By Bill Thomas | April 29th
The concept of “legacy” is a familiar one for many in the pediatric cancer community.
For parents of young cancer patients, a happy, healthy child who has overcome their illness is the greatest legacy they can leave behind. For childhood cancer survivors, helping others to weather the same trials and tribulations they experienced is a way to foster their own positive legacy. For cancer researchers, legacy is an ongoing process; their work is built upon the data and findings of those who came before them, and their own discoveries will help serve as a foundation for other researchers to build on in the future.
Here at Pediatric Cancer Research Foundation, legacy is about the long-lasting relationships we nurture with our researchers and the game-changing, life-saving innovations those relationships facilitate. Nothing better exemplifies our commitment to sustaining momentum and accelerating progress toward improved treatments and potential cures than the PCRF Legacy Researcher Grant.
Of the various grants we provide, only the most promising initiatives receive the Legacy Researcher Grant. It is awarded to researchers who have received PCRF funding for at least five years with clear evidence of significant milestones and outcomes in pediatric cancer research. These are researchers whose work demonstrates consistent progress and impactful outcomes, with a proven potential for future breakthroughs.
This year, two researchers who have demonstrated the kind of advancements necessary to earn a PCRF Legacy Researcher Grant are Dr. Brian Crompton at Dana-Farber Cancer Institute and Dr. Elliot Stieglitz at UCSF Benioff Children’s Hospitals.
Drs. Crompton and Stieglitz are among the best and brightest researchers working in the field of pediatric cancer today. We believe that their work is yielding tangible results that will contribute to a lasting legacy we can all be proud of.
Dr. Elliot Stieglitz: Risk-Stratifying Patients to Reduce Toxicity & Improve Outcomes
Juvenile myelomonocytic leukemia (JMML) is a rare but aggressive form of pediatric blood cancer with only one currently known curative treatment: allogeneic hematopoietic stem cell transplantation (HSCT). This intensive and potentially dangerous treatment has numerous short and long-term side effects and is only effective in about 50% of cases.
Dr. Elliot Stieglitz knew there had to be a better option. There had to be a way to avoid putting so many young cancer patients through such a difficult and risky therapy, a way to identify which patients would be most likely to benefit from it and which patients would be better suited for less invasive, less toxic therapies.
With funding support from PCRF, in 2019 Dr. Stieglitz began researching the use of DNA methylation for risk stratification of children diagnosed with JMML. In 2024, Dr. Stieglitz was able to progress his PCRF-funded research into a phase 1 clinical trial. Following the success of that trial, Dr. Stieglitz is now in the process of overseeing a National Institutes of Health-backed phase 2 clinical trial.
“It’s the first time that any trial with JMML has given different intensities of therapy to different patients. It used to be that treatment was ‘one size fits all,’ but now we’re tailoring the intensity to patient-specific risk factors” Dr. Stieglitz says. “There is a tremendous opportunity for us to personalize the type of treatments that we offer based on biomarkers.”
In addition to his DNA methylation research, PCRF has also funded Dr. Stieglitz’s work with the drug trametinib, a MEK inhibitor that has shown promise as a less-intensive treatment option for JMML patients who haven’t responded to other forms of treatment.
“If this drug works, it’s not going to be just for JMML. There are many cancers in pediatric oncology that are driven by RAS mutations,” Dr. Stieglitz says. “If we can show that this works in a purely RAS-driven disease, even though it’s rare, we can then apply those findings to more common diseases.”
Though hopeful about the future, Dr. Stieglitz is quick to note that recent NIH policy changes and cuts to federal funding only reinforce the vital role that philanthropic organizations like PCRF play in advancing pediatric cancer research.
“Working with PCRF is a rewarding experience. There is a personal touch to it that makes me feel like I’m part of the PCRF family,” Dr. Stieglitz says. “It means so much that PCRF continues to support our lab’s work and that we’re able to leverage the resources they have provided to build tests and develop clinical trials and give back to the greater pediatric cancer community.”
Dr. Brian Crompton: Using Liquid Biopsies for Early Detection & Treatment Monitoring
One of the most important tools in an oncologist’s toolkit is the biopsy. Biopsies make it possible for doctors to accurately diagnose cancers, determine treatment options, and monitor disease progress. Unfortunately, traditional biopsies are invasive, requiring either surgery or the insertion of an endoscope or needle so that doctors can acquire a sample of the patient’s tumor.
In recent years, liquid biopsies – which require only a blood draw – have emerged as a promising alternative to traditional biopsies. Liquid biopsies are safer, simpler, quicker, and less painful for patients; however, liquid biopsies remain a relatively new technology, with significant research and development necessary before their full potential can be realized.
When Dr. Brian Crompton received his first PCRF grant award in 2014, he was an emerging investigator exploring new treatment approaches for Ewing sarcoma and other pediatric bone and soft tissue cancers. In 2018, Dr. Crompton’s PCRF-funded research proved that ctDNA, small fragments of tumor DNA which circulate in a patient’s peripheral blood, could be used as biomarkers enabling liquid biopsies to reliably detect bone and soft tissue cancers.
Now, Dr. Crompton’s breakthrough promises not only to change the way oncologists diagnose such cancers, but also how they treat them.
“We’ve never had really good biomarkers for most pediatric solid tumors before this. We just knew that some patients were cured and some were not. So, over the last 30-40 years, we just kept intensifying therapy for everyone,” Dr. Crompton says. “Using ctDNA as a biomarker could help us determine how likely a patient is to be cured with a specific treatment, which would enable us to subject them to less intense levels of therapy while still curing them.”
In 2025, Dr. Crompton’s discovery led to the creation of BrightSeq, a collaborative research and testing initiative of the Dana-Farber Cancer Institute, Boston Children’s Hospital, and Broad Clinical Labs. The purpose of BrightSeq is to design, validate, and implement a suite of novel clinical diagnostic and prognostic assays specifically tailored to rare pediatric cancers.
“Ten years from now, I believe it’s going to be routine that liquid biopsies are used to help doctors make critical decisions about how to treat pediatric cancers,” Dr. Crompton says. “It’s very hard to advance new technologies like this without the support of donors and organizations like PCRF. This research could not have happened without PCRF’s help.”
Are you interested in becoming part of a lasting legacy that is reshaping how pediatric cancer patients are treated and cared for? Would you like to help facilitate critical research projects like those being overseen by Dr. Stieglitz and Dr. Crompton? If so, please consider donating to PCRF’s Powering Research Fund, which enables us to continue supporting work that reduces toxicity, improves patient quality of life, boosts survival rates, and accelerates cures.
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