Little Warrior Foundation Awards $500,000 in Ewing Sarcoma Research Grants
In year one, we’re swinging for the fence in hopes of a grand slam. In reality, we’ll take a single home run. Heck, we’d get hit by a 95mph pitch if that’s what it takes to move the needle forward for kids and families impacted by Ewing Sarcoma (EwS). But luckily -- thanks to our donors and dedicated researchers -- we’re loading the bases with high-potential research projects.
Our singular focus is to find a complete and permanent cure for Ewing Sarcoma. We believe these projects can have a positive impact now, the near future, and in the not-too distant future for our children. Collectively, we have granted $500,000 to these 4 different research initiatives in our first year as a foundation:
Preventative Medicine - Liquid Biopsy (ctDNA) Test
Dana-Farber Cancer Institute | Brian Crompton, MD and David Shulman, MD - $150,000
We don’t know what they feed the doctors at Dana-Farber, but in addition to being cutting-edge practitioners and researchers, they are extremely approachable and down-to-earth human beings. In that regard, both Dr. Crompton and Dr. Shulman check these boxes.
On the heels of Foundation One’s new FDA-approved liquid biopsy (CDx) test and at the request of several motivated EwS families, Dr. Crompton and Dr. Shulman will study and track circulating tumor DNA (ctDNA) levels over time for patients currently in remission.
The central hypothesis is that those patients with detectable ctDNA at the end of therapy or one year out from treatment (equating to higher levels of minimal residual disease), will have inferior outcomes as opposed to those with undetectable levels of ctDNA. And if this correlation can be made, then this test may have practical applications of being able to detect relapse sooner than standard imaging, by monitoring ctDNA levels via liquid biopsy over time.
Across all cancer types, it has been well documented that early detection of cancer leads to superior treatment outcomes, translating to saved lives. On the merits of early cancer detection, one prominent oncologist and advocate proclaims “early detection is the most compassionate and humane solution for the cancer problem. The future is in preventing cancer by identifying the earliest markers of the first cancer cell rather than chasing after the last” - Dr. Azra Raza (MD), The First Cell.
This study will utilize Foundation One’s liquid CDx test as a means of providing real-time data back to patients and their respective doctors for clinical decision-making. Blood samples will also be collected for testing validation to be carried out in-house by Dr. Crompton’s lab at Dana-Farber Cancer Institute (this data will not be shared with patients and is only for study purposes).
This study is an amendment to an ongoing Dana-Farber study (DFCI 18-138) that is currently tracking ctDNA levels in patients during treatment. The new amended portion of the study will be open to all (localized and metastatic disease), regardless of location in the US, with a starting point of at the end of treatment (start of NED). Followup samples and testing will be done every 6 months for 2 years.
When: More details are coming soon, expected start date late spring / early summer 2021. The plan is to coordinate a video webinar with Dr. Crompton and/or Dr. Shulman to help kick-off this study for the EwS community. Patient families, if you would like to be invited to this webinar, please email info@spadasarcomafoundation.org
SBRT and ImmunoOncology
Cleveland Clinic - Pete Anderson, MD, PhD and Erin Murphy, MD - $100,000
Practically the whole Ewing’s community knows Dr. Pete Anderson at the Cleveland Clinic, as he does over 150 virtual visits a year for the Sarcoma community at large and his name frequently shows up on FB discussion boards regarding second opinions. Truly, it is our belief that he never sleeps.
In collaboration with his colleague, the equally talented and caring Dr. Erin Murphy (Radiation Oncology), they are working on a team approach to optimize the use of Stereotactic Body Radiotherapy (SBRT) as an aggressive and impactful form of local control. Dr. Murphy sees SBRT (similar to gamma knife radiosurgery technology) as an underutilized tool in the pediatric Sarcoma population, especially for those with isolated sarcoma tumors that form in hard to resect locations such as the spine and lungs. In addition to this novel local control strategy, the team will measure and monitor various lab panels before, during, and after radiotherapy to help track and better understand patient response. The team will also compile these responses in a new database system (REDcap) to help better understand the efficacy of this treatment relative to other therapies over time.
Big picture, this seed money sets the stage for proof of concept in controlling various biomarkers, like inflammation, during SBRT, promoting Immunological Cell Death (ICD) - a concept which Dr. Anderson theorizes is key for a successful ImmunoOncological intervention at the tailend of treatment. And according to this theory, this ImmunoOncological intervention is necessary to help boost the immune system for proper surveillance to help keep Ewings at bay (hopefully for good). Further details will be outlined in a future post.
When: SBRT happening now at Cleveland Clinic. ImmunoOncological intervention coming soon.
Precision Medicine Model
Beat Childhood Cancer (BCC) - Giselle Sholler, MD - $100,000
We’ve teamed up with a gritty set of parents at the BCC Foundation, and in conjunction with the Sam Day Foundation, are supporting the launch of BCC’s precision EwS medicine model at Atrium Health / Levine’s Children (Charlotte, NC). In a relapsed setting, and as outlined in a recent review article by some leading Ewing specialists, “the care of patients with relapse EwS remains a significant challenge and must be individualized for each patient’s disease and goals of care. Outcomes for this group of patients remain poor, with less than 25% being long-term survivors.”
Truly, in Dr. Sholler’s vision, treatment for relapsed patients can be individualized. The central idea to this therapy is to treat patients according to their respective tumor biology in a molecular guided manner. For this to happen, tumor samples will be genetically analyzed by next-generation sequencing for mutations, RNA sequencing, and epigenetically assessed (lab work by Sema4). From this data set, an individualized tumor map will be created, with the aim of finding and (if deemed appropriate by the tumor board) using druggable targets to combat EwS. In parallel, another branch of this research group, will compile the data set in a database to assess the tumor variability amongst all patients, with some tumor samples being sent back to the lab for further testing in mice models. The goal of this lab work is to discover new and novel treatment options for Ewing Sarcoma that could eventually lead to new clinical trials.
If the potential of this therapy seems too good to be true, take a look at BCC’s track-record in the neuroblastoma space.It’s a story (should be a movie) about how a truly determined set of parents (now the BCC Foundation) and one bold and daring doctor, can make significant strides within a disease space by using true grit and gumption! We are hopeful that Dr. Sholler can have similar success for our children with EwS.
Learn More: The IN:Formation Project
SISU Maintenance project
Children’s Cancer Therapy Development Institute - Charles Keller (MD) and Jinah Kim (PhD) - $150,000
Dr. Keller and Dr. Kim remain busy with their work on the SISU Maintenance project. After successful in-vitro (latin for “in glass”) tests, they have progressed to in-vivo testing in mice models. For an in depth discussion on this project please see our previous blog post.
When: Now
A special thank you to our Medical Advisory Board for helping us vet proposals and dig into the details.
In closing, thank you to our motivated and generous donor base for making this a reality! Without you, we couldn’t put this money to work. Of considerable note are the contributions made by the “Gus Tough” Frank Family, Burton Family, Zimbal Family, and 1-Million-4-Anna.
With Hope and Warrior Spirit,
- Team Little Warrior