Last summer, I received an important message from Jeff Toretsky, M.D., Chair Emeritus of the CCF Scientific Advisory Board.
It was an important reminder – why CCF continues to do what we do every day.
The message noted:
“It is a pleasure to share this paper with you that CCF had a significant role in the earliest parts of this project. Without Shirley’s support from roughly 2000-2004, this clinical trial would not have happened. This early, critical, support allowed us to make progress on an idea most said was impossible. Scientists and doctors called our target ‘undruggable’ and there was no belief that our idea would get traction. This paper is the culmination of the past almost 25 years of work. I am grateful for the contributions and continued support of CCF for improving the care of pediatric, adolescent, and young adults with cancer.”
In the article, published in the Journal of Clinical Oncology, the flagship journal of the American Society of Clinical Oncology, CCF is acknowledged as an early and critical funder in the success of a clinical trial:
“We are grateful and thankful to all study participants and their families for willing to participate in, to our knowledge, a first-in-human clinical trial. The Children’s Cancer Foundation, Inc. supported J.A.T. in the earliest phases of experiments that led to the discovery of YK-4-279, without which this clinical trial might not have happened; for this, we are grateful.”
This phase I/II clinical trial provides proof of concept that fusion oncoproteins can be targeted with manageable side effects. This research demonstrates that the challenges in biomarker development and pharmacologic delivery can be overcome, resulting in either the preferable oral dose formulation or less frequent IV dosing that is easier for patients to receive.
Dr. Toretsky is leader on the team who discovered YK-4-279 and part of the group that moved the clinical trial forward to help treat Ewing Sarcoma. While I do not fully understand the science behind the success of this drug, I do understand the importance of investing early and investing often in pediatric cancer research.
While survival rates for many pediatric cancers have improved over the past 40 years, survival rates for many of the rare cancers has not kept pace. When we talk about children, one death is one too many, which is why CCF continues to support critical research and vital programs to ensure that kids, and their families, facing a diagnosis of cancer, are provided with treatments and cures that were not around a generation ago.
Many pediatric cancer patients have benefitted from Dr. Toretsky’s early advancements in pediatric cancer research. His commitment to CCF is unwavering. He enthusiastically led the CCF Scientific Advisory Board from 2015-2024 and launched the CCF Research Symposium in 2016. CCF is honored to announce that we will present Dr. Toretsky with the 2025 CCF Hero Award at our annual Gala on November 1.
CCF just launched our 2025 Call for Proposals, inviting local researchers to apply for CCF funding to advance their own pediatric cancer research. Our intention is that investing in research will lead to the next clinical trial.
CCF relies on the generosity of our community to ensure that these grants continue. Without research, the promise of a healthy, cancer-free future is not guaranteed. We must change that. Together, with Dr. Jeff Toretsky on our team, we will.
