Through the Acceleration Initiative, CureSearch developed three Grand Challenges aimed at addressing the most challenging barriers in the pediatric oncology research field so that areas that are most critical to achieving breakthroughs in treatment and cures could be funded. $5 million was available to grants that addressed the barriers and met 3 pillars:
- addressed an unmet need
- accelerated science to clinical application; and
- provided an innovative and novel approach.
Grants were also required to demonstrate that the work would have measurable, patient-centric outcomes such that research was designed to meet specific benchmarks, identified on the front end. An RFA was issued in spring 2013 and 49 Letters of Intent were submitted. The Scientific Review Committee then invited 11 to submit full applications, which were reviewed blindly by the Committee. The following grants were awarded.
- Develop approaches for metastatic disease with broad applicability to multiple cancers and/or patient populations and that have a high probability of clinical application within 3 years.Mary Beckerle, PhD
Huntsman Cancer Institute at the University of Utah
A Novel Epigenetic Strategy to Treat Ewing Sarcoma
A team at Huntsman Cancer Institute that includes Mary Beckerle, PhD, Steve Lessnick, MD, PhD, Sunil Sharma, MD, and Alana Welm, PhD has received a $1.73 million grant from CureSearch to test a novel targeted treatment for Ewing sarcoma that hopefully will disrupt the cancer’s growth and spread. It is hypothesized that Ewing sarcoma spreads because the tumor cells have lost their ability to be tied together in the bone matrix where they begin. In a preclinical trial, Dr. Beckerle and her team will use a mouse model of metastatic Ewing sarcoma that mirrors the cancer in humans to test a small molecule targeted therapy they developed for its success in preventing the spread of cancer and toxicity of the treatment. If both are successful, the study will then move to a Phase I clinical trial in patients. Read more
- Develop technologies or therapeutic approaches to overcoming drug resistance of refractory cancers (i.e. newly diagnosed or relapsed) that have a high probability of clinical application within 3 years.William Weiss, MD, PhD
The Regents of the University of California, San Francisco
Overcoming Resistance in High Risk Medulloblastoma
A team of international researchers led by William Weiss, MD, PhD at the University of California, San Francisco, has been awarded a $1.88 million grant by CureSearch to investigate their hypothesis that drugs that reprogram (normalize) the epigenome, will block the inappropriate activation or silencing of DNA, thus normalizing gene expression. If this occurs, it will lead to improved outcomes for patients who do not respond to treatment. Read more
- Develop approaches to tackling critical scientific and therapeutic roadblocks that, if successful, accelerate the delivery of promising therapeutic interventions.
Kathleen Sakamoto, MD, PhD
Development of CD47 Monoclonal Antibody Therapy for Pediatric Tumors
A research team at Stanford University led by Drs. Kathleen Sakamoto and Irv Weissman will study the effects of an antibody targeting CD47 in children with various types of relapsed tumors. CD47 is a cell surface protein that is overproduced in many types of childhood cancers, and prevents scavenger cells, known as macrophages, from killing tumor cells. The CureSearch grant will support a Phase I clinical trial at Lucile Packard Children’s Hospital to study whether CD47 antibody is safe and effective in children with relapsed cancer. The hope is that the CD47 antibody will boost the body’s immune system by allowing the macrophages to “eat” the tumors cells, and prevent cancer from growing. If successful, this study will lead to a new approach to treat childhood cancer and both improve the overall survival and quality of life for children with cancer. Read more
From left to right: William L. Carroll, MD; Stuart Siegel, MD; Sid Midtra, PhD; William Weiss, MD, PhD; Mary Beckerle, PhD; CureSearch CEO Laura Thrall