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"Let's bridge the funding gap so we can finally help our kids get the medicine they need to beat this cancer beast called rhabdomyosarcoma."

Charles Keller, M.D.

Scientific Director
Children's Cancer Therapy Development Institute

On the Children's Cancer Therapy Development Institute website, Dr. Charles Keller explains below what personally motivates him to persevere in the field of pediatric cancer research.

Every night I read to my daughters at bedtime, then head back to the lab for a second shift. One night, my youngest daughter, then 4 and a half years old, asked, “Daddy, you leave for your research every night...but have you ever saved anyone’s life from the research you do in your lab, ever?” My answer was, unfortunately, “No. Not from my research.” I could have said that nobody really ever has, but the path from basic science to clinical applicability seemed too complicated to explain. It got me thinking: wouldn’t children with cancer ask the same question, too?

Fast forward to Spring 2014, at the annual Children’s Oncology Group meeting. The Chair announced dramatic cuts in the National Institute of Health’s budget for the COG...suggesting that not every childhood cancer could have an open clinical trial: only the ones with preclinical justification. The standard approach (inserting adult cancer drugs into trials for children) simply hadn’t worked. At the same meeting, the NCI announced that as a result of the sequester, the budget of the Pediatric Preclinical Testing Program was cut earliest, and deepest, of any NCI program (by 40 percent)! 

Amongst colleagues who were leaders in pediatric oncology at academic centers, the feeling was that preclinical testing of basic science findings, to move exciting discoveries to clinical trials, was too tedious and narrow-scoped for university laboratories and government programs. As a result, the best and brightest scientific discoveries for childhood cancers never actually make it into the clinic. Rare cancers are the hardest hit, with survival rates remaining stagnant for decades. Knowledge that could save kids’ lives simply languishes in this black hole: the preclinical gap. 

By chance, my reading material for the airplane ride home was A Life Decoded, the book by J. Craig Venter. In this story of the first group to sequence the human genome, Dr. Venter achieved remarkable speed and cost efficiency by “going outside the box” of academia. Curious, I drove straight from the airport to a biotech incubator. Renting a 250 sq ft lab space for per year: a mere $10,500.

The “what if’s” began: what if we could change the paradigm of research grants leading to publications (leading to more grants and papers, but never tangible results)? What if we could bridge the preclinical gap as a mission...with scientists partnering with families to achieve the cures they so desperately desired?  What if science driving drugs into the clinic existed as a singular mission?

My research team and I simply wanted to know how a non-profit biotech could answer my daughter’s question. The result is the Children’s Cancer Therapy Development Institute (www.cc-tdi.org). We continue to pursue many of the same publication and grant funding goals as we had in academics (we have been a continually NIH-funded laboratory for 15 years, with our most recent NIH/NCI R01 (4th percentile) having begun in August 2015)... but we also value our pharmaceutical partners, give parents a seat at the table, and really listen to the clinical trialists. All in the name of converting scientific discovery into clinical trials for children with rare and underserved cancers.

Summary

Children's Cancer Therapy Development Institute, (cc-TDI), has the autonomy to discover, research and develop drugs that the will address the complex genomics of rhabdomyosarcoma. With financial support cc-TDI completes the bridge of taking drugs to trial by delivering the needed proof of concept to pharmaceutical companies. Financial contributions are critical to cc-TDI's research and discovery.

Why is this important?

By shaking up the box and supporting this non profit bio-tech, the Children's Cancer Therapy Development Institute scientists that have already proven their investment in improving rhabdomyosarcoma outcomes can target their research and discovery.  Successes are then delivered to pharmaceutical companies that will sponsor clinical trials leading to better outcomes for our children.

Who will benefit?

Ultimately, children with the frightening cancer diagnosis of rhabdomyosarcoma will benefit with better, less toxic, targeted therapies that extend their lives. All funds raised will go to cc-TDI's specific rhabdomyosarcoma research.

Budget

The funds raised will be used by cc-TDI on their rhabdomyosarcoma research.