By Robyn Spoon, CEO/Founder of Elevate 

   The first official event I attended as a representative of Elevate was the Children’s Cancer Therapy Development Institute’s (cc-tdi) Wilms tumor and Rhabdomyosarcoma (Rhabdo/RMS) Nano-Course in Beaverton, Oregon just outside of Portland in August. Cc-tdi is a non-profit independent laboratory focused on developing therapies for children’s cancers, like RMS and Wilms. It was an easy decision to attend once I saw that the week would be focused on the two diseases that drive our work at Elevate: RMS and Wilms.

   Charles Keller, M.D. and Chief Scientist of the lab, along with the amazing team at cc-tdi, brought together leading scientists, pathologists, therapy-developers, industry partners and families for an intense course in understanding these two complex children’s cancers as well as the therapy development pipeline! I’ll never forget the first day when Dr. Keller told the presenters to treat us all as if we had been through medical school and not to water anything down! I literally took 50 pages of notes during the two days of intense presentations.

  The various experts led educational sessions on a range of topics, and also allowed me to develop foundational relationships with many important experts in the field. I had the amazing opportunity to meet Dr. Mike Ortiz, of Sloan Kettering, and rockstar in the Wilms world. Dr. Ortiz spent his time sharing the challenges of bringing therapies into trial so our children have access to them. We also learned from Dr. Jack Brzezinski from Toronto SickKids about his work examining genetic markers for Wilms. Additional experts, like Dr. Sonja Chen, of Nationwide Children’s Hospital in Columbus, Ohio, provided us with a lab demonstration to help us understand the differences between the different forms of Wilms as well as RMS.
Others presented on the challenges of designing drug trials for kids with these diagnoses. Cc-tdi lab scientists demonstrated various stages of their work so that we could all better understand the tools, equipment and process being used to test compounds against different cell lines. We then heard from those who’ve spent their lives bringing therapies to market, like Lou Stancato of Indiana Biosciences Research Institute, as well as the President of Day One Pharmaceuticals. It was an amazing week of learning, understanding and coalition-building.

   The patient advocates broke into two teams: Wilms and RMS to identify a scientific need and create a roadmap to solve it! I was partnered with a team of high achieving and talented high school interns to tackle the RMS problem. Because of my years as an educator working with high school students in various research fields, I was pleased to see the lab developing future scientists with a passion for children’s cancer research! Over the course of three days, these students developed a plan to develop a PROTAC (Proteolysis Targeting Chimeric) to target the fusion found in my son’s cancer. I can only imagine a future when instead of giving those diagnosed with this disease the myriad of chemotherapy, they might instead receive this type of precision drug! Spending time with these young people lifted my spirits and gave me hope for a future filled with brilliant scientists readying for a time when the therapies for childhood cancer are targeted and less toxic!

   I also had the opportunity to spend time getting to know other advocates whose lives have been upended by childhood cancer. We are grateful that two of these advocates have joined our efforts within our Elevate Wilms Workgroup! At the end of the week, I felt more prepared than ever to tackle this complex, expensive, messy and challenging work of bringing new therapies forward for those diagnosed with a childhood cancer like RMS or Wilms. 

   Thank you cc-tdi for an amazing week of community-building and learning!