Osteosarcoma is the most common type of bone cancer, impacting between 400 and 600 children, adolescents and young adults per year in the United States. It has historically been a very complicated cancer to study, leaving many researchers and advocates bewildered and discouraged. However, in recent years there has been strong philanthropic and scientific investment into this cancer, and the progress is paying off.
A common obstacle for the treatment of many sarcomas is the protective shield surrounding a tumor known as the Tumor Microenvironment (TME). This complex network of cells manages to guard against the impact of many chemotherapy and immunotherapy drugs.
We now understand that a particularly aggressive subtype of Osteosarcoma is marked by the presence of c-MYC, and a genetic mutation known as c-MYC amplification pertains to poor prognosis. c-MYC in Osteosarcoma cells can be enhanced by high levels of an immune suppressor molecule called TGF-ß. If scientists could inhibit the production c-MYC by disrupting TGF-ß signaling in Osteosarcoma, it’s possible that immunotherapy approaches could be effective in fighting this devastating disease, even in c-MYC amplified Osteosarcoma.
This SDF funded project is focused on a combination of drugs intended to target the problem of TGF-ß in c-MYC amplified osteosarcoma with lung metastasis. Early studies have been optimistic, and we believe this approach is very worthy of our support, and these patients are in desperate need of more options.
Dr. Alex Huang is the Principal Investigator on this project. He is a PhD and MD, and the Director of the Center for Pediatric Immunotherapy at Rainbow Babies and Children’s Hospital in Cleveland Ohio. His reputation and accomplishments are outstanding. He cares deeply about young people with sarcomas, and we are very proud to be working with him.