Childhood Cancer

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Expanding access to new agents for children with refractory cancers: the experimental therapeutics program at Children's Hospital of Colorado.

Children’s Hospital Colorado
Lia Gore, MD
Grant Type: 
Phase I/II Infrastructure Grants
Year Awarded: 
Type of Childhood Cancer: 
General Pediatric Cancer
Project Description: 

“We have been able to offer promising treatments to the children and families who need them most. There are kids who are alive today who would not be without the treatment that we were able to offer.” - Lia Gore, MD


The majority of children diagnosed with cancer today will be cured. Unfortunately some, including those with refractory solid tumors and some brain tumors, continue to have a particularly dismal prognosis that has not changed for over 30 years. The surge in research on childhood cancer has frequently excluded patients with brain tumors due to their complex physical and emotional challenges. Ironically, these are the children who need new treatments most desperately.

Project Goal

To conduct phase I and II trials of a variety of novel cancer therapies that have shown promise in early work.


I understand the grants were to help support the Pediatric Experimental Therapeutics Program both in terms of expanding access to new agents and generally advancing program development and support. Can you provide me some examples of how this has been successful?
With support from Alex’s Lemonade Stand, we have been able to open more trials and to conduct trials that have novel biological studies embedded within them, to allow us to better understand the impact of specific new treatments on the “drivers” of various pediatric cancers. With this support, we could accommodate more patients on studies, increase the research nursing support, and open more clinical trials to better address the unmet need of enhancing access to the children who need better therapies the most.

What does this mean for children with cancer and their families?
More options for patients and families, the opportunity to get new promising therapies closer to home instead of having to travel to another institution and the ability to be one of just a handful of centers around the world to open some trials.

Next Steps

To continue to fund the program for its incredible growth. The sustaining of funding is the biggest challenge we have because traditional granting mechanisms don't pay for the things of greatest need (nursing staff, research staff, regulatory staff). Without these staff in place at all times, the ability to treat a patient when the need arises, with the most promising available therapies, is severely compromised.


Lia Gore received a 2013 Phase I/II Infrastructure Grant.