Innovator Spotlight

Q: What prompted the creation of The Nicholas Conor Institute?
In 2005, when our son Nicholas Conor was 16 months old, he was diagnosed with high-risk neuroblastoma, a deadly childhood cancer that arises from immature nerve cells outside the cranium. Much like other solid cancers in children, the "one-size-fits-all" treatment was and still is chemotherapy at levels greater than what an adult would receive and in combinations that render most of the children with lifelong side effects and a decrease life expectancy if the child survives. Unlike most parents who are told, "Your child has cancer," we were cancer researchers and already armed with a wealth of knowledge that would ultimately guide our son's treatment. Histology concluded Conor's cancer was high-risk and aggressive. The recommended treatment was a standard-of-care bone marrow transplant. However, a limited number of biomarkers suggested otherwise. We chose a less intense chemotherapy regimen over the recommended bone marrow transplant. Today, Conor is a healthy, active 10-year-old without any detectable side effects from his treatments.

As cancer researchers, we were painfully aware of published biomarkers that if available would have further assisted us in refining Conor's treatment protocol and promising less toxic, targeted therapies that had not yet made their way into the clinic. The development pipeline between academic research centers and the pediatric cancer clinics appeared to be non-existent.

The Nicholas Conor Institute (TNCI) was created to accelerate the development and implementation of innovative, safer therapies and treatment guidance diagnostics to empower parents and treating physicians.

Q: What is the challenge in pediatric cancer that The Nicholas Conor Institute helped address?
Despite only two drugs having been clinically developed and approved for pediatric cancers in the past 40 years, the overall five-year survival rate of childhood cancer patients has improved but only by increasing the dosage and optimally combining highly toxic, adult chemotherapy agents. Throwing the kitchen sink approach at childhood cancer may have increased the five-year survival rate but it doesn't come without a price. Over 90 percent of our approximately 350,000 childhood cancer survivors suffer from long-term side effects, and many will not survive beyond 30 years. Safe, targeted therapies are desperately needed to effectively treat pediatric cancer and increase the life expectancy of childhood cancer survivors.

When TNCI was founded in 2007, less than 4 percent of the National Cancer Institute (NCI) research budget was allocated to pediatric cancer, numerous childhood cancer foundations sponsored academic research projects in the hope that promising medical breakthroughs would be made, and the pharmaceutical industry was focusing on larger adult markets. The development pipeline for promising breakthroughs in pediatric cancer was non-existent. If promising discoveries were made, they would likely languish in the halls of academia without further investment. The notion that foundations support breakthroughs across the Valley of Death was not even considered.

Once realizing the Valley of Death was not on the childhood cancer foundations' and advocates' radar, TNCI began introducing the concepts of drug development to the community, pointing out the existence of the Valley of Death and the need to continue to support discoveries through pre-clinical development and into clinical trials. As a result of increased awareness and advocacy, NCI's funding for pediatric cancer has not increased but has become directed towards translational and pre-clinical development, foundations have begun directing a portion of their funds to support the next stage of development, and industry is beginning to show considerable interest in investing in pediatric cancer opportunities. Although the childhood cancer community now recognizes the need to support drug development, the community as a whole has not yet fully embraced current models that many TRAIN organizations have adopted.

Q: TNCI is unique in that it isn't a traditional foundation nor a stand-alone research entity. Describe your business model.
The Accelerator of Cancer Treatments in Children (TACTICTM) is TNCI's mode of operation. TNCI acts as an innovation scout on the hunt for promising pediatric cancer discoveries either languishing in the halls of academia or shelved in industry. Once identified, TNCI collaborates with industry partners to move the technology forward and/or identifies funding to support its next stage of development.

While implementing TACTIC, we discovered barriers that prevent promising discoveries from moving forward into the next stage of development irrespective of the technology. The inability to simply access high-quality tumor samples and patient outcome data in a timely manner is perhaps the largest barrier we've encountered. To overcome this barrier, TNCI in collaboration with multiple technology companies are exploring cloud-based, virtual tumor sample and data repository technology.

The lack of significant funding from a single source for translational projects and transformative initiatives is another barrier. To overcome this obstacle, TNCI proposed a CURE Partners Program, a Collaborative Umbrella of Research-granting Enterprises, at the 2012 Partnering for Cures Conference. CURE Partners is similar to the Leukemia and Lymphoma Society's Therapy Acceleration Program, with the nuance that CURE Partners investment capital would be derived from multiple foundations pooling their resources into a portfolio of projects and initiatives.

Q: What do you consider TNCI's greatest accomplishment?
Although TNCI was initially created to accelerate the development of promising discoveries for pediatric cancer into the clinic, we quickly realized the childhood cancer community overall is laser-focused on exclusively funding research that may result in a promising discovery rather than further supporting their research investment through the development pipeline. Perhaps TNCI's greatest accomplishment to date is accelerating the childhood cancer community's understanding of the Valley of Death and the need to support the entire innovation pipeline from research through development.

Q: What are your top goals over the next year?
In the coming year, TNCI's goals are to:

  • Secure commitments from the childhood cancer community for a collaborative investment venture, CURE Partners, to provide adequate funding to further support discoveries through the Valley of Death and into clinic trials
  • Design and initiate platform development to directly address tumor sample and patient outcome data procurement issues
  • Advocate for legislation that would incentivize the medical industry to devote additional resources towards rare diseases, including childhood cancer