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Promising New Treatments

Major improvements in treating children with cancer have been made in the past 50 years.  This may be due to the fact that over 50% of children diagnosed with cancer enroll in clinical trials compared to just 5% of adults diagnosed with cancer.  Promising new treatments are typically first available through clinical trials.

You may be offered a clinical trial by your medical oncologist.  Clinical trials often separate patients into two groups: one who will receive the current ‘gold standard’ or therapy we know works best vs. a new treatment that showed promise in earlier studies.  

In addition, you also can search for new treatment options by looking for options both nationally or internationally via the clinicaltrials.gov website.  This website enables you to enter the cancer type, location (if you want to limit travel) and keywords you may have heard like “immunotherapy’ or others to narrow your clinical trials search.  Hint: click on “Recruiting and Not Yet Recruiting” to narrow your search to the studies that will most likely be open to enrollment now.

What are Clinical Trials?

Clinical trials are experiments involving people (‘human subjects’) to try to understand multiple aspects about treatments or diseases that were previously unknown.  In patients with cancer, this typically involves receiving a novel type of treatment that may help doctors establish a new clinical standard. Clinical trials want to determine:

  1. The optimal dose of the medication
  2. The optimal schedule (how frequently should the drug be given to be effective but result in the fewest side-effects)
  3. Can or should this treatment be combined with other known treatments to achieve better clinical outcomes?
  4. What are the side-effects associated with this treatment?
  5. How effective is this treatment compared to other treatments that are already available?

Before new treatments are given to people, a lot of scientific work is done to make sure that clinical trials are testing the best scientific advances have to offer.  There are cell studies, animal studies, and finally Phase I, Phase II, and Phase III studies.

Phase I:

  • Establishes optimal dose
  • Assesses how the treatment interacts with the human body
  • Evaluates toxicity 

This tries to establish the dose that is most effective with the least side-effects.

Phase II:

  • Compares this new treatment to other treatments
  • Attempts to determine how much better (or not) this new treatment will be

 Questions to consider:

  • Is it much more effective?
  • What are the significant side-effects?
  • Are there additional medical needs that this treatment helps to meet?

Phase III:

  • Compares the new treatment to the current standard of care
  • Larger studies
  • Usually done at multiple institutions

The Cost of Research:

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