand - these are proven drugs being studied to see we can best use them, not brand new drugs that have never been tested before.

So, what you are saying is that children don’t get placebo medication, they get drugs you know work?
That’s correct. In Phase III clinical trials, we are trying to figure out which drugs of those that are already out there are the most effective with the least side effects. If you look at Wilms Tumor for example, decades ago there were two medications each known to treat the disease. Experts disagreed about which was “the best.” Thanks to a clinical trial that tested the two against each other and the two in combination, we know that giving children both drugs brings cure rates upwards of 90%. In addition, we’ve learned over time that we no longer have to treat kids with Wilms Tumor for 2 years. In fact, we can treat them for 6 months. This was also discovered through clinical trials and means that children have fewer side effects of treatment, because they receive 6 months of chemotherapy instead of 2 years.

How have clinical trials changed in the years that you have been treating children?
One of the biggest changes has to do with initial surgery to remove the tumor. At surgery we now save tissue to not only diagnosis the tumor but also to better understand its biology and genomics (genetic make-up). We are able to take the tissue (or blood in the case of liquid cancers) and study it and compare it to other patients’ tissue of the same cancer. So, we now have banks of tissue and data related to all of the samples that provide us with information about the biology of the tumors. This is leading researchers to understand the genetic footprints of many types of cancer which in time, will lead to testing new medications to treat and hopefully cure, children with cancer.

What does the future hold for clinical trials?
We are about 25% of the way to the next generation of clinical trials in which I expect we will sub-characterizing children into needing more or less therapy based on the genomics of their individual disease. For example, if two children present at the same time with neuroblastoma, we will be able to look at each of their cancers in the laboratory and we will see how the molecular and genetics of neuroblastoma are different in both children. Based on that information, we will customize – or individualize – the treatment to each patient so it can be as effective for that specific child as possible.

Christine Bork
Email Christine
(800) 458-6223

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