For more than 40 years the main treatment for pediatric osteosarcoma has not changed. Patients with this type of aggressive bone cancer, most often diagnosed in teens, are in desperate need of new options. Fewer than 30% of patients survive when osteosarcoma has spread beyond the primary tumor at diagnosis.
To make a significant impact for kids fighting osteosarcoma, five funding partners have banded together with St. Baldrick’s to support a new grant – The Fight Osteosarcoma Together (FOT) Super Grant. Today we are excited to announce that the recipient of this three-year, $1.5 million grant is Dr. Patrick Grohar, at the Children’s Hospital of Philadelphia. The goal: a clinical trial to test a new treatment within three years.
Patrick J. Grohar, M.D., Ph.D.
“Our group is honored and humbled to have been selected for this award,” says Dr. Grohar. “I have dedicated my life to the care of patients with bone tumors. As you know, we are still using the same, decades-old chemotherapy regimen to treat patients with osteosarcoma.”
Dr. Grohar’s goal is to discover targeted therapies for osteosarcoma. One of the major challenges in treating osteosarcoma is the presence of different genetic mutations that drive tumor growth in individual patients. However, a significant percentage (12-39%) of these tumors share a common genetic change known as an amplification in a gene called MYC, which has been linked to aggressive disease and poor prognosis.
To shed light on this specific subset of osteosarcoma, Dr. Grohar and colleagues will investigate the role of the MYC gene in the development and aggressive nature of the disease. Working with Vuja De Sciences, this team will identify, characterize and prioritize the development of MYC pathway targeting therapies. Dr. Grohar plans to find compounds that are not only tailored to MYC-amplified osteosarcoma but are also effective treatment options.
The team will explore various approaches, such as utilizing these compounds alone, in combination with chemotherapy, or specifically targeting metastasis prevention (the spread of cancer). “Using models, we will test drugs with the goal of generating data that would provide rationale for the development of a pediatric osteosarcoma clinical trial of the most promising drugs identified” said Dr. Grohar’s colleague, Dr. Filemon Dela Cruz, at Memorial Sloan Kettering Cancer Center.
By delving into this distinct subgroup of osteosarcoma and collaborating with other scientists, Dr. Grohar hopes to discover new ways to treat MYC-amplified osteosarcoma and give patients a better outcome.
Meet the funding partners
Michael & April Egge
Michael Egge, founding member of the Osteosarcoma Collaborative, began fundraising to improve treatments and outcomes for children with osteosarcoma when daughter, Olivia, was diagnosed in February 2017.
Michael Egge is a funding partner and one of the driving forces behind this project. “Watching your child survive osteosarcoma, with all its challenges and uncertainties, shook me to my core,” he says. “Olivia’s experience, and that of the many other children and families I have met along the way, inspire me to stay focused and dig in, because we can and we must do better for children confronting this disease. I am grateful for Olivia’s survival, and awed by what she has taught me about resilience, perseverance, and faith, and I pray and hope that we all persevere, lean in, and fight for a cure through creative, large, competitive grants like this.”
Mr. Egge had previously partnered with St. Baldrick’s and the Osteosarcoma Collaborative in 2018 to fund a $1.35 million grant for Dr. Alex Huang.
The Osteosarcoma Collaborative (OSC), which Mr. Egge co-founded, is made up of passionate and dedicated parents, friends and family. It brings together data, researchers, patients and families to find new cures for osteosarcoma. The organization focuses on advocacy, fundraising, education and awareness, and supports new treatment options for osteosarcoma patients. To date, OSC has raised over $1.65 million for osteosarcoma research.
Founding member and chairperson, Miriam Cohen, lost her son Austin to the disease in 2017. “Austin did nothing wrong, he methodically thought through and endured all of the recommended treatments, always offering someone a smile,” she says, “but the lack of medical research failed him.” Living through the realization that there were no medical treatments available for Austin motivated her to turn her grief into change by co-founding OSC in 2017 and most recently, spearheading the group’s fundraising for this grant. Christiane Robbins, the chief scientific advisor for the group, is a Scientific Review Officer for the National Institutes of Health, whose 18-year-old son David passed away in 2017 to pediatric osteosarcoma. Christiane put her professional background to work as she provided guidance with the scientific review process for the grant.
Fueled by her son’s relentless positivity, OSC Board member Sunita Krishna and the Team Arnav Foundation are supporting OSC in reaching their Fight Osteosarcoma Together Super Grant goal. Founded to honor her son’s legacy, the foundation supports groundbreaking research initiatives. Led by Arnav’s twin brother, Dhruv, the organization brings together researchers and innovators united in their pursuit of scientific breakthroughs. “My brother was a beacon of hope, let’s follow his lead and find the cure.” So far Team Arnav has funded close to $150K in research.
In 2019, a group of determined moms of children affected by osteosarcoma came together to form Battle Osteosarcoma, an organization helping to blaze new trails in the fight against this devastating disease. In memory of Charlotte, Dylan, and Tyler, three teens from a small community who passed away from osteosarcoma, Battle Osteosarcoma is committed to raising funds to support research for better treatment options and outcomes for osteosarcoma patients and their families. The organization is also focused on investing in the next generation of researchers. Shortly after Battle Osteosarcoma was formed, they teamed up with St. Baldrick’s to fund a $1.35 million research grant to support the Sweet-Cordero Lab at the University of California, San Francisco to study a precision oncology approach for relapsed osteosarcoma. The impact of Battle Osteosarcoma’s funding to Dr. Sweet-Cordero includes supporting a clinical trial, testing hundreds of drug combinations, developing new models and collaborating with other researchers globally. The results have been transformative and brought much needed attention to osteosarcoma. To date, Battle Osteosarcoma has raised over $2.35 million for osteosarcoma research, thanks to the incredible support of their community.
Battle Osteosarcoma at their Cocktails for a Cure fundraising event
Seeing the exciting outcomes in the Huang lab and Sweet Cordero lab, Mr. Egge asked Battle Osteosarcoma, the Osteosarcoma Collaborative, and other like-minded donors to work with St. Baldrick’s to make another sizable game-changing pediatric osteosarcoma grant possible, using the same model: a grant selected through a winner-take-all competitive process based strictly on scientific review. This model has a proven track record in translating lab science to clinical trials and patient benefit, with other foundational learning along the way. That made it easier for Mr. Egge to persuade other leading organizations to come together and make The Fight Osteosarcoma Together Super Grant.
New partners joining to fund this grant are CureSearch for Children’s Cancer and the Zach Sobiech Osteosarcoma Fund of Children’s Cancer Research Fund.
CureSearch for Children’s Cancer
In addition to funding, CureSearch played a vital role in the review process, by assessing the clinical translation and drug development potential of the grant. Composed of esteemed leaders from pharmaceutical, biotech, and clinical research organizations, the CureSearch Industry Advisory Council actively champions pediatric cancer drug development, forwarding CureSearch’s mission to drive targeted and innovative research with measurable results in an accelerated time frame.
“Real progress in childhood cancer research requires collaboration, so we are honored to be a funding partner on this award,” said Holly Zink, Director of Research and Programs at CureSearch. “Dr. Grohar’s project aligns with our laser focus on addressing critical, unmet need in pediatric cancer treatment. We look forward to seeing the impact of this groundbreaking research on pediatric osteosarcoma patients who are counting on novel treatment options.”
The Zach Sobiech Osteosarcoma Fund
The Zach Sobiech Osteosarcoma Fund, which supports groundbreaking osteosarcoma research nationwide through Children’s Cancer Research Fund, is also a funding partner. Before Zach passed away from osteosarcoma in 2013, he and his family started the Zach Sobiech Osteosarcoma Fund, in the hopes that his legacy would make cancer treatment safer and more effective for kids diagnosed after him. His hit song “Clouds,” written about his experience of living while knowing he was dying, went viral shortly before his death – proceeds from Zach’s music also support his fund. In 2023, Zach’s Fund topped $4 million raised since the teen singer/songwriter passed away 10 years ago. To date, it has enabled 16 new discoveries about the genetics of osteosarcoma, brought a national clinical trial to fruition, and now will support The Fight Osteosarcoma Together Super Grant.
“I often think about a moment during Zach’s fight where he was sharing a hospital room with a young boy, who also had cancer,” said Laura Sobiech, Zach’s mom. “He said to me, ‘Mom, if I had to die so that little boy could live, I would.’ Ever since, that’s been the goal of the Zach Sobiech Osteosarcoma Fund – to use Zach’s story and legacy to fund research that will save the lives of kids with osteosarcoma.”
With the collaboration of these funding partners and a stellar group of researchers led by Dr. Grohar, the St. Baldrick’s Foundation is excited about this opportunity to advance pediatric osteosarcoma research, paving the way for brighter futures for children battling this disease.
“We have lit a fire,” says Mr. Egge. “Let’s add more fuel and make it rage for discovery, innovation and a cure.”
Interested in teaming up with St. Baldrick’s to support a specific type of research?
Please contact Becky Chapman Weaver, Chief Mission Officer, to explore possibilities.
Email firstname.lastname@example.org or call 626.792.8247, ext. 212.
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