Children's Cancer Therapy Development Institute: A New Attempt at Bridge-Building for Childhood Cancer Research

We live in an era of immense hope for the future of cancer research and the quest for new and promising treatments. The identification of new genetic mutations as viable markers for therapeutic development is driving researchers and clinicians to focus on specific pathways for targeted therapies. Scientific sequencing and analysis to locate these mutations is becoming more reasonably priced. Accordingly, more people diagnosed with cancer rely upon precision medicine to treat their disease. President Obama, in the recent 2015 State of the Union Speech, even called for the government to provide new research funding for precision, or personalized medicine.

Consequently, more people with cancer today have renewed and increased hope for survival. Unfortunately, this was not my family's personal experience when my wife and I were told about our two-year old daughter's terminal brain cancer in April 2008.

For children diagnosed with cancer, the number one cause of death by disease in the United States, amazing progress has been made over the course of the past thirty to forty years. Thankfully the result is that for many children with cancer there are effective treatments and protocols, and five-year survival statistics have increased dramatically. Unfortunately, for one in five children with cancer, overall survival numbers for those specific cancers have stagnated with little to no improvement.

There are some forms of childhood cancer that have experienced few treatment advances in the past thirty to forty years. When our daughter Alexis was diagnosed in April 2008 with DIPG, or diffuse intrinsic pontine glioma, an inoperable brain tumor located on the brainstem, we were told at diagnosis that she was terminal. As we learned more about the tumor overall, the lack of effective treatment options was particularly disturbing. Alexis, despite a long and inspiring battle, died on January 14, 2011 two weeks shy of her fifth birthday. She is considered a longer-term survivor of DIPG. For other forms of childhood cancers, the prognosis is only slightly better, and recurrence generally means that there are few effective treatments available.

The slow pace of translation for promising research from the laboratory to the clinic has created what is known as the "pre-clinical gap" or also referred to as the "valley of death." In simple terms, this means that much of the basic scientific research being done is not seeing the inside of a clinic and used to treat children. This so-called gap continues to grow ensuring that new drugs and clinical trials are not reaching the patients. Translational and pre-clinical research unfortunately has become more difficult to fit within the traditional academic research model. The basic translational work that is the backbone of pre-clinical discovery is tedious, challenging and expensive within this paradigm. Another significant impediment is the grant to paper to grant model that slows or often ensures that the research never sees the clinic. Accordingly, current research models and the pace of development of promising new drugs and clinical trials for children with cancer are failing the one in five who do not survive.

The good news is that there are researchers who are seeking to bridge this widening gap. One such researcher undertaking significant strides to accelerate the pace of research and build a bridge across the valley is Charles Keller, III, MD. Dr. Keller recently made the bold decision to leave his well-respected and well-funded laboratory at Oregon Health & Science University, and put it all on the line by founding a 501(c)(3) non-profit laboratory known as Children's Cancer Therapy Development Institute, or cc-TDI for short, in Fort Collins, Colorado. ( Dr. Keller seeks to create a unique independent non-profit laboratory, inspired by the pace of the private biotech industry, by removing some of the impediments that exist as obstacles to translation of the research from lab to clinic. cc-TDI has one mission: to bridge this gap. In short, the research at cc-TDI will be aimed at testing new and existing drugs against newly identified and known targets in childhood cancers in an effort to create more effective clinical trials.

As a nation, and as a planet, we as humans have much to be hopeful for with respect to the treatment of diseases once thought incurable. It seems every day you read the paper or turn on the news there is a new discovery. These discoveries lead to the creation of treatments that provide hope. And that is what I truly believe cc-TDI represents, and why Dr. Keller took a jump without a net. Hope. It is hope that is necessary for the next set of parents who hear those four chilling words, "your child has cancer." And it is hope in the form of these selflessly dedicated childhood cancer researchers like Dr. Keller that will allow the next set of parents to fight for the life of their child with utter determination and resolve. Yes, there is hope.