Childhood and Young Adult Cancer Research funded by the Liddy Shriver Sarcoma Initiative

$250,000 research grant for an international collaborative study on bone sarcomas: The grant brings together researchers from Austria, Italy and the United States in a two-year study of these rare childhood cancers.

 

Osteosarcoma and Ewing's sarcoma of the bone are aggressive cancers that tend to strike children and young adults. The major problem facing pediatric and young adult sarcoma patients is the ability of these tumors to spread (metastasize) and invade other organs. Metastasis is almost always associated with a poor prognosis, with an overall survival rate of just 10-20%.

Dr. David Loeb, who recruited the other investigators to the project, is motivated by his patients: "During the course of my career, I have seen the treatments for localized Ewing sarcoma improve markedly, and we are now curing many more patients than ever before; however, our ability to treat patients with metastatic disease is no better than it was when I was in medical school."

Dr. Heinrich Kovar also wants to see research lead to more effective treatments for patients: "Twenty-four years ago, I started research into Ewing’s sarcoma. By coincidence, my brother was diagnosed with this rare disease only a few months later. He asked me many questions about the pathogenesis of his tumor and future treatment options that I could not answer. Today, we know a lot more, but not enough to translate our findings into more effective treatments."

 

The Collaborative Model
The Liddy Shriver Sarcoma Initiative awards international collaborative grants like this one in order to increase the power of private donations and to accelerate research that has the potential to save and improve lives.

In separate interviews, each member of the research team mentioned the slow and frustrating nature of science. Each also expressed the hope that collaboration might bring about real results for patients. Dr. Katia Scotlandi explained: "Our lives are too short for science. I mean that research is difficult and frequently frustrating, and only a few significant results can be achieved in a doctor's lifetime. Collaborating with other scientists is a means to speed up the process and possibly to increase the number of significant results that we see."

Dr. Lawlor added, "Many aspects of the research will move faster because we can share our resources, intellectual and technical expertise. International collaborations are challenging, but with modern telecommunication tools and motivated investigators they can, and do, work. For rare tumors such as sarcomas, it is essential that research efforts cross traditional boundaries and borders."

Each investigator involved in this study has an established infrastructure of equipment, specimens, models, and supporting personnel for sarcoma research. In addition, each member of the research team has demonstrated a track record of productivity and accomplishment in studying pediatric sarcomas. Investigators include:

  • David M. Loeb, MD, PhD - the Director of the Musculoskeletal Tumor Program at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University. In this project, Dr. Loeb's team will test new compounds using osteosarcoma and Ewing sarcoma mouse models.
  • Elizabeth R. Lawlor, MD, PhD - an Associate Professor of Pediatrics and Pathology at the University of Michigan. In this project, Dr. Lawlor's team will study the role of Rho-MKL signaling in bone sarcoma metastasis and test novel compounds to block this signaling pathway.
  • Katia Scotlandi, PhD - the Chief of the CRS Development of Biomolecular Therapies at the Orthopaedic Institute in Rizzoli, Bologna. In this project, Dr. Scotlandi's team will work to understand the functions of CD99 in osteosarcoma and Ewing sarcoma.
  • Heinrich Kovar, PhD - a professor of molecular biology at the Children’s Cancer Research Institute in Vienna, Austria. In this project, Dr. Kovar's team will investigate the downstream gene regulatory pathway of Rho signaling and how it is influenced by the central mutation that characterizes Ewing’s sarcoma.

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